Category Archives: Stem Cell Therapy

Acute Myeloid Leukemia

Historical complete response (CR)/CR with incomplete hematologic recovery rates with approved IDH inhibitors, enasidenib (Idhifa) and ivosidenib (Tibsovo), hover around 30% and have a median overall survival (OS) of approximately 9 months in the relapsed/refractory setting, said Shammo, a professor of medicine and pathology at Rush University Medical Center, in a virtual presentation during the2021 ASCO Direct HighlightsTMwebcast in Chicago, a program developed by Physicians Education Resource LLC.

Olutasidenib, a highly potent, orally active, selective IDH1 inhibitor, was developed to improve outcomes in the 7% to 14% of patients with AML who harbor IDH1 mutations.

In the phase 1/2 2102-HEM-101 trial (NCT02719574), 150 mg of oral olutasidenib was evaluated as a single agent and in combination with azacitidine across 8 cohorts of patients with acute myeloid leukemia (AML) and myelodysplastic syndromes. Findings from the planned interim analysis, which were presented during the 2021 ASCO Annual Meeting, focused on cohort 1, which enrolled 153 patients with relapsed/refractory, IDH1-mutant AML.1

In the efficacy-evaluable population (n = 123), 37% of patients had secondary AML, 73% of patients had intermediate cytogenetic risk, 44% of patients were refractory to their last line of therapy, and 11% had failed allogeneic stem cell transplant, reflecting a difficult population to treat, said Shammo.

The primary end point, CR/CR with partial hematologic recovery (CRh), was 33% (95% CI, 25.1%-42.4%), and most CR/CRh responders had a CR (30%; 95% CI, 22.1%-39.0%).

Transfusion independence was achieved in all response groups, particularly those achieving a CR, said Shammo.

The agent also demonstrated durable CR/CRh benefit, with a median duration of CR/CRh that was not reached and a median duration of response (DOR) of 11.7 months. In a sensitivity analysis with transplant considered as the end of a response, the median duration of CR/CRh response was 13.8 months.

Those who responded well had a much longer DOR, and a fraction of those patients managed to go onto stem cell transplant, which is what you would like to do in relapsed/refractory AML, said Shammo.

Better response was strongly associated with longer survival as well. At a median follow-up of 9.7 months, the median OS was not reached in patients who had achieved a CR/CRh, with an estimated 18-month OS rate of 87%. In the safety population (n = 153), the median OS was 10.5 months (95% CI, 7.7-15.5). Among nonCR/CRh responders and non-responders, the median OS was 15.0 months (95% CI, 5.0not evaluable) and 4.1 months (95% CI, 3.2-5.8), respectively.

Clinical benefit, as evidenced by DOR and OS, extended to patients who responded but did not achieve CR/CRh, said Shammo.

The primary treatment-emergent adverse effects (AEs) included leukocytosis (all-grade, 25%; grade 3/4, 9%) and febrile neutropenia (all-grade, 22%; grade 3/4, 20%), said Shammo. AEs of special interest reflected reports of differentiation syndrome (all-grade, 14%; grade 3/4, 7%), QTc prolongation (all-grade, 8%; grade 3/4, <1%), and liver abnormalities (all-grade, 21%; grade 3/4, 10%).

Olutasidenib was well tolerated with a safety profile largely consistent with that of other IDH inhibitors, but patients should be monitored for differentiation syndrome and liver abnormalities, said Shammo.

These results are very promising and very impressive, and, hopefully, we will see more results not only with the single agent but also with the combination with azacitidine in other myeloid malignancies, added Shammo.

Turning to acute lymphoblastic leukemia (AML), Shammo discussed the results of the phase 1/2 ZUMA-3 trial (NCT02614066), which evaluated the CD19-directed chimeric antigen receptor (CAR) T-cell therapy brexucabtagene autoleucel in patients with relapsed/refractory ALL.2

Approximately 40% to 50% of adults with B-ALL experience relapse after initial treatment, with a 1-year OS rate of 26% after first salvage therapy, said Shammo in explaining the rationale for the study.

In the phase 1 portion of the study, brexucabtagene autoleucel demonstrated a CR/CRi rate of 83% and manageable safety profile. In the phase 2 portion, 71 patients were enrolled, and 55 patients received brexucabtagene autoleucel.

Patients could have received prior treatment with blinatumomab (Blincyto) and underwent conditioning chemotherapy with fludarabine and cyclophosphamide prior to treatment with the recommended phase 2 dose of brexucabtagene autoleucel: 1 x 106 CAR T cells/kg.

Brexucabtagene autoleucel was successfully manufactured in 92% of patients, and the median time from leukapheresis to manufacturing release was 13 days for patients in the United States, which Shammo called remarkable.

Regarding baseline demographics, 27% of patients had Philadelphia chromosomepositive disease, 100% of which had central nervous system disease at baseline, and 47% of patients had received at least 3 lines of prior therapy.

Additionally, 45% of patients received prior blinatumomab, 42% underwent prior allogeneic transplant, and the median blast count was 65.0 and 59.0 at screening and preconditioning after bridging chemotherapy, respectively.

At a median follow-up of 16.4 months, the primary end point of CR/CRi rate by central assessment, was 70.9% (CR, 56.4%; CRi, 14.5%); 31% of responders were in ongoing remission at the data cutoff.

[We saw] a high and durable response rate in heavily pretreated adults with relapsed/refractory B-ALL, most of whom had high disease burden, said Shammo.

The median time to initial CR/CRi was 1.1 months. The minimal residual disease (MRD)negativity rate was 97% in responders, and 10 patients, including 9 with CR/CRi and 1 with blast-free hypoplastic or aplastic bone marrow underwent allogeneic transplant a median of 98 days (range, 60-207) following CAR T-cell infusion.

This is exactly what you would like to do: induce patients into remission to take them to allogeneic transplant, said Shammo. Whats remarkable is that MRD negativity in patients who had this treatment approximated 100%.

Among patients with CR/CRi (n = 39), the median OS was not reached (95% CI, 16.2-NE) and the median relapse-free survival (RFS) was 14.2 months (95% CI, 11.6-NE). All-treated patients (n = 39) had a median OS and RFS of 18.2 months (95% CI, 15.9-NE) and 11.6 months (95% CI, 2.7-15.5) respectively. Non-CR/CRi responders (n = 16) had a median OS and RFS of 2.4 months (95% CI, 0.7-NE) and 0.0 months (95% CI, NE-NE), respectively.

Regarding safety, any-grade cytokine release syndrome (CRS) occurred in 89% of patients, and grade 3 or higher CRS occurred in 24% of patients, with a median onset of 5 days and manifestation of pyrexia and hypotension.

Thats relatively favorable compared with what you might expect with [CAR T-cell therapy], said Shammo.

No cases of grade 5 CRS were reported, although 1 patient had grade brain herniation related to study treatment.

Any-grade neurologic events were reported in 60% of patients, and grade 3 or greater events occurred in 25% of patients, with a median onset of 9 days and manifestation of tremor and confused state.

Tocilizumab (Actemra), steroids, and vasopressors were given to 80%, 75%, and 40% of patients, respectively.

The safety profile was manageable, and AEs were largely reversible, said Shammo. The efficacy, rapid manufacturing, and manageable safety support the promising potential of brexucabtagene autoleucel to provide long-term clinical benefit in adults with relapsed/refractory B-ALL.

On April 2, 2021, a supplemental biologics license application was submitted to the FDA for brexucabtagene autoleucel as a treatment for adult patients with relapsed/refractory B-cell precursor ALL.3

If approved, brexucabtagene autoleucel would become the first and only CAR T-cell therapy approved for adults aged at least 18 years old with relapsed/refractory ALL.

The final study Shammo highlighted was the phase 2 OPTIC trial (NCT02467270), which evaluated 3 daily starting doses of ponatinib45 mg (n = 93), 30 mg (n = 93), and 15 mg (n = 91)in patients with chronic-phase chronic myeloid leukemia (CP-CML) resistant to a prior second-generation BCR-ABL1 TKI inhibitor or with a T315I mutation.4

Patients in the 45-mg and 35-mg arms were dose reduced to 15 mg daily upon achievement of 1% or less BCR-ABL1 and across arms were dose reduced to 10 mg daily in the presence of AEs.

Ponatinib, the only pan-BCR-ABL1 inhibitor, is a third-generation TKI designed to inhibit BCR-ABL1 with or without any single resistance mutation, including T315I.

In the pivotal, phase 2 PACE trial (NCT01207440), ponatinib demonstrated deep and durable responses to 45 mg of ponatinib in patients with resistant and intolerant CP-CML. However, a high incidence of arterial occlusive events (AOEs) was reported in the study, thought to be dose dependent, which ultimately compromised the utility of the drug, said Shammo.

[OPTIC] asked what dose is needed to be sure that the patients disease is under control and that perhaps we had mitigated the AOEs, said Shammo.

Notably, more than half of patients across dose cohorts had received at least 3 prior TKIs, and approximately a quarter of patients had a T315I mutation, said Shammo.

At a median follow-up of 32 months, the percentage of patients with 1% or less BCR-ABL1 at 12 months was 44.1% (95% CI, 31.7%-57.0%), 29.0% (95% CI, 18.4%-41.6%), and 23.1% (95% CI, 13.4%-35.3%) in the 45-mg, 30-mg, and 15-mg cohorts, respectively.

In the 30-mg and 15-mg cohorts, patients with less-resistant disease and without a T315I mutation at baseline had greater benefit than those with [a] T315I [mutation], said Shammo.

The 3-year OS probabilities were very reasonable, said Shammo, at 89.29%, 88.58%, and 91.71%, respectively. Notably, robust survival outcomes were reported in patients with and without BCR-ABL1 mutations, said Shammo.

What was interesting is that if you had the T315I mutation at baseline, you do need the 45-mg dose, because the response rates [with that dose] seem to be so much better than [those] patients who received a lower dose of ponatinib, said Shammo.

In terms of safety, any-grade treatment-emergent AOEs occurred in 9.6% of patients in the 45-mg arm, 5.3% in the 30-mg arm, and 3.2% in the 15-mg arm; grade 3 or greater rates occurred in 5.3%, 5.3%, and 3.2% of patients, respectively.

At this primary analysis, novel response-based ponatinib dosing regimens had clinically manageable safety and AOE profiles, with an optimal benefit-risk profile achieved with a 45-mg starting dose reduced to 15 mg upon response, concluded Shammo.

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IDH1 Inhibitors, CAR T-Cell Therapy, and Third-Generation TKIs Refresh Relapsed/Refractory Leukemia Landscape - OncLive

Palm Coast, FL, Aug. 04, 2021 (GLOBE NEWSWIRE) -- After months of careful consideration, Gold River Productions, Inc. (OTC Pink: GRPS) (GRPS) opens its Rejuvenation Division and to welcome Michael Berkowitz as the newest member to the Gold River Team. His extensive background in the transformative field of cell-based therapy provides a significant pathway for sustainable revenue and directly compliments our new herbal formulations. With the coupling of our current product line and the considerable advances in stem cell therapeutic development, our direction brings viable attractive options to those suffering from an expansive range of degenerative conditions.

According to Fortune Business Insights, the global stem cells market is projected to grow from $11.90 billion in 2021 to $27.65 billion in 2028 at a CAGR of 12.8%. The factors contributing to the growth include increasing demand for biologics, technological advancement in pluripotential cell therapeutics, and a rising focus on the development of personalized medicines. The growing prevalence of chronic diseases worldwide is anticipated to boost demand through 2028.

As resourcing options are realized, the company intends to bring physical presence to these dynamic and unique options by offering a number of primary free-standing facilities to supply and educate our rapidly growing physician network.

Commenting as the newest member of the team, I am humbled by the support and dedication of Dr. Goulding and his associates at GRPS. Dr. Goulding shares my vision. Ive personally witnessed the effects that stem cells can have with appropriate administration and dosing in over 5000 cases, says Mr. Berkowitz, who will head the Rejuvenation Division of the GRPS subsidiary StemSpa. Ive worked with a number of universities and FDA approved manufacturers in recording and reporting data. Weve been involved in studies including M.S., joint issues, PTSD, and cancer. Im eager for the opportunity to continue and expand this with GRPS.

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Berkowitz continued, What intrigues me most about Dr. Bond and Dr. Goulding is how their products work in concert with pluripotential cells, and our InflammaplexTM formulation will serve as a foundation of support for the cells. Furthermore, almost anyone seeking relief will require adjunctive anti-inflammatory products for their pain and underlying issues. Empowering doctors with these unique adjuvants and giving their patients access to more preferable choices will be a game-changer.

Continuing, he stated, We are in negotiations to construct stand-alone facilities in strategic locations to establish StemSpa, which will serve as centers of excellence in the United States and offshore. We will begin with approximately 1000 doctors thanks to the relationship built between GRPS, heliosDx, and CEO Ashley Sweat of Rushnet (OTC Pink: RSHN). Im very excited to have doctors in this continuously expanding group utilize our inflammatory-reducing product line and have our technology at their fingertips. This will offer an amazing alternative to their patients as well as help us develop broad data collection and deliver data that these biologics are not only effective, but in the right hands and with the right techniques, possess endless possibilities to those suffering.

We are excited to have Mr. Berkowitz on board, says Richard Goulding, M.D., Chairman of the Board of GRPS. Mr. Berkowitz has been involved with over 5000 cases utilizing biologics and pluripotential cells and has a great deal of experience not only procuring appropriate cells, but in targeting specific concerns. The amount of video footage and anecdotal evidence is quite compelling. Theres a tremendous fit here, and the possibilities are exciting. Furthermore, we suspect that, with the proper base of nutrient support, we are optimizing the conditions for obtaining maximum efficacy with these techniques. We intend to be an industry leader for proper technique, procurement, and nutritional support in this complex field.

About Gold River Productions, Inc. Dedicated to changing peoples lives, Gold River is a groundbreaking company aiming at improving quality of life and longevity. Our diverse products and interests include nutraceuticals, CBD, land, education and more. With a unique staff of talented physicians, cultivators, and CBD experts, we are poised to tackle different disease states at an unprecedented level. Utilizing years of experience in herbals, rare cannabinoids, traditional and non-traditional methods of patient care, we are in the unique position to utilize CBD and herbals in the most effective ways possible. Emphasis on what already works, then augmenting effective formulas with high-quality cannabinoids in therapeutic levels, can achieve unprecedented symptom control in a myriad of disease states.

This document contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to a number of risks and uncertainties, many of which are outside Gold Rivers control. These include but are not limited to the impact of competitors products, services and pricing; product demand; market acceptance; new product development; reliance on key strategic alliances; the regulatory environment; fluctuations in operating results; and other risks which are detailed from time to time in the Companys filings with the Securities and Exchange Commission and/or OTC Markets. Gold River Productions disclaims any obligation to update or alter its forward-looking statements whether as a result of new information, subsequent events or otherwise.

CONTACT: Gold River Productions, Inc.info@grpsinc.com http://www.GRPSInc.com

About heliosDx: heliosDX is a National Clinical Reference Laboratory offering High-Complexity Urine Drug Testing (UDT), Behavioral Drug Testing, Allergy Droplet Cards, Oral Fluids, Infectious Disease (PCR), and NGS Genetic Testing. We are contracted in 44 of the lower 48 states and looking to expand our reach and capabilities. Always staying ahead of the curve, by continually investing in our infrastructure with the most efficient scientific proven instruments, and latest cutting-edge software for patient and physician satisfaction. This allows heliosDX to provide physicians fast and accurate reporting meeting and exceeding industry benchmarks. We excel in patient and client care through physician designed panels that aid in testing compliance and reporting education.

Contact: Ashley Sweatasweat@heliosdx.comwww.heliosdx.com (Under Development)@dx_helios

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Gold River Productions, Inc. (GRPS) Appoints Michael Berkowitz to spearhead the Rejuvenation Division with StemSpa - Yahoo Finance

TheGlobal Stem Cell Therapy Market To Garner Immense Returns Over 2020-2026A fundamental outline of theStem Cell Therapy Marketniche is presented by the Stem Cell Therapy Market report that entails definitions, classifications, applications together with industry chain framework. TheStem Cell Therapy Marketreport provides a far-reaching evaluation of necessary market dynamics and the latest trends. It also highlights the regional market, the prominent market players, as well as several market segments [Product, Applications, End-Users, and Major Regions], and sub-segments with a wide-ranging consideration of numerous divisions with their applications.

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Anterogen Co., Ltd., RTI SurgicalInc., Pharmicell Co., Ltd., MEDIPOST Co., Ltd., JCR Pharmaceuticals Co., Ltd., Holostem Terapie Avanzate S.r.l., NuVasiveInc., and AlloSource.

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Chapters Covered in Research Report are :

Chapter 1,2 :The goal of global Stem Cell Therapy Market covering the market introduction, product image, market summary and development scope.

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Chapter 8,9,10:Global Market Analysis by Application, Cost Analysis, Marketing Strategy Analysis, Distributors/Traders

Chapter 11,12 :Market information and study conclusions, appendix and data sources.

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Global Stem Cell Therapy Market to witness exponential proliferation during 2020-2026 The Manomet Current - The Manomet Current

Preimplantation Genetic Testing Application in Embryo HLA Typing for Stem Cell Therapy will Increase, Surpassing US$ 70 Mn by 2031

The study of the preimplantation genetic testing market offers compelling insights into key factors affecting market growth trajectory. The survey report discloses insights into preimplantation genetic testing demand outlook in terms of test type, end user, application, and technology. The report also highlights the scope of preimplantation genetic testing over the forecast period

Fact.MR A Market Research and Competitive Intelligence Provider: As per a survey conducted by Fact.MR, the global preimplantation genetic testing market reached a valuation of US$ 550 million in 2020, expanding at a CAGR of 8% between 2016 and 2020.

Owing to the increase in incidence of chronic diseases and disabilities across the globe, the market for preimplantation genetic testing is estimated to surge at a robust CAGR of 9% over the forecast period 2021 to 2031.

Preimplantation genetic testing is gaining traction on the back of rising prevalence of genetic disorders among children and disabilities among the aged population. According to a study by Genetic Alliance, in the U.K., out of every 25 children, 1 child is affected by a genetic disorder and nearly 30,000 babies are diagnosed with the disease every year. Besides this, over 500 million adults and children are living with a genetic disorder in the country.

As preimplantation genetic testing is proven as an effective solution for diagnostics of genetic disorders, it is increasingly being used by healthcare providers and institutions for identifying defects present in embryos before implantation.

Thus, the need for effective diagnostic solutions to curb the rising prevalence of genetic disorders in newborns will spur the demand for preimplantation genetic testing over the coming years.

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Embryo Human leukocyte antigen (HLA) typing for stem cell therapy is anticipated to dominate the market, accounting for over 40% of the revenue share. In response to the increasing application of preimplantation genetic testing in the procedure, the segment is projected to surpass a valuation of US$ 70 million by the end of 2031.

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Competitive Landscape

The global market for preimplantation genetic testing is highly consolidated, where a small group of leading players are accounting for nearly half of the global sales. Key player in the landscape are focusing on engaging into strategic collaboration with other players to strengthen their market footprint. For instance,

Some of the prominent preimplantation genetic testing providers profiled by Fact.MR are:

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Explore Fact.MRs Coverage on the Healthcare Domain

Genetic Testing Services Market - The future expansion of genetic testing services is heavily reliant on the advancement of information technology. Throughout the projected period, North America is expected to dominate the worldwide genetic testing services market. Increased demand for genetic testing to determine ancestry, as well as an increase in demand for in-vitro fertilization (IVF) and pre-implantation testing, are some of the primary reasons boosting market share. According to projections, the North American genetic testing market will account for over two-fifths of the total.

Rheumatoid Arthritis Stem Cell Therapy Market - Over the forecast period, the global market for rheumatoid arthritis treatments is expected to rise moderately. Over the projected period, the allogeneic mesenchymal stem cell segment is expected to lead the global rheumatoid arthritis stem cell therapy market. As the most accessible channel, hospitals are likely to account for a sizable portion of the global rheumatoid arthritis stem cell therapy market.

Serological Transplant Diagnostics Market - Serological transplant diagnosis, which is a critical component of successful and efficient organ transplantation, has grown rapidly in recent years. Growth in important distribution channels, such as hospitals and organ transplantation centres, to name a few, has boosted demand during the last five years, from 2016 to 2020, attracting more stakeholders to the business. Serological transplant diagnostic equipment sales are being fueled by an increase in incidence of serious lung, liver, and kidney disorders, as well as medical and diagnostic developments.

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Preimplantation Genetic Testing Demand to Grow by 9% CAGR Annually, through 2031 - BioSpace

TOKYO & HAIFA, Israel--(BUSINESS WIRE)--Minovia Therapeutics, Ltd. (CEO; Natalie Yivgi-Ohana, Ph.D., Minovia) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas) today announced a worldwide strategic collaboration and license agreement for the research, development, and commercialization of novel cell therapy programs for diseases caused by mitochondrial dysfunction.

Through this strategic collaboration, Astellas and Minovia aim to accelerate the creation of allogeneic mitochondrial cell therapy programs. The two companies will jointly research cell therapy program candidates comprised of cells derived from Astellas proprietary genetically-engineered, induced pluripotent stem cells and augmented with Minovias proprietary MAT platform technology. The goal of these programs will be to treat diseases caused by mitochondrial dysfunction, through the transfer of healthy mitochondria to restore the patients tissues.

Minovia is a leading company in the field of mitochondrial cell therapy that utilizes mitochondrial transfer to deliver healthy mitochondria to a patients diseased cells. Minovia has a unique technology platform called Mitochondrial Augmentation Therapy (MAT), where the patients own cells are isolated, loaded with healthy mitochondria obtained from a healthy donor, and then re-infused back into the patient. Minovia is currently conducting research, development and clinical studies with MAT in mitochondrial diseases.

Astellas is engaged through its US subsidiary companies, Astellas Institute for Regenerative Medicine (AIRM) and Universal Cells Inc., to advance allogeneic, off-the-shelf, differentiated cell therapy programs derived from pluripotent stem cells1. The new collaboration with Minovia extends Astellas capabilities in mitochondrial biology, and follow its recent acquisition of Mitobridge, Inc. and Nanna Therapeutics Limited2,3.

We are excited and honored to collaborate with Astellas, stated Minovias Co-founder and Chief Executive Officer, Natalie Yivgi-Ohana, Ph.D. We share with Astellas both their passion for mitochondrial science and their commitment to patients in need of new therapies. As Minovia continues the development of Mitochondrial Augmentation Therapy, we believe this partnership is critical to accelerate the development of off-the-shelf, allogeneic cell therapy programs for the many patients living with mitochondrial diseases caused by mitochondrial dysfunction.

Naoki Okamura, Representative Director, Corporate Executive Vice President, Chief Strategy Officer and Chief Financial Officer, at Astellas said, "We, at Astellas, have positioned mitochondrial biology as one of the Primary Focuses of our research and development strategy to develop therapies for patients with unmet medical needs. One of the aspirations of this Primary Focus is to establish a mitochondrial cell therapy platform. Minovia is pioneering mitochondrial cell therapy and has unique technologies for enhancing delivery of healthy mitochondria to the patients cells. This strategic collaboration with Minovia will accelerate and expand our pipeline of treatment options for patients with diseases with underlying mitochodrial dysfunction.

Under the terms of the agreement, Minovia receives an upfront cash payment of $20M USD. Through the joint research program with Minovia, if Astellas develops and commercializes product candidates for diseases caused by mitochondrial dysfunction, Minovia is eligible to receive up to $420M USD per product in future development, regulatory and commercial milestone payments from Astellas.

*1: R&D Meeting (December 10, 2020). Available at: https://sw4503.swcms.net/ja/ir-library/ir-meetings/inframe/main/014/teaserItems1/07/linkList/0/link/RDmeeting2020_pre_jp.pdf *2: Astellas Corporate website Accelerating the discovery and development of novel drugs that target mitochondrial functions.. Available at: https://www.astellas.com/jp/en/stories/science/mitobridge *3: Astellas Corporate website Primary Focus - Mitochondria Biology. Available at: https://www.astellas.com/jp/en/partnering/primary-focus#Mitochondria-Biology

About MinoviaMinovia Therapeutics, Ltd. is a clinical stage company and the first to use a mitochondrial cell therapy approach with the hope of bringing life-changing therapies to patients living with mitochondrial diseases, through their proprietary Mitochondrial Augmentation Therapy (MAT) platform. Minovia has an on-going autologous cell therapy program in clinical development, utilizing MAT for the future potential treatment of primary and secondary mitochondrial diseases. For more information, please visit our website at https://www.minoviatx.com.

About AstellasAstellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+ healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en.

Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on managements current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

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Astellas and Minovia Therapeutics Announce Strategic Collaboration for Novel Mitochondrial Cell Therapy Programs - Business Wire

Despite recently getting its third drug approved, Bluebird Bio (NASDAQ:BLUE) only has a $1.7 billion market cap. Will the company overcome its past pricing issues and fly to new highs? And could its lead in sickle cell disease clinical trials translate into massive gains for investors?

Bluebird Bio already had a multiple myeloma CAR T-cell therapy, Abecma, approved earlier this year, and now it has not one, but two gene therapy approvals for rare genetic diseases in the EU. On July 21, the rapidly maturing biotech received EU approval for its latest gene therapy, Skysona, for cerebral adrenoleukodystrophy (CALD). This rare pediatric neurodegenerative disease affects boys, with just over 1,000 patients having been diagnosed in the U.S., and just over 6,000 in Europe. Devastating and irreversible, it worsens over time, causing deterioration of nerve cells in the brain. Most patients die within two years of diagnosis.

Before Skysona was approved, the only option available to CALD patients was a stem cell transplant -- a treatment that is less likely to cause complications if the donor is a sibling. However, it is estimated that fewer than 20% of CALD patients have a matched sibling donor.

Image source: Getty Images.

Skysona, a one-time gene therapy, has proven very effective against CALD -- 90% of patients who underwent the treatment were free from major functional disabilities at least 24 months afterward. But cost is liable to be an issue. Bluebird Bio priced its first gene therapy, Zynteglo (marketed for a different rare genetic disease, transfusion-dependent beta-thalassemia), at $1.8 million, so I expect it to price Skysona similarly. With approximately 6,000 CALD patients in Europe, that's an addressable market worth just over $10 billion.

Despite Zynteglo's great benefits for patients, Bluebird has had trouble reaching reimbursement agreements with payers for the treatment. The biotech even withdrew Zynteglo from the German market in April after it could not agree on a price with that nation's public health insurance system. There is also the question of who is diagnosing such a rare disease. In most EU countries, newborns are not screened for CALD, and in the U.S., only 20 states and the District of Columbia screen for it. Unfortunately, the lack of testing may be leaving a significant percentage of CALD patients undiagnosed.

All of this may at least be partially factoring into Wall Street's weak revenue forecasts for Skysona. One analyst at Jefferies projects peak annual sales of only $74 million for the CALD gene therapy.

While Skysona can be a life-extending therapy for CALD patients, as Zynteglo is for transfusion-dependent beta-thalassemia patients, these are incredibly rare diseases. Bluebird Bio's next target, sickle cell disease, is more common, affecting approximately 100,000 Americans and 1 out of every 365 African-American births, according to the Centers for Disease Control and Prevention. Given that its sickle cell disease treatment utilizes a lentivirus-based delivery system similar to the ones used by Zynteglo and Skysona, the company will have quite a bit of safety data to lean on when it goes to the FDA for approval of its sickle cell treatment. That's tentatively scheduled to happen in 2023.

While CRISPR-based treatments have generated a lot of hype,the sickle cell treatment utilizing CRISPR that is the most advanced in clinical trials is CTX001, which is being developed jointly by CRISPR Therapeutics (NASDAQ:CRSP) and Vertex (NASDAQ:VRTX). Thus far, they have only delivered data on seven CTX001 recipients in a phase 1/2 trial. By contrast, Bluebird Bio's sickle cell therapy has already moved into phase 3 trials after showing significant promise in its earlier-stage studies. So Bluebird Bio seems to have at least a two- to three-year lead on its CRISPR-based rivals in its efforts to get a therapy approved for this large potential market.

In a sense, Zynteglo and Skysona are warmups for Bluebird Bio as it prepares for all of the pricing issues and negotiations that will come with the massive addressable market that is sickle cell disease gene therapy. With approximately 100,000 people suffering from the disease in the U.S. alone, even at just 5% penetration, this could be a $10 billion a year opportunity. If it can approach that level of market penetration, Bluebird Bio could surge past its current market-cap peak to reach over $15 billion -- which would make it just under a 10-bagger from here. And that does not factor in the estimates for more than $1 billion in annual sales for Abecma by 2026.

Considering that Bluebird Bio currently has a market cap of $1.7 billion with just over $1 billion in cash on the books, there is a lot of potential upside here. Given its significant head start in the race to FDA approval for a sickle cell gene therapy, plus significant experience in the gene therapy field from its other endeavors, I'm rooting for this cutting-edge biotech to overcome its prior missteps and reward both patients and investors.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Is This Small-Cap Biotech a Buy After Its Second Approved Gene Therapy? - Motley Fool

Gilles Salles, MD, discusses the need for new therapies to treat diffuse large B-cell lymphoma.

Gilles Salles, MD, the lymphoma service chief at Memorial Sloan Kettering Cancer Center, discusses the need for new therapies to treat diffuse large B-cell lymphoma (DLBCL).

According to Salles, once patients fail primary therapy, limited options remain. For approximately 50% of the patient population, those with limited comorbidities or those under a certain age, the standard of care is salvage chemotherapy followed by stem cell transplant.

However, for the approximately half of patients who are not eligible for this route, the treatment is usually immuno-chemotherapy, according to Salles. For most regimens, the response rate is limited. For patients who do respond, the duration of response (DOR) is typically only between 4 to 6 months. The median survival after the second-line therapy is about a year.

News agents have made progress in this space. For example, chimeric antigen receptor T cells show promise, but eligibility and access remain a major hurdle for many patients. New agents are also in the pipeline, though according to Salles, many have a short DOR.

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Improving the Treatment Gap in Diffuse Large B-Cell Lymphoma - Targeted Oncology

Myelodysplastic syndromes, known commonly as MDS, are a group of bone marrow diseases characterized by bone marrow failure, or an inadequate production of blood counts called cytopenia.

In a presentation at the CURE Educated Patient Leukemia Summit, Dr. Rami Komrokji, section head for Leukemia and MDS and Vice Chair of the Department of Malignant Hematology at Moffit Cancer Center, gave a run down on the diagnosis and staging process for MDS.

Diagnosing MDS

Komrokji explained that the myelo- prefix means bone marrow, and -dysplasia means abnormal-looking cells. When a patient has cytopenia, they may experience certain symptoms.

If patient is anemic, they will have shortness of breath, fatigue, palpitations, said Komrokji in an interview with CURE. If they have low platelets, they will have bleeding tendency, bruising. If they have low white blood cell counts, they will have maybe infections. So usually, either some of those symptoms will prompt blood testing, or on routine physical exam, the patients are found to have low blood counts. So that's usually the initial step.

Doctors will usually look into nutritional deficiencies such as B12, folate and ferritin, said Komrokji. Eventually, the patients will get a bone marrow aspirate and biopsy to diagnose their disease, which includes several parts.

There is the morphologic part, which means the pathologists are looking at the cells under a microscope, explained Komrokji. And then there is also some genetic testing. We look at cytogenetics nowadays, we look at gene mutations. So we put all of this information together to make the diagnosis.

The hematopathologist must see dysplasia, increased myeloblasts (immature cells known as blasts within the bone marrow) or certain cytogenetic abnormalities to make their diagnosis, Komrokji said.

Sometimes the diagnosis is straightforward, but sometimes it could be challenging, he added. It truly depends on an experienced hematopathologist to make the diagnosis.

Staging and Risk Stratification

Once a patient receives an MDS diagnosis, their doctor will go over risk stratification, or understanding what the risk of their disease is, which is what they consider staging, Komrokji said.

Now in MDS, its not like a lung cancer or colon cancer, he said. The disease does not spread around. The staging is based on the blood counts, on the percentage of those myeloblasts or immature cells (and) the chromosomal makeup of the cells. And nowadays, we sometimes also incorporate the presence of gene mis-happenings as well. So we get a lump score to estimate the risk.

Doctors typically use the International Prognostic Scoring System (IPSS) to categorize patients into one of five categories very low, low, intermediate, high and very high. The risk is the impact on survival and whether the disease will transform to leukemia, Komrokji explained. The disease risk must be known in order to tailor the patients treatment to them.

I always advise patients to see a specialized center in MDS, because obviously, those are not that common diseases, he said. A community oncologist could see a few (cases) per year, while an experienced center like in our place, we see like 15 to 20 per week.

Gene Mutations

Komrokji said that understanding gene mutations is an evolving field that is slowly becoming routine.

I advise all patients to inquire if theyve gotten genetic testing or not, he said. This sort of testing will help them understand any abnormalities. Doctors can look at a patients individual gene levels and detect for mutations, of which at least one was identified in 90% of patients with MDS.

Understanding the patients mutation(s) helps them tell whether there is a clonal hematopoiesis or mis-happening that occurred. It can also impact prognosis and allow them to further understand the disease risk.

And finally, some of them are targetable or important to follow through the treatment, said Komrokji. So patients should probably definitely have a genetic testing done. And sometimes after a treatment failure, we repeat it because we see other mutations that we could target with new drugs.

What Causes MDS?

In most cases, the cause of a patients MDS diagnosis is unknown.

We think it's phenomena of senescence or aging of those stem cells in the bone marrow that produces the blood, said Komrokji. Obviously, the process is very complicated. We have billions and billions of cells divide billions of times a day. So you know, as those cells age, mistakes can happen in them.

In most cases, he said, the mis-happenings which lead to the disease are random and at no fault of the patient. It is extremely rare for MDS to be inherited through familial genes.

There are, however, several known risk factors of MDS. If someone has history of another form of cancer and has received chemotherapy or radiation therapy, they may have possible stem cell damage and can develop MDS this is called therapy-related MDS. There has also been association of the disease with benzene exposure, chemical exposure and radiation exposure. Patients who have connective tissue diseases such as rheumatoid arthritis and lupus are at a slightly higher risk of getting MDS due to inflammation in the body and certain medications used to treat those diseases.

I would say there's a lot of better understanding of the disease in the past several years, of genetic mutation testing and incorporating them in practice, Komrokji said. And I think, you know, there are a lot of new treatments on the horizon for patients; there are several clinical trials in advanced phase.

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The Basics of MDS: Diagnosis and Staging - Curetoday.com

Adam Sperling, MD, PhD, discusses future research with CAR T-cell therapy in multiple myeloma.

Adam Sperling, MD, PhD, a physician at the Dana-Farber Cancer Institute, and an instructor in medicine at Harvard Medical School, discusses future research with CAR T-cell therapy in multiple myeloma.

Randomized phase 3 studies of idecabtagene vicleucel (ide-cel; Abecma) and ciltacabtagene autoleucel (cilta-cel) compared with standard-of-care therapies in relapsed/refractory multiple myeloma are ongoing, says Sperling. Although no data are yet available, it is likely that CAR T-cell therapies will outperform standard therapies in this setting, Sperling adds.

Additionally, CAR T-cell therapy is being evaluated in earlier lines of treatment for patients with multiple myeloma, Sperling says. The products may have utility in high-risk patients who progress following first-line treatment, or as an option after or instead of stem cell transplant, Sperling says. Combination strategies with CAR T-cell therapy are also emerging to determine whether patients can achieve deeper and more durable responses, Sperling explains.

Finally, ongoing research efforts are focused on understanding and overcoming mechanisms of resistance to CAR T-cell therapy, Sperling says. Dual-targeting CAR T-cell therapies or combination approaches with CAR T-cell therapy and agents, such as gamma secretase inhibitors, may be useful in combatting resistance to CAR T-cell therapy in multiple myeloma, concludes Sperling.

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Dr. Sperling on Future Research With CAR T-Cell Therapy in Multiple Myeloma - OncLive

SAN DIEGO--(BUSINESS WIRE)--Stemson Therapeutics announced today the closing of a DCVC Bio-led $15 million Series A financing to advance development of Stemsons proprietary therapeutic solution to cure hair loss. Genoa Ventures, AbbVie Ventures and other investors join in supporting Stemsons efforts to restore human hair growth with a novel cell regeneration technology using the patients own cells to generate new hair follicles.

In addition, Kiersten Stead, Ph.D., Co-Managing Partner at DCVC Bio and Jenny Rooke, Ph.D., Managing Director at Genoa Ventures will join Stemsons Executive Chairman Matt Posard and Chief Executive Officer and co-founder Geoff Hamilton on the board of directors. Dr. Stead invests in early-stage companies that build novel deep tech businesses in the life sciences. Stead received a Ph.D. in Molecular Biology & Genetics and an MBA in finance from the University of Alberta. Dr. Rooke is founder and Managing Director at Genoa Ventures where she specializes in early-stage companies innovating at the convergence of technology and biology. Rooke received a Ph.D. in Genetics from Yale University and a degree in physics from the Georgia Institute of Technology.

We are excited and honored to welcome DCVC Bio and a fantastic syndicate of investors to the Stemson team. The Series A funding will help us optimize our solution for human skin structure and environment so we can go into our first human clinical trial with high confidence for a positive outcome. We have the technical and biological building blocks to successfully address hair loss that overcomes failures of past therapies, said Hamilton. The addition of key venture capital investors DCVC Bio, Genoa Ventures and AbbVie Ventures broadens and strengthens our investor base. DCVC Bio and Genoa Ventures are successful early-stage development investors, and I am pleased to welcome Dr. Stead and Dr. Rooke, our newest board members, to the team. In addition, the AbbVie Venture investment comes on the heels of an initial seed investment from Allergan Aesthetics in 2020, and the continued industry interest in our technology is encouraging.

Globally, hundreds of millions of men and women suffer from various forms of hair loss. Though there are many possible causes of hair loss, including chemotherapy, autoimmune disease, scarring, and genetics, all can result in a loss of self-esteem and cause depression, anxiety and other mental health disruption for those affected. The hair restoration market is expected to exceed $13.6 billion by 2028, and no solution today is capable of generating an unlimited new supply of healthy follicles for patients in need.

Almost 30 years have passed since the last FDA-approved hair loss treatment, yet millions still suffer the physical and mental impact of losing their hair each year, stated Dr. Stead. Stemsons novel stem cell engineering platform has the potential to cure hair loss once and for all, treating not only the physical symptoms of this complex problem, but the mental burden as well.

"The team at Genoa is impressed with Stemsons vision to blend biology and technology and apply it beyond traditional biotech," added Dr. Rooke. "By combining exciting advancements in iPSCs with novel technologies in materials and data sciences, Stemson exemplifies the kind of chimeric teams Genoa seeks to support on their journey to become a category-defining company."

The Series A financing brings the total funding raised to date to $22.5 million and allows Stemson to further the next stage of research and development of its cell engineering platform, where is it being combined with bioengineered material and robotic delivery as a novel solution for natural hair replacement. Currently, Stemsons research and development efforts are focused on developing an optimized solution for human skin structure environment in larger animal models. Stemsons Induced Pluripotent Stem Cell (iPSC) based technology is capable of producing the cell types required to initiate hair follicle growth and have been successfully tested in small animal models.

About Cell Regeneration Technology

Human Induced Pluripotent Stem Cells (iPSC) have the unique capability to replicate indefinitely and give rise to all cell types of the human body, including the cell types required for repair. iPSC-based technology is capable of producing the cell types required to initiate hair follicle growth. As a new therapeutic platform, iPSCs represent an emerging area of regenerative cell therapy. Stemson is one of a growing number of companies at the forefront in developing iPSC-based treatments.

About DCVC Bio

For over twenty years, DCVC and its principals have backed brilliant entrepreneurs applying Deep Tech, from the earliest stage and beyond, to pragmatically and cost-effectively tackle previously unsolvable problems in nearly every industry. DCVC Bio specializes in supporting life sciences platform companies at the intersections of engineering and therapeutics, industrial biotechnology and agriculture. For more information, please visit https://www.dcvc.com/companies.html#dcvc-bio

About Genoa Ventures

Genoa Ventures invests in early-stage companies working at the convergence of biology & technology to accelerate the pace of innovation, transform industries, and solve some of the most fundamental challenges to life. Genoa, identifies opportunities early and focuses its investments and expertise to empower the next great category-defining companies. The Genoa team has a unique chimeric blend of experience from scientific research and discovery to executive management in the life sciences and technologies sectors. The team applies this diverse experience to provide expert guidance to its companies and stellar returns to its investors.

About AbbVie Ventures

AbbVie Ventures is the corporate venture capital group of AbbVie. We are a strategic investor, investing exclusively in novel, potentially transformational science aligned with AbbVie's core R&D interests. We measure success primarily by the extent to which our investments foster innovation with potential to transform the lives of patients that AbbVie serves. AbbVie Ventures enables its portfolio companies with both funding as well as access to AbbVie's internal network of experts across all phases of drug development, from drug discovery through commercialization. For more information, please visit http://www.abbvie.com/ventures

About Stemson Therapeutics

Stemson Therapeutics is a pre-clinical stage cell therapy company founded in 2018 with a mission to cure hair loss by leveraging the regenerative power of Induced Pluripotent Stem Cells. Based on the breakthrough innovation by Stemson Therapeutics co-founder, Dr. Alexey Terskikh, Stemson uses iPSC to regenerate the critical cells required to grow hair and which are damaged or depleted in patients suffering from hair loss. The iPSC-derived cells are used to grow de novo hair follicles, offering a new supply of hair to treat people suffering from various forms of Alopecia. Today, there are no available treatments capable of growing new hair follicles. Stemsons world class team of scientists, advisors and collaborators are passionate about delivering a scientifically based, clinically tested cure for hair loss to the millions of hair loss sufferers who seek help for their hair loss condition. Stemson Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.stemson.com.

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Stemson Therapeutics Secures $15M Series A Funding to Cure Hair Loss - Business Wire