Category Archives: Regenerative Medicine

Newswise The American Medical Society for Sports Medicine (AMSSM) has released a position statement on Principles for the Responsible Use of Regenerative Medicine in Sports Medicine.

This position statement provides sports medicine physicians with information on regenerative medicine terminology, a brief review of the basic science and clinical studies, regulatory considerations, and best practices for introducing the orthobiologic classification of regenerative therapies into their clinical practice.

The document is being published in the Clinical Journal of Sport Medicine, with accompanying editorial highlights published in the British Journal of Sports Medicine. Both are freely accessible on their respective websites.

Sports medicine physicians would benefit from decision-making guidance about whether to introduce orthobiologics into their practice and how to do it responsibly, said Dr. Jonathan Finnoff, the Chief Medical Officer of the United States Olympic and Paralympic Committee and the lead author of the statement. The information within this statement will help sports medicine physicians make informed and responsible decisions about the role of regenerative medicine and orthobiologics in their practice.

In 2019, the AMSSM Board of Directors established a Regenerative Medicine Task Force, with a subgroup charged to develop a regenerative medicine position statement. The Task Force brought together a writing group that included sports medicine physicians and scientists who are recognized leaders in bioethics, research, and regenerative medicine clinical applications to produce this statement.

The field of regenerative medicine, and the sub-classification of orthobiologics, involves a variety of therapies and techniques focused on the repair or replacement of damaged or diseased tissue to restore function. Despite these novel therapies being very attractive to sports medicine physicians and patients alike, this is a complex and controversial topic.

Common orthobiologics that are employed in research and medical practice are being combined under the umbrella of stem cell therapy in a manner that is confusing to both patients and the public, said Dr. Shane Shapiro, one of the lead authors of the statement. The need for scientifically validated treatments for non-healing orthopedic and sports conditions has increased interest in orthobiologics and other regenerative therapies to address existing treatment gaps.

The document contains brief discussions of the basic science, proposed therapeutic mechanisms of action, and clinical evidence related to regenerative medicine products, including uses for platelet-rich plasma and other cellular therapies. Additionally, the statement features sections regarding regulatory considerations and an in-depth portion on introducing regenerative medicine into clinical practice.

Ultimately, this AMSSM position statement on regenerative medicine advocates for the advancement of orthobiologic science, patient safety and education towards the responsible translation of regenerative therapies, said Dr. Kenneth Mautner, co-lead author of the position statement and an AMSSM Board member.

About the AMSSM: AMSSM is a multi-disciplinary organization of sports medicine physicians dedicated to education, research, advocacy and the care of athletes of all ages. The majority of AMSSM members are primary care physicians with fellowship training and added qualification in sports medicine who then combine their practice of sports medicine with their primary specialty. AMSSM includes members who specialize solely in non-surgical sports medicine and serve as team physicians at the youth level, NCAA, NFL, MLB, NBA, WNBA, MLS and NHL, as well as with Olympic and Paralympic teams. By nature of their training and experience, sports medicine physicians are ideally suited to provide comprehensive medical care for athletes, sports teams or active individuals who are simply looking to maintain a healthy lifestyle. http://www.amssm.org

Read more:
AMSSM Releases Position Statement on Regenerative Medicine in Sports Medicine - Newswise

-Achieved target enrollment in CTX001 clinical trials for beta thalassemia (TDT) and sickle cell disease (SCD); regulatory submissions planned for late 2022-

-Reported positive results from the ongoing Phase 1 CARBON clinical trial evaluating the safety and efficacy of CTX110 for CD19+ B-cell malignancies; enrollment continues, with potential registrational trial incorporating consolidation dosing expected to initiate in Q1 2022-

-Implementing consolidation dosing protocols for CTX120 and CTX130 clinical trials; enrollment continues, with top-line data expected to report in 1H 2022-

-Regenerative medicine and in vivo programs continue to progress and remain on track-

ZUG, Switzerland and CAMBRIDGE, Mass., Nov. 03, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the third quarter ended September 30, 2021.

The third quarter marked significant progress across our portfolio, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. With our partner Vertex, we achieved target enrollment for the CTX001 clinical trials in patients with beta thalassemia and sickle cell disease, which can support regulatory submissions in late 2022. Additionally, we demonstrated proof of concept for our allogeneic CAR-T platform with positive data from our CARBON trial of CTX110, which showed that immediately available off-the-shelf cell therapies can offer efficacy similar to autologous CAR-T with a differentiated safety profile for patients with large B-cell lymphomas. Based on these encouraging results, we plan to expand the CARBON trial into a potentially registrational trial in the first quarter of 2022. Furthermore, we hope to bring these transformative allogeneic CAR-T therapies to patients in outpatient and community oncology settings, enabling broad access."

Story continues

Recent Highlights and Outlook

Third Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $2,477.4 million as of September 30, 2021, compared to $2,589.4 million as of June 30, 2021. The decrease in cash of $112.0 million was primarily driven by cash used in operating activities to support ongoing research and development of the Companys clinical and pre-clinical programs.

Revenue: Total collaboration revenue was $0.3 million for the third quarter of 2021, compared to $0.1 million for the third quarter of 2020. Collaboration revenue primarily consisted of revenue recognized in connection with our collaboration agreements with Vertex.

R&D Expenses: R&D expenses were $105.3 million for the third quarter of 2021, compared to $71.0 million for the third quarter of 2020. The increase in expense was driven by development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs, as well as increased headcount and supporting facilities related expenses.

G&A Expenses: General and administrative expenses were $24.4 million for the third quarter of 2021, compared to $21.5 million for the third quarter of 2020. The increase in general and administrative expenses for the year was primarily driven by headcount-related expense.

Net Loss: Net loss was $127.2 million for the third quarter of 2021, compared to a net loss of $92.4 million for the third quarter of 2020.

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex CollaborationVertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About CTX110CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBONThe ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR THERAPEUTICS word mark and design logo, CTX001, CTX110, CTX120, and CTX130 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy, data and clinical progress of CRISPR Therapeutics various clinical programs, including CTX001, CTX110, CTX120 and CTX130; (ii) the status of clinical trials and preclinical studies (including, without limitation, the expected timing of data releases and development, as well as initiation and completion of clinical trials) and development timelines for CRISPR Therapeutics product candidates; (iii) expectations regarding the data that has been presented from our various clinical trials (including our CARBON trial) as well as data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials or to support regulatory filings; (iv) the actual or potential benefits of regulatory designations; (v) the potential benefits of CRISPR Therapeutics collaborations and strategic partnerships; (vi) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (vii) the sufficiency of CRISPR Therapeutics cash resources; and (viii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies including as compared to other therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates (including, without limitation, availability and timing of results and whether such results will be predictive of future results of the future trials); uncertainties about regulatory approvals to conduct trials or to market products; the potential impacts due to the coronavirus pandemic such as (x) delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; (y) the timing and progress of clinical trials, preclinical studies and other research and development activities; and (z) the overall impact of the coronavirus pandemic on its business, financial condition and results of operations; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

Three Months Ended September 30,

Nine Months Ended September 30,

2021

2020

2021

2020

Revenue:

Collaboration revenue

$

329

$

148

$

900,733

$

349

Grant revenue

495

1,331

Total revenue

$

824

$

148

$

902,064

$

349

Operating expenses:

Research and development

105,321

71,008

304,163

184,581

General and administrative

24,352

21,539

78,675

62,442

Total operating expenses

129,673

92,547

382,838

247,023

(Loss) income from operations

(128,849

)

(92,399

)

519,226

(246,674

)

Total other income, net

1,101

160

3,806

5,804

Net (loss) income before income taxes

(127,748

)

(92,239

)

523,032

(240,870

)

Benefit (provision) for income taxes

595

Read more here:
CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2021 Financial Results - Yahoo Finance

The top rated Orthopediatrician in Tucson, AZ are:

Tucson Orthopaedic Instituteis the largest and most modern multi-specialty orthopedic organization in southern Arizona. Tucson Orthopaedic Institutes Board-certified physicians have been setting the highest standard for orthopedic care in Southern Arizona since 1994. Their mission has always been to provide world-class care with positive outcomes.

After 25 years, they have developed into Tucsons largest and most comprehensive private orthopedic clinic. In Southern Arizona, they offer the most comprehensive orthopedic care available. They have a team of experts that can help you with orthopedic surgery, fracture care, and other orthopedic treatments throughout its six locations.

Products/Services:

General Orthopedics, Pediatric Orthopedics, Physiatry & EMG, Regenerative Medicine, Sports Medicine, Total Joint Replacement

LOCATION:

Address: 6320 N La Cholla Blvd #200, Tucson, AZ 85741Phone: (520) 382-8200Website: http://www.tucsonortho.com

REVIEWS:

Dr . Thompson is great and his staff is tremendous. I was running late for my first appointment and they were understanding and very professional. They handled my paperwork efficiently and with little delay. Once in the exam room, I was seen quickly. Dr. Thompson was very thorough in his questions and examination. He instructed me in-home exercises and allowed time for any questions I had. It was a pleasure to see him. William L.

Pima Orthopedic Physiciansfriendly and professional doctors and staff have been caring for patients and offering high-quality, innovative medical care for years. These board-certified orthopedic surgeons are determined to improve their patients lives through surgery and non-surgical treatment options. Their patients receive the best possible care since they specialize in regenerative and sports medicine, orthopedic surgery, and joint replacement.

Their team of skilled board-certified orthopedic surgeons uses a holistic approach to address each patients individual needs. They use cutting-edge diagnostic techniques to deliver a precise diagnosis, which includes a complete examination of bones, cartilage, joints, muscles, tendons, and ligaments. Total and partial knee and hip replacement are among the orthopedic therapies they provide. They also treat arthritis, tendinitis, bursitis, ligament, tendon, and cartilage injuries, as well as fractures, sprains, and strains.

Products/Services:

Independent Medical Examinations, Sports Medicine, Knee Preservation, Knee Replacement, Orthopedic Oncology, Hip Preservation, Hip Replacement

LOCATION:

Address: 1707 St. Marys Street, Suite 205, Tucson, AZ 85745Phone: (520) 624-0888Website: http://www.pimaortho.com

REVIEWS:

Pima Orthopedic is the best. I am in a scooter they even come down to the parking lot with a wheelchair to bring me upstairs for my injection. I have had 3 injections and I am so happy with my progress. My pain was so bad from arthritis in my left knee. Always crunching and grinding. I do not have to take ANY ibuprofen anymore. Thank you for giving me the opportunity to walk again. Trudy M.

Childrens Orthopedic Specialists was founded in 1998 as Southern Arizonas first full-time pediatric orthopedist and scoliosis practice. Fractures in growing children, scoliosis and other back problems, issues with growth and development in the shoulder,hips, knees, and feet, and concerns about function in these areas, as well as other muscular and bone treatments for children ages 0-18, are all areas of special interest to them.

The Pediatric Orthopaedic Society of North America is where their doctors belong. Dr. Vincent and Dr. Henderson, as well as their staff, are committed to providing your kid with the finest possible treatment. They collaborate with Southern Arizonas pediatricians, family doctors, emergency rooms, and other pediatric medical and surgical specialists.

Its practice encompasses everything from office consultations to sophisticated surgical procedures. Less invasive procedures and techniques are used whenever possible. Dr. Vincent, for example, was the first clinician in Arizona to commit to the Ponseti approach, a minimally invasive clubfoot therapy technique.

Products/Services:

Pediatric Orthopedic

LOCATION:

Address: 1605 E River Rd # 101, Tucson, AZ 85718Phone: (520) 296-5437Website: http://www.orthoforkids.com

REVIEWS:

The Drs here are patient with the kids & their families. They talk in layman terms so that everyone understands whats going on. Would recommend them to family & friends. Darmonica N.

Northwest Bone & Jointprovides a variety of services, including family medicine,internal medicine, cardiothoracic surgery, orthopedics, gastroenterology, general surgery, and more. Their doctors are at the center of Northwest Healthcares services, and they can help you coordinate your treatment with Outpatient Imaging, The Womens Center at Northwest, The Wound Care Center at Northwest, Outpatient Therapy, and Inpatient Rehabilitation, among other services. The health care professionals at Northwest Primary & Specialty Care are dedicated to keeping you and your family healthy.

Products/Services:

Orthopedics, Breast Surgery, Cardiology, Cardiovascular Surgery, Family Medicine, Gastroenterology, General Surgery, Internal Medicine, Neurology, Podiatry, Sports Medicine

LOCATION:

Address: 1871 W Orange Grove Rd #135, Tucson, AZ 85704Phone: (520) 382-3050Website: http://www.nwalliedphysicians.com

REVIEWS:

I had such a great experience here. I was able to get an appointment quickly and the ladies at the front desk were so pleasant and helpful. I saw Dr. Ritiker and she was so sweet and really listened which is hard to find these days. I highly recommend this place and will be telling all of my family and friends. Sarah H.

Southwest Shoulder Elbow & Hand Center PCtreats various types of upper extremity ailments, including traumatic, arthritic, pediatric, congenital, sports-related, and repetitive injuries. They endeavor to serve all patients with comprehensive and sensitive care. They are available for emergency and urgent treatment as a regional care center, and theyare easily accessible to referral physicians and practitioners.

Southwest Shoulder Elbow & Hand Center, P.C. is a practice that specializes in shoulder, elbow, and hand injuries. They are an inclusive clinic that treats adults and children with a variety of arm and hand disorders. They primarily serve Tucson and southern Arizona, but theyalso see patients from all across the country and throughout the world.

They are a group of six highly skilled Orthopedic Surgeons with the primary purpose of providing thoughtful, caring, and outstanding medical care. They offer advice on both operative and non-operative treatments, and then implement those recommendations in collaboration with a team of medical experts.

Link:
5 Best Orthopediatrician in Tucson, AZ - Kev's Best

Conference Call and Webcast Scheduled for 9:15 a.m. Eastern Time

BRISBANE, Calif., November 04, 2021--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported third quarter financial results and provided business and clinical highlights.

"We are delighted to share clinical data and business updates across several programs demonstrating that Sangamo has three important assets progressing toward late-stage development. Our gene therapy portfolio is advancing with accumulating safety and efficacy data in our Fabry and hemophilia A programs, and preliminary proof-of-concept data demonstrate the clinical potential of our zinc finger genome engineering technology in sickle cell disease. These data readouts show the progression of our first-generation genomic medicine pipeline and potentially pave the way for new treatments. Our next generation programs focus on genome regulation and allogeneic CAR-Treg cell therapy, where we have a robust preclinical pipeline in neurological and autoimmune diseases. We are energized by this momentum and look forward to continued execution of our corporate strategy," said Sandy Macrae, Chief Executive Officer of Sangamo.

Recent Clinical and Business Highlights

Fabry Disease First four patients dosed exhibited above normal -Gal A activity; Phase 3 planning initiated

Earlier today, we announced preliminary clinical data from the first four patients treated in our Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, our wholly owned Fabry disease gene therapy product candidate. Data as of the September 17, 2021 cutoff date from the four patients in the first two dose cohorts showed that isaralgagene civaparvovec was generally well tolerated. All four patients exhibited above normal alpha-galactosidase A (-Gal A) activity, which was maintained for up to one year for the first patient treated and through 14 weeks for the most recently treated patient. Activity of 2-fold to 15-fold above mean normal was observed at last measurement as of the cutoff date. Withdrawal from enzyme replacement therapy (ERT) has taken place for one patient and is planned for the other patient on ERT, based on the stability of their -Gal A activity following treatment.

The fifth patient in the STAAR study, who is the first patient in the third cohort (3e13vg/kg), was dosed after the cutoff date. The sixth patient is currently in screening also for the third dose cohort. We expect to provide updated data throughout 2022 and present these results at a medical meeting.

Based on the STAAR study results to date, we have initiated planning for a Phase 3 Fabry disease clinical trial.

Sickle Cell Disease Preliminary-proof-of-concept data will be presented at ASH as clinical program advances

Story continues

Preliminary proof-of-concept results from the Phase 1/2 PRECIZN-1 study investigating SAR445136, formerly BIVV003, an investigational zinc finger nuclease gene edited cell therapy, in patients with severe sickle cell disease (SCD) will be presented at the 63rd Annual Meeting of the American Society of Hematology (ASH) on December 12, 2021. Results as of the June 25, 2021 cutoff date show that all four treated patients did not require blood transfusions post engraftment and had no adverse or serious adverse events related to SAR445136 through 65 weeks of follow-up for the longest treated patient. The four treated patients all experienced increases in total hemoglobin, fetal hemoglobin and percent F cells.

We and Sanofi continue to advance the sickle cell disease program. We recently obtained manufacturing requirements guidance from FDA in preparation for further potential clinical studies. Separately, we and Sanofi made the business decision to cease development of the beta thalassemia indication in order to focus resources on the sickle cell disease program. ST-400 for beta thalassemia was developed with the support of a grant from the California Institute for Regenerative Medicine (CIRM).

Hemophilia A Four patients at highest dose experienced mean FVIII activity of 30.9% at week 104

Updated follow-up results from the Phase 1/2 Alta study of giroctocogene fitelparvovec gene therapy in patients with severe hemophilia A will be presented at ASH on December 12, 2021. For the four patients in the highest dose 3e13vg/kg cohort who have reached 104 weeks of follow-up as of the May 19, 2021 cutoff date, mean Factor VIII (FVIII) activity was 30.9% at week 104 as measured by chromogenic assay. In this cohort, the annualized bleeding rate was zero for the first year after treatment and 0.9 throughout total duration of follow-up. Giroctocogene fitelparvovec was generally well tolerated.

We and Pfizer also announced that some of the patients treated in the Phase 3 AFFINE trial of giroctocogene fitelparvovec experienced FVIII activity greater than 150% following treatment. None of these patients have experienced thrombotic events and some have been treated with direct oral anticoagulants to reduce thrombotic risk. Pfizer voluntarily paused screening and dosing of additional patients in the trial to implement a protocol amendment intending to provide guidance regarding the management of patients with FVIII levels that exceed 150%. On November 3, 2021, Pfizer was informed that the FDA has put this trial on clinical hold. The next step is to share the proposed protocol amendment with health authorities and respond to the clinical hold, after which the Companies will be able to provide updated timing for the trial.

Renal Transplant First patient enrolled, expect two patients to be dosed by mid-2022

The first patient has been enrolled in our Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous HLA-A2 CAR Treg cell therapy product candidate treating patients receiving an HLA-A2 mismatched kidney from a living donor. We expect the first two patients in this study to be dosed by the middle of 2022 following kidney transplantation. We continue to open study sites and screen patients.

Research, Manufacturing, and Corporate Updates

Biogen announced type 1 myotonic dystrophy (DM1) as the previously undisclosed neuromuscular preclinical target in our collaboration.

We recently completed and brought online our in-house cell therapy manufacturing facility in our Brisbane, California headquarters and remain on track to complete our in-house cell therapy manufacturing facility in Valbonne, France by year-end.

We appointed D. Mark McClung as Chief Operating Officer, an important organizational step to support the multiple advancing wholly owned and partnered programs.

Third Quarter 2021 Financial Results

Consolidated net loss attributable to Sangamo for the third quarter ended September 30, 2021 was $47.7 million, or $0.33 per share, compared to a net loss attributable to Sangamo of $1.6 million, or $0.01 per share, for the same period in 2020.

Revenues

Revenues for the third quarter ended September 30, 2021, were $28.6 million, compared to $57.8 million for the same period in 2020, a decrease of $29.2 million.

The reduction in revenue was primarily due to a $39.3 million decrease related to our giroctocogene fitelparvovec and C9ORF72 collaboration agreements with Pfizer, resulting from the completion of our activities in 2020, and a $2.3 million decrease related to our collaboration agreement with Sanofi. These decreases were partially offset by higher revenues of $11.5 million and $1.3 million related to our collaboration agreements with Novartis and Biogen, respectively.

GAAP and Non-GAAP operating expenses

Three Months EndedSeptember 30,

Nine Months EndedSeptember 30,

(In millions)

2021

2020

2021

2020

Research and development

$

62.5

$

45.3

$

179.0

$

128.3

General and administrative

14.5

16.2

47.1

50.2

Total operating expenses

77.0

61.5

226.1

178.5

Stock-based compensation expense

(7.9

)

(6.7

)

(24.9

)

(19.1

)

Non-GAAP operating expenses

$

69.1

$

54.8

$

201.2

$

159.4

Total operating expenses on a GAAP basis for the third quarter ended September 30, 2021 were $77.0 million compared to $61.5 million for the same period in 2020. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the third quarter ended September 30, 2021 were $69.1 million compared to $54.8 million for the same period in 2020.

The increase in total operating expenses on a GAAP basis was primarily driven by our higher clinical and manufacturing supply expenses along with our increased headcount to support the advancement of our clinical trials and our ongoing collaborations.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of September 30, 2021 were $519.0 million compared to $692.0 million as of December 31, 2020.

Revised Financial Guidance for 2021

We are revising our full-year operating expense guidance initially provided on February 24, 2021 and reiterated most recently on August 5, 2021 as follows:

(in millions)

Initially Provided February 24, 2021;Reiterated May 4, 2021and August 5, 2021

Updated on November 4, 2021

Estimated GAAP Operating Expenses

$285 to $305

$300 to $310

Estimated Non-GAAP Operating Expenses

$255 to $275*

$265 to $275**

*excludes estimated stock-based compensation of $30 million

**excludes estimated stock-based compensation of $35 million

Conference Call

Sangamo will host a conference call today, November 4, 2021, at 9:15 a.m. Eastern Time, which will be open to the public. The call and live Q&A will be webcast.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 5178059. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 5178059.

About Sangamo Therapeutics

Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently dont exist. For more information about Sangamo, visit http://www.sangamo.com.

Forward-Looking Statements

This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the therapeutic and commercial potential of our product candidates, the anticipated plans and timelines of Sangamo and our collaborators for screening, enrolling and dosing patients in and conducting our ongoing and potential future clinical trials and presenting clinical data from our clinical trials, the anticipated advancement of our product candidates to late-stage development including potential future Phase 3 trials, anticipated implementation of a protocol amendment for the Phase 3 AFFINE clinical trial of giroctocogene fitelparvovec and the resumption of the dosing of additional patients in the trial; our revised 2021 financial guidance related to GAAP and non-GAAP total operating expenses and stock-based compensation; our continued execution of our corporate strategy; the anticipated completion of our in-house cell therapy manufacturing facility in Valbonne, France; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the effects of the evolving COVID-19 pandemic and the impacts of the pandemic on the global business environment, healthcare systems and business and operations of Sangamo and our collaborators, including the initiation and operation of clinical trials; the research and development process, including the enrollment, operation and results of clinical trials and the presentation of clinical data; the uncertain timing and unpredictable nature of clinical trials and clinical trial results, including the risk that any protocol amendment for the Phase 3 AFFINE trial of giroctocogene fitelparvovec may not be accepted by the relevant review bodies in a timely manner, or at all, or that the FDA may not lift its clinical hold on the Phase 3 AFFINE trial in a timely manner, or at all, each of which could further delay or preclude further patient dosing in the trial as well as the risks that therapeutic effects observed in clinical trial results will not be durable in patients and that final clinical trial data will not validate the safety and efficacy of our product candidates; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results; our limited experience manufacturing biopharmaceutical products, including the risks that we may be unable to maintain compliant manufacturing facilities, build additional facilities and manufacture our product candidates as intended; and our ability to achieve expected future financial performance.

There can be no assurance that we and our collaborators will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and our collaborators. These risks and uncertainties are described more fully in our Securities and Exchange Commission filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2020 as supplemented by our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021. Forward-looking statements contained in this announcement are made as of this date, and we undertake no duty to update such information except as required under applicable law.

Non-GAAP Financial Measure

To supplement our financial results and guidance presented in accordance with GAAP, we present non-GAAP total operating expenses, which exclude stock-based compensation expense from GAAP total operating expenses. We believe that this non-GAAP financial measure, when considered together with our financial information prepared in accordance with GAAP, can enhance investors and analysts ability to meaningfully compare our results from period to period and to our forward-looking guidance, and to identify operating trends in our business. We have excluded stock-based compensation expense because it is a non-cash expense that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. This non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business.

SELECTED CONSOLIDATED FINANCIAL DATA

(unaudited; in thousands, except per share data)

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Sangamo Therapeutics Reports Recent Business and Clinical Highlights and Third Quarter 2021 Financial Results - Yahoo Finance

Third Quarter Net Sales of $63.1 Million Versus $64.3 Million in 3Q20

Adjusted Net Sales of $62.8 Million Include a 13% Increase in Core Portfolio Sales Versus 3Q20

Year-to-Date Adjusted Net Sales Increase of 16% in Core Portfolio Sales Versus Prior Year PeriodReflects Expansion in Surgical Applications

Company to Host Conference Call on November 3, 2021, at 8:30 AM ET

MARIETTA, Ga., Nov. 02, 2021 (GLOBE NEWSWIRE) -- MiMedx Group, Inc. (Nasdaq: MDXG) (MIMEDX or the Company), an industry leader in utilizing amniotic tissue as a platform for regenerative medicine, today announced the filing of its third quarter 2021 Form 10-Q for the period ended September30, 2021.

Timothy R. Wright, MIMEDX Chief Executive Officer, commented, The growth in our core portfolio of tissue and cord products this quarter demonstrates consistent execution on multiple initiatives designed to reinforce and convey the differentiation, clinical evidence and economic value of our brands. We are expanding our presence in surgical applications with our AMNIOFIX brand and continuing to gain traction with our EPICORD Expandable product line, which enables customers to address a broader range of lower extremity wounds in a cost-effective way. The success in our base business fuels our investments in clinical operations, medical education, and product development initiatives including portfolio innovation and expansion, ultimately driving better outcomes for patients in need of advanced, evidence-based treatment options.

Mr. Wright continued, Since announcing the top-line data from our late-stage musculoskeletal clinical trials in mid-September, we have been extensively reviewing the study results to determine the reasons behind the observed differences between patient cohorts in the Knee Osteoarthritis (KOA) trial. As part of our statistical analysis plan, third-party biostatisticians have validated the probability values (p-values) from the Phase 2B KOA study and substantiated both a statistically significant and clinically meaningful outcome in Western Ontario and McMaster UniversitiesOsteoarthritis Index (WOMAC)Total, WOMAC Pain, andWOMAC Function scores for thePre-Interim Analysis Cohort of 190 patients, with p-values less than 0.05 at three months and less than 0.01 at six months. These positive efficacy signals, while limited to thePre-Interim Analysis Cohort of 190 patients, provide us with important insights into our approach for our future Phase 3 studies, and we are confident in the therapeutic potential of micronized dehydrated Human Amnion Chorion Membrane (mdHACM). Our recent scientific publications continue to broaden our understanding of the products mechanism of action, disease modification potential, and long-term utility as a platform for regenerative medicine. We look forward to reviewing these dataand reasons for our continued optimism at our upcoming Investor Day on December 7, 2021, when we also plan to highlight our strategy for achieving sustainable growth across our immediate focus areas of acute and chronic wound care, surgical recovery, and international expansion.

Third Quarter 2021 and Recent Operating Highlights:

Key Third Quarter 2021 Financial Metrics

MIMEDX reported net sales for the three months ended September 30, 2021, of $63.1 million, compared to $64.3 million for the three months ended September 30, 2020. Net sales for the three months ended September 30, 2021, includes revenue recognized on the Remaining Contracts (as defined below) of $0.3 million, compared to $1.0 million for the three months ended September 30, 2020.

Adjusted net sales for the three months ended September 30, 2021, which excludes cash collected on the remaining contracts outstanding at the time of the change in the Company's revenue recognition methodology, were $62.8million compared to $63.3million for the three months ended September 30, 2020, which included $8.2million of revenue related to Section 351 products. Excluding sales of Section 351 products, adjusted net sales increased $7.2 million, or 13.1% over the prior year, reflecting growth in surgical applications with the Company's AMNIOFIX sheet portfolio, and in the EPICORD Expandable product line.

MIMEDX has two primary classes of products: (1) Advanced Wound Care, or Section 361, products, consisting of its tissue and cord sheet allograft products, and (2) Section 351 products, consisting of the Companys micronized and particulate products, which prior to the end ofthe Food and Drug Administration's (FDA) period of Enforcement Discretion(as defined below) were used to treat a variety of patient needs, including both advanced wound care and musculoskeletal applications. Advanced Wound Care is further disaggregated between the Companys Tissue/Other and Cord products. A summary of the Company's revenue, including revenue derived from its Section 351 products, is included in the table below (amounts in thousands):

Gross profit margin for the three months ended September 30, 2021, was 83.9% compared to 84.0% for the three months ended September 30, 2020.

Selling, general and administrative expenses for the three months ended September 30, 2021,were $46.3 million, compared to $48.0 million for the three months ended September 30, 2020. The decrease in selling, general and administrative expenses during the period was driven by lower professional fees on legal and other matters. These lower fees were partially offset by increases in travel expenses over the prior year period,when the Company implemented travel restrictions in the midst of the COVID-19 pandemic.

Research and development expenses were $4.4 million for the three months ended September 30, 2021, compared to $3.4 million for the three months ended September 30, 2020. The increase reflectshigher personnel costs, driven by increases in headcount to support clinical research efforts. While the Company has increased its investments in clinical studies, it did not incur as much research and development expenses as anticipated, due to the delayed timing of clinical trials. Such costs are expected to increase as the Company plans and executes new trials, however the amount and timing of these expenses are partially dependent on whether the clinical trials merit further investment and other factors.

Investigation, restatement and related expenses for the three months ended September 30, 2021, were $3.2 million compared to $12.0 million for the three months ended September 30, 2020. During the three months ended September 30, 2020, MIMEDX incurred expenses toward the advancement of legal fees of certain former officers and directors of the Company. These expenses were not as significant during the same period in 2021. While the Company expects to continue to incur some litigation costs moving forward, a continued reduction in investigation, restatement and related expenses is anticipated, other than costs related to resolution of the securities class action matter, the amount and timing of which are highly uncertain.

Net loss for the three months ended September 30, 2021, was $2.3 million compared to a net loss of $19.4 million for the three months ended September 30, 2020.

Adjusted EBITDA for the three months ended September 30, 2021, was $6.8 million, or 10.8% of adjusted net sales, compared to $6.9 million, or 11.0% of adjusted net sales, for the three months ended September 30, 2020.

As of September30, 2021, the Company had $90.6 million of cash and cash equivalents, compared to $95.8 million as of December 31, 2020.

Outlook for 2021The Company expects that adjusted net sales for fiscal year 2021 will be between $245 million to $255 million, including $16.7 million of Section 351 products sold in the United States for the six months ended June 30, 2021, prior to the end of the period of Enforcement Discretion. Adjusted net sales for fiscal year 2020 were $240.5 million, including $31.8 million of Section 351 products.

Conference Call and WebcastMIMEDX will host a conference call and webcast to review its third quarter 2021 results on Wednesday, November 3, 2021, beginning at 8:30 a.m., Eastern Time. The call can be accessed using the following information:

Webcast: Click here

U.S. Investors: 877-407-6184International Investors: 201-389-0877Conference ID: 13723750

A replay of the webcast will be available for approximately 30 days on the Companys website at http://www.mimedx.com following the conclusion of the event.

Important Cautionary Statement This press release includes forward-looking statements. Statements regarding: (i) future sales or sales growth; (ii) the status, timing, and expected results of the Companys clinical trials and planned regulatory submissions, and our expectations regarding our ability to potentially accelerate the timing of any trial or regulatory submission and eventual BLA approvals; (iii) the timing of our disclosure of clinical trial results; (iv) the results of future scientific studies; (v) expectations regarding our ability to sell EPIFIX in other countries, (vi) the effectiveness of amniotic tissue as a therapy for any particular indication or condition, and (vii) future increases in research and development spending. Additional forward-looking statements may be identified by words such as "believe," "expect," "may," "plan," goal, outlook, "potential," "will," "preliminary," and similar expressions, and are based on management's current beliefs and expectations.

Forward-looking statements are subject to risks and uncertainties, and the Company cautions investors against placing undue reliance on such statements. Actual results may differ materially from those set forth in the forward-looking statements. Factors that could cause actual results to differ from expectations include: (i) future sales are uncertain and are affected by competition, access to customers, patient access to healthcare providers, and many other factors; (ii) the status, timing, results and expected results of the Companys clinical trials and planned regulatory submissions, and our expectations regarding our ability to potentially accelerate the timing of any trial or regulatory submission, depend on a number of factors including favorable trial results, patient access, and our ability to manufacture in accordance with Current Good Manufacturing Practices (CGMP) and appropriate chemistry and manufacturing controls; (iii) the Company may change its plans due to unforeseen circumstances, or delays in analyzing and auditing results, and may delay or alter the timeline for future trials, analyses, or public announcements; (iv) the results of scientific research are uncertain and may have little or no value; (v) our ability to sell our products in other countries depends on a number of factors including adequate levels of reimbursement, market acceptance of novel therapies, and our ability to build and manage a direct sales force or third party distribution relationship; (vi) the effectiveness of amniotic tissue as a therapy for particular indications or conditions is the subject of further scientific and clinical studies; and (vii) we may alter the timing and amount of planned expenditures for research and development based on the results of clinical trials and other regulatory developments. The Company describes additional risks and uncertainties in the Risk Factors section of its most recent annual report and quarterly reports filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and the Company assumes no obligation to update any forward-looking statement.

About MIMEDXMIMEDX is an industry leader in utilizing amniotic tissue as a platform for regenerative medicine, developing and distributing placental tissue allografts with patent-protected, proprietary processes for multiple sectors of healthcare. As a pioneer in placental biologics, we have both a base business, focused on addressing the needs of patients with acute and chronic non-healing wounds, and a promising late-stage pipeline targeted at decreasing pain and improving function for patients with degenerative musculoskeletal conditions. We derive our products from human placental tissues and process these tissues using our proprietary methods, including the PURION process. We employ Current Good Tissue Practices, Current Good Manufacturing Practices, and terminal sterilization to produce our allografts. MIMEDX has supplied over two million allografts, through both direct and consignment shipments. For additional information, please visit http://www.mimedx.com.

Contacts:

Investors:Jack HowarthInvestor Relations404.360.5681jhowarth@mimedx.com

Media:Hilary Dixon Corporate & Strategic Communications 770.651.9307 hdixon@mimedx.com

Selected Unaudited Financial Information

Reconciliation of GAAP Net Sales to Adjusted Net Sales and Reconciliation of GAAP Net Income to EBITDA and Adjusted EBITDA

In addition to our GAAP results, we provide certain non-GAAP metrics including Adjusted Net Sales, Earnings Before Interest, Taxes, Depreciation and Amortization (EBITDA) and Adjusted EBITDA. We believe that the presentation of these measures provides important supplemental information to management and investors regarding our performance. These measurements are not a substitute for GAAP measurements. Company management uses these Non-GAAP measurements as aids in monitoring our ongoing financial performance from quarter-to-quarter and year-to-year on a regular basis and for benchmarking against comparable companies. Adjusted Net Sales is intended to allow one to understand the trend, if any, in sales and to facilitate comparison of sales amounts in periods that used different revenue recognition methods. EBITDA is intended to provide a measure of the Companys operating performance as it eliminates the effects of financing and capital expenditures. EBITDA consists of GAAP net loss excluding: (i) depreciation, (ii) amortization of intangibles, (iii) interest expense, net, (iv) loss on extinguishment of debt, and (v) income tax provision. Adjusted EBITDA is intended to provide a normalized view of EBITDA and our broader business operations that we expect to experience on an ongoing basis by removing certain non-cash items and items that may be irregular, one-time, or non-recurring from EBITDA; most significantly those expenses related to the investigation conducted by the Audit Committee (the "Audit Committee") of the Company's Board of Directors (the "Board") into prior-period matters relating to allegations regarding certain sales and distribution practices at the Company and certain other matters (the "Investigation" or the "Audit Committee Investigation"), the restatement of our consolidated financial statements previously filed in our Annual Report on Form 10-K for the year ended December 31, 2016, as well as selected unaudited condensed consolidated financial data as of and for the years ended December 31, 2015 (Restated) and 2014 (Restated), which reflected adjustments to our previously filed consolidated financial statements as of and for the years ended December 31, 2015 and 2014 (collectively, the "Restatement"), and related litigation. This also includes share-based compensation, which is predominantly settled in shares. This enables us to identify underlying trends in our business that could otherwise be masked by such items. Adjusted EBITDA consists of GAAP net loss excluding: (i) depreciation, (ii) amortization of intangibles, (iii) interest expense, (iv) loss on extinguishment of debt, (v) income tax provision, (vi) costs incurred in connection with Audit Committee Investigation and Restatement, (vii) the effect of the change in revenue recognition on net loss, and (viii) share-based compensation.

A reconciliation of GAAP net sales to Adjusted Net Sales appears in the table below (in thousands):

A reconciliation of GAAP net loss to EBITDA and Adjusted EBITDA appears in the table below (in thousands):

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MIMEDX Announces Third Quarter 2021 Operating and Financial - GlobeNewswire

NASHVILLE, Tenn., Nov. 4, 2021 /PRNewswire/ --Cryoport, Inc. (NASDAQ: CYRX) ("Cryoport" or the "Company"),a global leader in temperature-controlled supply chain solutions for the life sciences industry,today announced financial results for the three- and nine-month periods ended September 30,2021.

Jerrell Shelton, CEO of Cryoport, commented, "We delivered an outstanding third quarter and nine months of the year for the Company with strength across the board in all areas of our business. During the third quarter, our total revenue grew to a record $56.7 million driven by 38% organic growth year-over-year from Cryoport Systems and CRYOGENE and continuing strong revenue performance by MVE Biological Solutions and CRYOPDP. Our robust performance was driven by superlative execution by our global teams across all our business units. Our markets are strong and growing. Demand for MVE Biological Solutions' products remained at record highs, Cryoport Systems added 38 new customers during the quarter, and we successfully expanded the footprints for both CRYOPDP and CRYOGENE.

"Our Biopharma/Pharma revenue increased 371% year over year in the third quarter of 2021 or 41%, organically. But the story does not end there, we now support a record 582 clinical trials, compared with 561 at the end of the second quarter of 2021 and 517 at the end of the third quarter of 2020. We also support eight commercial therapies in regenerative medicine, including Novartis' KYMRIAH, Gilead/Kite's YESCARTAand TECARTUS, bluebird bio's ZYNTEGLO andSKYSONA, Bristol Myers Squibb's BREYANZI and ABECMA and Orchard Therapeutics' LIBMELDY. Additionally, four of the approved therapies received extended or supplemental approvals in the third quarter.

"Our revenue by market for the three- and nine-months ended September 30, 2021, as compared to the same periods in 2020 was asfollows:

Cryoport, Inc. and Subsidiaries

Total revenues by market

(unaudited)

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

2021

2020

% Change

2021

2020

% Change

Biopharma/Pharma

$ 46,001

$ 9,760

371%

$ 133,878

$ 27,120

394%

Animal Health

8,261

223

3598%

25,655

664

3762%

Reproductive Medicine

2,431

1,189

105%

6,635

2,551

160%

Total revenues

$ 56,693

$ 11,172

407%

$ 166,168

$ 30,335

448%

"Our solutions are experiencing accelerating global demand as a record number of cell and gene therapies are slated for commercialization in the coming months and years."

Mr. Shelton concluded, "We continue to set the pace and the standard for supply chain solutions for the regenerative medicine industry which continues to be in its very early stages of development. To support our continued global growth, we have expanded into 33 facilities in 16 countries and have initiated further expansion within the fast-growing Asia-Pacific (APAC) and EMEA (Europe, Middle East, and Africa) regions.We believe our strong momentum will continue to build through the remainder of the year and beyond as we realize the large commercial revenue potential of our vast pipeline of clinical trials supported. Our performance is a testament to the power of our strategy and our team's commitment to Cryoport and its mission, and, with that, we expect significant worldwide opportunities ahead to continue building sustainable, long-term value for shareholders."

Biopharma/Pharma

Our total Biopharma/Pharma revenue increased by $36.2 million, or 371%, to $46.0 million for the third quarter of 2021 compared to $9.8 million for the third quarter of 2020, driven by strong revenue contributions from all business units. For the third quarter of 2021, Biopharma/Pharma revenue grew organically by $4.0 million, or 41%, to $13.8 million compared to third quarter in the prior year.

As of the end of the third quarter, we supportedanettotalof582 clinical trials, compared with 561 at the end of the second quarter 2021 and 517 in third quarter 2020. The number of trials by phase and region are as follows:

Cryoport Supported Clinical Trials by Phase

Clinical Trials

September 30,

2021

2020

2019

Phase 1

240

207

180

Phase 2

272

244

191

Phase 3

70

66

54

Total

582

517

425

Cryoport Supported Clinical Trials by Region

Clinical Trials

September 30,

2021

2020

2019

Americas

459

411

360

EMEA

92

83

55

APAC

31

23

10

Total

582

517

425

A total of nine (9) Cryoport supported Biologic License Applications (BLAs) or Marketing Authorization Applications (MAAs) were filed in the nine months ended September 30, 2021, based on internal information and forecasts from the Alliance for Regenerative Medicine, of which three (3) were filed during the third quarter of 2021. Looking forward, we anticipate up to another four (4) BLA and MAA submissions for Cryoport-supported products during the remainder of 2021 and, at this time, an additional twenty-one (21) filings in 2022. Additionally, a total of four (4) Cryoport supported therapies received extended or supplemental approvals in the third quarter.

Animal Health

Our revenue from the Animal Health market increased by $8.0 million, or 3,598%, to $8.3 million for thethird quarter ended September 30, 2021,ascomparedtothesameperiodin2020 andwas primarily driven byouracquisitionofMVE Biological Solutions,whichhasastrongandlongstanding presenceinthismarket. Third quarter revenue grew organically by 31% over the prior year demonstrating successful execution of our engagement strategy within the animal health space.

Reproductive Medicine

Reproductive Medicine revenue more than doubled to $2.4 million for the third quarter of 2021 compared to $1.2 million for the third quarter of 2020, an increase of $1.2 million, or 105%. We see continuing strong demand for our CryoStork solutionprovided by Cryoport Systems driven by fertility clinic networks that are looking for global standardization on our best-in-class solution. MVE Biological Solutions also contributed revenue to our Reproductive Medicine market through its portfolio of cryogenic shipper and freezersolutions. We plan to continue to add agreements with new fertility clinics to our network globally during the remainder of 2021 and beyond to drive increased adoption of our services as well as expand our support efforts within this space to EMEA and APAC.

Financial Highlights

Note: All reconciliations of GAAP to adjusted (non-GAAP) figures above are detailed in the reconciliation tables included later in the press release.

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Cryoport Reports Record Third Quarter and Nine Months Revenue for 2021 - PRNewswire

Human interneuron progenitors derived from induced pluripotent stem cells (iPSCs) can be successfully transplanted and integrate into mouse brains, mature, and reverse signs of hippocampal network dysfunction associated with Alzheimer's disease, GABAeron scientists reported at ISSCR/JSRM.

Published: Oct. 27, 2021 at 7:00 AM CDT|Updated: 18 hours ago

SAN FRANCISCO, Oct. 27, 2021 /PRNewswire/ -- GABAeron, Inc. today presented promising preclinical data on their first-in-class, iPSC-based cell therapy product for Alzheimer's disease at the International Society for Stem Cell Research (ISSCR) and Japanese Society for Regenerative Medicine (JSRM) international meeting "Stem Cells: From Basic Science to Clinical Translation". The data, the first to be publicly shared since the company was founded in 2017, highlight the potential of transplanted, human iPSC-derived interneuron progenitors in treating Alzheimer's disease as well as other neurological disorders with interneuron deficit or loss.

GABAeron scientists successfully differentiated GABAergic interneuron progenitors from human iPSCs, and showed that when transplanted into the brains of an Alzheimer's disease mouse model carrying the major genetic risk factor apolipoprotein E4 (APOE4) the cells could mature, integrate into the hippocampus, and reverse signs of the hippocampal network dysfunction associated with Alzheimer's disease.

"We are incredibly excited by these data, which show the safety and efficacy of our novel iPSC-based approach in an Alzheimer's disease mouse model," said Robert W. Mahley, MD, PhD, chief executive officer and chief scientific officer of GABAeron. "Based on these results, we plan to continue our work to develop a cell replacement therapy to treat patients with APOE4-positive Alzheimer's disease."

Over the course of his career, Mahley president emeritus of the Gladstone Institutes and professor of pathology and medicine at the University of California, San Francisco has illuminated the importance and molecular details of the protein APOE. The gene for APOE comes in several versions and we now know that people with the APOE4 version of the gene have an increased risk of Alzheimer's disease and an earlier age of disease onset compared to people with the more common APOE3 version. Strikingly, APOE4 is associated with 6075% of all Alzheimer's disease cases.

GABAeron scientific co-founder Yadong Huang, MD, PhD, director of the Center for Translational Advancement at the Gladstone Institutes, San Francisco, discovered one important reason for the association between APOE4 and Alzheimer's disease. APOE4, his lab demonstrated, leads to the impairment and loss of hippocampal GABAergic interneurons cells critical for maintaining normal hippocampal activity, required for normal learning and memory, and damaged or lost in Alzheimer's disease brains.

"GABAeron was founded on the premise that if we can replace those interneurons via cell-based therapy, we can restore normal hippocampal activity and thus slow or reverse many of the memory and cognitive impairments associated with Alzheimer's disease," said Huang. "If this approach works, it will be a single treatment with long-lasting impact for Alzheimer'spatients."

In the new study, researchers led by Wen-Chin (Danny) Huang, PhD, and Iris Avellano, developed a novel effective method of coaxing human iPSCs carrying the APOE3 gene to differentiate into GABAergic interneuron progenitors. The resulting cells showed high viability, purity, and robust functionality with more than 90% committed to the correct developmental lineage.

A team led by Wan-Ying Hsieh, PhD, transplanted these interneuron progenitors into the hippocampus of 10-month-old mice carrying the human APOE4 gene; the interneuron progenitors showed robust survival and matured into functional GABAergic interneurons. At 7 months post-transplantation, more than half of the surviving cells had migrated out of the local area, populated the hippocampal subregions, and established connections with other existing neurons throughout the hippocampus.

"These exciting data reveal the high quality of human iPSC-derived interneuron progenitors generated at GABAeron and highlight the feasibility of their long-term survival and integration into mouse brains," said Hsieh. "Importantly, there was no tumor formation from the transplanted cells in over a hundred mice."

They next carried out electrophysiological recordings to study hippocampal network activity in the mice. As expected, the APOE4 mice had deficits in hippocampal activity that can underlie the memory impairments associated with Alzheimer's disease. Specifically, the mice had fewer sharp-wave ripples and their associated slow gamma power in the hippocampus both of which are critical for memory formation and retrieval. When each mouse was transplanted with approximately 120,000 iPSC-derived human interneuron progenitors carrying APOE3, these measurements of hippocampal activity both improved to the levels seen in healthy mice 7 months later.

"These results as a whole represent a critical step toward a potential interneuron-based therapy for APOE4-related Alzheimer's disease," said Qin Xu, PhD, senior director at GABAeron. "This builds up a solid foundation for our further work with clinical-grade human iPSCs."

GABAeron scientists are now adapting their culture techniques for the mass production of clinical-grade human iPSC-derived GABAergic interneuron progenitors. They are also working to identify the molecular characteristics of the mature GABAergic interneurons, which become successfully integrated into the hippocampus in the Alzheimer's disease mouse model.

"With this critical milestone reached, GABAeron will move forward, with great confidence, toward IND-enabling studies and future trials with clinical-grade human iPSC-derived interneuron progenitors for treating APOE4-related Alzheimer's disease," said Sheng Ding, PhD, scientific co-founder of GABAeron and a serial entrepreneur who co-founded two leading public companies, Fate Therapeutics (FATE) and Tenaya Therapeutics (TNYA). "We also plan to explore the usefulness of such a cell-based therapy for other neurological diseases with interneuron deficits or loss."

The ISSCR/JSRM international meeting "Stem Cells: From Basic Science to Clinical Translation" runs from October 2729, 2021 and is being held virtually this year.

About GABAeron

GABAeron, Inc. is a biopharmaceutical company founded in 2017, based on pioneer work initiated at the Gladstone Institutes, to build on the promise of combining precision medicine, regenerative medicine, and pharmaceutical intervention. The company is exploring a new first-in-class IND candidate to replace or restore neurons injured or lost in the brains of patients suffering from neurodegenerative and neurodevelopmental disorders.

For more information about GABAeron, please visit http://www.GABAeron.com.

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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GABAeron Presents Promising Preclinical Data on Stem Cell-Based Therapy for Alzheimer's Disease - KFYR-TV

Washington, DC, Oct. 21, 2021 (GLOBE NEWSWIRE) -- via NewMediaWire -- The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies, today announced the election of its 2022 Officers, Executive Committee, and Board of Directors.

The Executive Committee and Board of Directors oversee the formation and execution of ARMs strategic priorities and focus areas for the coming year.

ARM welcomes the diverse expertise and experience of our 2022 Board of Directors, said ARM Chief Executive Officer Janet Lambert. Our sector is poised to shape healthcare for years to come and our Board will be instrumental in advancing the delivery of transformative therapies for patients globally, while helping to eradicate barriers and legacy policy that could slow access.

ARM 2022 Officers:

Emile Nuwaysir, Ph.D. Chief Executive Officer, Ensoma (Chairman)Usman 'Oz' Azam, M.D. President and Chief Executive Officer, Tmunity Therapeutics (Vice Chairman)Amy Butler, Ph.D. President, Biosciences, Thermo Fisher Scientific (Secretary)Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies (Treasurer)

ARM 2022 Executive Committee:

Usman 'Oz' Azam, M.D. President and Chief Executive Officer, Tmunity TherapeuticsAmy Butler, Ph.D. President, Biosciences, Thermo Fisher ScientificMiguel Forte, M.D., Ph.D. Chief Executive Officer, Bone TherapeuticsClaudia Mitchell, MBA, Ph.D. Senior Vice President, Product and Portfolio Strategy, Astellas PharmaEmile Nuwaysir, Ph.D. Chief Executive Officer, EnsomaBob Smith, MBA Senior Vice President, Global Gene Therapy Business, PfizerDevyn Smith, Ph.D. Chief Executive Officer, Arbor BiotechnologiesArthur Tzianabos, Ph.D. President and Chief Executive Officer, Homology MedicinesChristopher Vann Senior Vice President and Chief Operating Officer, Autolus Therapeutics

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ARM 2022 Board of Directors* New to the Board for 2022

Usman 'Oz' Azam, M.D. President and Chief Executive Officer, Tmunity TherapeuticsMark Battaglini Senior Vice President, Global External Affairs & US Government Payer, bluebird bioAmy Butler, Ph.D. President, Biosciences, Thermo Fisher ScientificBradley Campbell, MBA President and Chief Operating Officer, Amicus TherapeuticsMiguel Forte, M.D., Ph.D. Chief Executive Officer, Bone TherapeuticsBobby Gaspar, M.D., Ph.D. Chief Executive Officer, Orchard TherapeuticsJerry Keybl, Ph.D. Senior Director, Cell & Gene Therapy, MilliporeSigmaBrett Kopelan Executive Director, Debra of AmericaDave Lennon, Ph.D. Former President, Novartis Gene TherapiesBruce Levine, Ph.D. Founding Director, Clinical Cell and Vaccine Production Facility, Abramson Cancer Center, University of Pennsylvania* Timothy Lu, M.D., Ph.D. Chief Executive Officer, Senti BiosciencesJohn Maslowski, M.S. Chief Commercial Officer, Forge Biologics* Chris Mason, M.D., Ph.D. Chief Scientific Officer, AVROBIO* Debra Miller Chief Executive Officer & Founder, CureDuchenneClaudia Mitchell, MBA, Ph.D. Senior Vice President, Product and Portfolio Strategy, Astellas Pharma* Alison Moore, Ph.D. Chief Technology Officer, Allogene TherapeuticsAdora Ndu, PharmD, J.D. Group Vice President, Worldwide Research & Development Strategy, Scientific Collaborations and Policy, BioMarinSusan Nichols President & Chief Executive Officer, Propel BioSciencesEmile Nuwaysir, Ph.D. Chief Executive Officer, EnsomaKarah Parschauer, J.D. Executive Vice President, General Counsel and Corporate Secretary, UltragenyxLouise Rodino-Klapac, Ph.D. Executive Vice President, Chief Scientific Officer, Sarepta TherapeuticsJeff Ross, Ph.D. Chief Executive Officer, Miromatrix Medical* Laura Sepp-Lorenzino, Ph.D. Executive Vice President, Chief Scientific Officer, Intellia TherapeuticsR.A. Session, MBA President, Founder & Chief Executive Officer, Taysha TherapeuticsCurran Simpson, M.S. Senior Vice President, Product Development and CTO, REGENXBIOSanjaya Singh, Ph.D. Vice President & Global Head, Janssen Pharmaceutical Companies of Johnson & Johnson, Janssen Research & DevelopmentBob Smith, MBA Senior Vice President, Global Gene Therapy, PfizerDevyn Smith, Ph.D. Chief Executive Officer, Arbor BiotechnologiesJoseph Tarnowski, Ph.D. Senior Vice President of Cell and Gene Therapy Platforms, Medicinal Science & Technology, R&DArthur Tzianabos, Ph.D. President and Chief Executive Officer, Homology MedicinesChristopher Vann Senior Vice President and Chief Operating Officer, Autolus TherapeuticsKristin Yarema, Ph.D. Chief Commercial Officer, Atara Biotherapeutics

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 400+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Kaitlyn Dupont8037278346kdupont@alliancerm.org

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The Alliance for Regenerative Medicine Announces Election of 2022 Officers, Executive Committee, and Board of Directors - Yahoo Finance

Leading regenerative medicine company to participate in initiatives to further educate and improve patient care.

Published: Oct. 27, 2021 at 12:00 PM EDT|Updated: 14 hours ago

MIAMI, Oct. 27, 2021 /PRNewswire/ --Vivex Biologics, Inc., a leading regenerative medicine company specializing in the development of naturally sourced treatment options, will be attending the Symposium on Advanced Wound Care (SAWC) in Las Vegas on October 29-31, 2021.

SAWC brings together wound care teams, including physicians, nurses, physical therapists, researchers, scientists, podiatrists and dietitians, to improve the overall outcome of patients through furthering wound care education. The event will be comprised of various presentations, case studies, clinical research and practice innovations on topics including wound healing and wound care issues.

VIVEX professionals will host booth #440 during the forum, showcasing VIVEX's portfolio of wound care products and solutions. The inherent properties of VIVEX's amniotic allograft products provide mechanical protection and act as a barrier for external wounds, such as diabetic foot ulcers, venous leg ulcers, pressure ulcers and burns while VIVEX's IntegrityProcessing retains the nutrient-rich growth factors essential for signaling.

In-person and virtual SAWC attendees at the Poster Grand Rounds on Oct. 30 at 5:20 p.m. will be able to review the "New Amniotic Allograft Used To Treat Clinically Challenging Chronic Wounds"poster, number CS-087, highlighting the real world experience of Sarah Hull, FNP-C, WCC, DWC with CYGNUS products. The poster demonstrates a series of clinically challenging chronic wounds, treatment methods and resulting patient outcomes.

"We are thrilled to attend SAWC for our first time and hear from the best and brightest industry experts in wound care," said Gail Farnan, VP and General Manager of VIVEX's Wound Care & Amnion Therapies business unit. "VIVEX is focused on creating the best products and solutions for patients and collaborating with wound care professionals to educate and find innovative ways to further our shared initiatives."

For more information on VIVEX and itsadvanced regenerative medicine solutions, please visit VIVEX.com.

About Vivex Biologics, Inc.

Vivex Biologics is a pioneer in regenerative medicine, specializing in the development of naturally sourced treatment options that improve clinical, surgical, and therapeutic patient care through innovation. With tissue damage resulting from a variety of diseases, direct injury or trauma, there is a significant need for advanced solutions. By leveraging the resources of the nation's oldest civilian tissue bank, VIVEX is channeling the body's inherent healing qualities to bring patients optimal care and to provide medical professionals and patients with innovative treatment options for a broad range of indications.

Media Contact: rbb CommunicationsRachel Gerardirachel.gerardi@rbbcommunications.com

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SOURCE Vivex Biologics, Inc.

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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Vivex Biologics, Inc. to Attend the Symposium of Advanced Wound Care - UpperMichigansSource.com

The COVID-19 pandemic has created a sense of urgency to generate new drugs and vaccines. In many cases, this urgency became a regulatory opportunity to bypass established regulatory pathways for new drugs.

While this has led to the fast emergence of many useful drugs and vaccines for COVID-19, it has also led to a general reduction in the quality of medical research from which to derive conclusions.

For example, according to Janet Woodcock, former director of the Food and Drug Administrations (FDA) Center for Drug Evaluation and Research, an FDA analysis found that 6% of clinical trials are yielding results the agency deems actionable.

The lack of regulation coupled with a sense of urgency has also led to overhype and rushed development of certain treatments, including cell-based therapies often sold as stem cell treatments.

While some of these products have undergone well-designed, adequately controlled trials, most are in the early stages. Some clinics are nevertheless offering these unproven and unlicensed treatments to people, promising to boost their immune system or overall health to protect against COVID-19.

Promoting and selling unproven and unlicensed treatments can harm public health and could lead many to undergo untested and potentially harmful treatments.

Recently, a group of researchers from the University of California, Irvine, the Georgia Institute of Technology, the University at Buffalo, NY, and the University of Melbourne in Australia, published a report outlining misinformation around cell-based treatments for COVID-19, calling for their stronger regulation.

Efforts to rapidly develop therapeutic interventions should never occur at the expense of the ethical and scientific standards that are at the heart of responsible clinical research and innovation, said Dr. Laertis Ikonomou, assistant professor of Oral Biology at the University at Buffalo, and co-author of the study.

Scientists, regulators, and policymakers must guard against the proliferation of poorly designed, underpowered, and duplicative studies that are launched with undue haste because of the pandemic, but are unlikely to provide convincing, clinically meaningful safety and efficacy data, said co-author Dr. Leigh Turner, professor of Health, Society and Behavior at the University of California, Irvine.

The researchers published their report in Stem Cell Reports.

Researchers conducted a study in August 2020 of 70 clinical trials involving cell-based treatments for COVID-19. They found that most were small, with an average of 51.8 participants, and only 22.8% were randomized, double-blinded, and controlled experiments.

The authors concluded that the cell-based interventions for COVID-19 were likely to have a relatively small collective clinical impact.

Cell-based treatments for COVID-19 are still at an experimental stage, Dr. Ikonomou told Medical News Today. There are tens of clinical trials, of varied complexity and rigor, that evaluate various cell types, such as mesenchymal stromal cells, for COVID-19 treatment.

Expanded or compassionate use of cell-based interventions has also been reported, but these individual cases are unlikely to tell us whether and how cell therapies could help with COVID-19 and do not substitute for the systematic clinical evaluation of cell-based products, he added.

A few completed phase 1/2 trials have shown a favorable safety profile, but larger size trials are required. Eventually, properly-powered, controlled, randomized, double-blinded clinical trials will help determine whether cell-based treatments are a viable therapeutic option for COVID-19 and its complications, he explained.

The urgency of the pandemic has made it easy to exaggerate early-stage research. The scientists highlight this is especially the case in press releases, where media professionals can over-hype findings and understate or omit limitations to gain more media coverage.

The researchers also say that even when online media include limitations and key aspects of studies, other communication channels can strip these away easily. What is left then gets amplified, as the public is desperate to see positive news.

To address this, the researchers say science communicators should ensure they have an accurate understanding of the information they report and highlight the required steps for the science to advance without exaggerating its speed.

The researchers also say that simply feeding the public more information in what is known as the information deficit model alone is insufficient. They also suggest science communicators should strive for an engaged or dialogue-based communication approach.

Over-hyping of promising treatments and in particular cell-based treatments has been a longstanding problem, and it did not first emerge with the COVID-19 pandemic, said Dr. Ikonomou. It has become a salient issue during these times due to the global nature of this health emergency and the resulting devastation and health toll.

Therefore, it is even more important to communicate promising developments in COVID-19-related science and clinical management [responsibly]. Key features of good communication are an accurate understanding of new findings, including study limitations and avoidance of sensationalist language, he explained.

Realistic timeframes for clinical translation are equally important as is the realization that promising interventions at preliminary stages may not always translate to proven treatments following rigorous testing, he added.

The researchers say that commercial investments by biotechnology companies to develop cell-based therapies for COVID-19 have led to well-designed and rigorous clinical trials.

However, some other businesses have overlooked the demanding process of pre-marketing authorization of their products. Instead, they made unsubstantiated and inaccurate claims about their stem cell products for COVID-19 based on hyperbolic reporting of cell-based therapies in early testing.

Some clinics advertise unproven and unlicensed mesenchymal stem cell treatments or exosome therapies as immune boosters that prevent COVID-19 and repair and regenerate lungs.

Often, these businesses make their treatments available via infusion or injection. However, one anti-aging clinic in California shipped its kits to clients, where they were to self-administer with a nebulizer and mask.

Such companies often market stem cell treatments via online and social media. In an initial review of many of these brands, the researchers could not find published findings from preclinical studies and clinical trials to support their commercial activities.

Instead, they found that these companies drew from uncritical news media reports, preliminary clinical studies, or case reports in which those diagnosed with COVID-19 received stem cell interventions.

Promoting such therapies that have not undergone proper tests for safety and efficacy have the potential for significant physical and financial harm.

Health experts have documented adverse events due to unlicensed stem cell products, including vision loss and autoimmune, infectious, neurological, and cardiovascular complications.

Early in the pandemic, scientific and professional societies, including the Alliance for Regenerative Medicine and the International Society for Stem Cell Research, have warned the public against businesses engaged in the marketing of cell-based treatments that have not undergone adequate testing.

The researchers highlight that it is unclear whether these warnings reached individuals and their loved ones or significantly affected public understanding of the risks of receiving unlicensed and unproven stem cell treatments for COVID-19.

They also indicate that it is unclear whether these societies and organizations have an important role in convincing regulatory bodies to increase enforcement in this space. Nevertheless, at the time of writing, the FDA and Federal Trade Commission have issued 22 letters to businesses selling unproven and unlicensed cell-based therapies.

And while many of these companies have ceased market activity, the presence of other companies continuing to pedal the same claims makes it clear that regulatory bodies must increase their enforcement.

Additionally, the researchers question whether warning letters are sufficient to disincentivize clinicians and others to sell unlicensed products. They write:

If companies and affiliated clinicians are not fined, forced to return to patients whatever profits they have made, confronted with criminal charges, subject to revocation of medical licensure, or otherwise subject to serious legal and financial consequences, it is possible that more businesses will be drawn to this space because of the profits that can be generated from selling unlicensed and unproven cell-based products in the midst of a pandemic.

The researchers conclude that regulators should increase enforcement against unproven and unlicensed therapies for COVID-19.

They also say that science communicators should report on scientific claims more realistically and include the public in more discourse.

In the U.S. and elsewhere, there are regulations and enforcement mechanisms that deal with harms caused by unproven and unlicensed cell-based interventions and false advertising claims, said Dr. Ikonomou. It may be preferable to implement existing regulations more vigorously than introduce new ones.

Stakeholders, such as scientific, professional, and medical associations, can contribute towards this goal with reporting and monitoring of cell therapy misinformation. There is a shared responsibility to combat cell-therapy related misinformation and disinformation that undercuts legitimate research and clinical efforts and portrays unproven interventions as silver bullets for COVID-19, he concluded.

For live updates on the latest developments regarding the novel coronavirus and COVID-19, click here.

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COVID-19: Researchers warn against overhyping early-stage therapies - Medical News Today