Category Archives: Regenerative Medicine

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.

The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

In addition to RMAT designation, CTX001 has received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered 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 and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex 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 treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

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 partnerships 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 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 regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of RMAT designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and 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 impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; 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, 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.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program and related global regulatory approvals, and expectations regarding the RMAT designation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-961-0534orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001 for the...

Except for hard metals like stainless steel, gold and titanium, most synthetic materials in the body create a very significant scarring response. Credit: INSERM

The power of needlework should never be underestimated. Where would we be without the textile industry providing natural and synthetic fibres to thousands of sectors across the globe? In addition to clothing our bodies, textiles are used to help heal them, from wound dressings to sutures and meshes. Some scientists are now going further, using weaving and knitting techniques to create medical fabrics from biological materials such as human cells.

Nicolas LHeureux, director of research at INSERM, the French National Institute of Health and Medical Research is exploring exactly this. He works on repairing damaged blood vessels with biological textiles grown in the laboratory.

Synthetic materials are recognised as not being a normal part of the body, LHeureux explains. Except for hard metals like stainless steel, gold and titanium, most create a very significant scarring response.

He likens it to a splinter. The body recognises the material as foreign and reacts to it by trying to push it out. If its not able to eject it, scar tissue will form and inflammation will be triggered. This can lead to redness, pain, swelling and scarring.

Some parts of the body are better at dealing with scarring than others. Sometimes a scar can actually be helpful, providing mechanical support to the structure. But in a blood vessel, a scarring response could recreate the same blockage surgeons were aiming to fix in the first place. Grafting and stenting can result in restenosis (where the treated vessel closes off again) over time due to this scarring behaviour.

A scarring response will create a lot of tissue that will clog the inner part of your tube. Then your blood will not flow well anymore, LHeureux says.

Synthetic grafts work best for large vessels such as the aorta, which is roughly two centimetres in diameter. If there is a little bit of scarring from the graft, the blood will likely still be able to flow normally. But for smaller blood vessels, rejection of synthetic materials can create a real problem.

LHeureux and his team are developing grafts that wont produce that rejection response. What is more likely to be accepted is a material the body is already familiar with. Researchers cultivate human cells in the laboratory (originally extracted from a skin biopsy), where various chemicals are used to influence them to form sheets of collagen. These sheets are then cut into thin threads of yarn-like material.

The material we collect in sheets is the extracellular matrix outside the cell. Thats what we get the cells to overproduce in the lab, LHeureux reveals. This is the material that they lay down in the right conditions at the bottom of the plastic containers where we grow the cells.

Because the makeup of collagen doesnt vary from person to person, it is hoped that each patient wouldnt need vessels produced from their own cells.

Once the yarn is ready, its time to get sewing. By weaving, braiding or knitting, the team can form tubes of collagen to replace the synthetic structures that are traditionally used in cardiovascular surgery.

We tend to use weaving because it makes a really nice tight wall which is really important if youre making a blood vessel, says LHeureux.

This is weaving as youve never seen it before, but it still requires a loom. The custom-made device has to be tiny so vessels of five millimetres in diameter can be produced. Its made out of stainless steel and plastic so it can be cleaned easily. And it is circular in shape to produce tubes from the collagen yarn.

How well these grafts will be tolerated by the body still needs to be tested though. The next step of the research is to see how the vessels perform in animals. Using genetically modified rodent models that dont reject human tissue, the team will be able to see if the biological textiles adapt well to the body environment.

But one problem with rats and mice is the small size of their blood vessels. So, the researchers are also working on producing similar medical textiles using sheep cells. Once animal model results prove promising, itll be time to think about transplanting the grafts into humans.

The sheep is about the same size as a human in terms of the blood vessels, so well be able to try surgeries that we would do in humans with vessels that would be the same size and using all the same instruments. So, its much more representative, LHeureux explains.

There are other research groups working on similar projects.The University of Minnesota Medical School recently grew human-derived blood vessels in a pig. While US company Humacyte is also trying to produce extracellular matrices for vascular and non-vascular applications. In the future, LHeureux hopes to collaborate with groups like these to find the best way of producing biological textiles at scale.

When we bring this material that is completely logical, completely human and integrates well inside the body to patients, well have a solution that weve never had before in medicine.

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Spin me a yarn: the future of medical textiles and regenerative medicine - Medical Device Network

CTX001is an investigational therapy that Vertex Pharmaceuticals and CRISPR Therapeutics are developing to treat inherited disorders of hemoglobin such assickle cell diseaseandbeta-thalassemia.

Sickle cell disease is caused by a mutation in the HBB gene. This gene provides instructions to make the protein hemoglobin. Hemoglobin is a molecule inside red blood cells that is responsible for carrying oxygen. In sickle cell disease, the mutations result in missing or deficient hemoglobin.

CTX001 uses gene-editing technology to make a genetic change to increase the production of fetal hemoglobin in patients red blood cells. Fetal hemoglobin is a form of hemoglobin that exists naturally in newborn babies. The body later replaces it with the adult form of hemoglobin. However, sometimes fetal hemoglobin persists in adults, providing protection for people with sickle cell disease and beta-thalassemia.

For the treatment, researchers first collect a patients hematopoietic stem cells. These are cells from the bone marrow that give rise to all the red and white blood cells. They then genetically modify these cells in the laboratory so they are able to produce high levels of fetal hemoglobin. Finally, they reintroduce them into the patients body, where they will produce large amounts of red blood cells containing fetal hemoglobin.

Researchers presented the results of preclinical experiments with CTX001 at the American Society of Hematology (ASH) Annual Meeting in December 2017. CTX001 was able to efficiently edit the target gene in more than 90% of hematopoietic stem cells to achieve about 40% of fetal hemoglobin production. Investigators believe this is sufficient to improve a patients symptoms. Study results also showed that CTX001 affects only cells at the target site, thereby appearing to be a safe potential treatment.

These positive results prompted CRISPR topartner with Vertex to further develop CTX001. The goal is to market CTX001 as a gene-editing treatment for inherited hemoglobin disorders, including sickle cell disease and beta-thalassemia.

A Phase 1/2 clinical trial (NCT03745287) called CLIMB-SCD-121 was started in November 2018 to investigate the use of CTX001 in sickle cell disease. The open-label, multi-site, single-dose trial is recruiting 45 patients, ages 18 to 35, with severe sickle cell disease in the U.S., Canada, Belgium, Germany, and Italy. Researchers will give participants a single intravenous (into the bloodstream) infusion of CTX001. They will monitor the safety and effectiveness of the treatment for six months to two years.

Researchers reported preliminary results for the first patient in November 2019. Before treatment, the patient averaged seven vaso-occlusive crises (VOCs) a year. At four months after treatment, they were free of VOCs and had hemoglobin levels of 11.3 g/dL. The estimated completion date of the trial is May 2022.

The U.S. Food and Drug Administration (FDA) granted CTX001 fast track designationin January 2019. This designationallows for faster development and review of drugs that treat a serious medical issue and fill an unmet need.

In May 2020, the FDA also granted the therapy the designation of regenerative medicine advanced therapy (RMAT) for treating severe sickle cell disease and transfusion-dependent beta-thalassemia. The purpose of RMAT is to expedite the development and review of new therapies that treat serious or life-threatening medical conditions, or when they show significant clinical benefits over existing therapies.

Last updated: May 13, 2020

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Sickle Cell Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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zge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

Link:
CTX001 for Treatment of Sickle Cell Disease and Other Blood Disorders - Sickle Cell Anemia News

DUBLIN, May 13, 2020 /PRNewswire/ -- The "Asia-Pacific CAR-T Pipeline" report has been added to ResearchAndMarkets.com's offering.

The Asia-Pacific CAR-T pipeline features over 50 regenerative medicine companies focusing on CAR-T in Asia. The report derives insights from in-depth primary and secondary research studies and a comprehensive pipeline analysis of companies from China, Taiwan, Japan, Korea, India, Australia & SEA. It includes a disease-wise product analysis of the latest technologies and CAR-T developments available, along with insights on stage of development, scale of operation, co-development and technology partners involved and future prospects.

The report will give an assessment of the clinical trial and regulatory approval timelines, financial performance of the companies involved and provide recommendations on possible co-development and technology partnerships in the near future. In addition, it pinpoints the pain points of CAR-T therapy companies and identifies gaps in addressing certain diseases, manufacturing and supply chain capabilities and technologies available. It also provides a company-wise swot analysis. The report seeks to inform the biopharmaceutical industry on the current status of CAR-T therapies in APAC, uncover the potential for CAR-T therapy success in the region and discover future opportunities for partnership.

Furthermore, the report covers region specific growth drivers and insights into ongoing CAR-T therapy clinical trials. In addition, the directory presents a regulatory landscape assessment and a review of co-development opportunities. Moreover, in-depth studies on the CAR-T landscape have supplemented the report with a swot analysis and future forecasts. It provides readers a closer look into the pipelines of Nanjing Legend Biotech, CARsgen Therapeutics, JW Therapeutics, AbClon, BiocurePharm, Eutilex, Daiichi Sankyo, Celgene, Otsuka and many more.

Key Topics Covered

Section 1

Section 2: Company-Wise

Section 3: Region-Wise

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/6ywu77

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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CAR-T Pipeline Assessment in Asia-Pacific, 2020 - Take a Closer Look Into the Pipelines of Nanjing Legend Biotech, CARsgen Therapeutics, JW...

Technology across the world is improving and innovatingwith time. Over the years, man-managed labor has almost finished from themarket and more and more technological and scientific gadgets are taking placemaking human labor more effective, efficient, and precise.

Medical science has also taken a lot of advantage fromthis scientific advancement therefore, we can say that doctors are making fulluse of science and technology and the world of medicine has evolved quiterapidly.

Orthopedic hospitalshave also seena remarkable transformation over time and the days when a regular orthopedicclinic only comprised of a few tools and a bad. The launch of innovativetechnologies, biologics, and hybrid items into the orthopedic industry isincreasingly growing.

Any of these emerging inventions gain regulatory approvalby showing significant equivalence to the US System of the Food and DrugAdministration (FDA) 510(k).

Surgeons play a key role in the implementation ofemerging technology to patients and will play a leading role in supportinghealthy, efficient, adequate, and cost-effective treatment, particularly forsurgical procedures. Surgeons will track and record the health results andadverse effects of their patients utilizing modern technologies and ensure thatthe new technology works as expected.

Ortho-biologics utilizes the regenerative ability ofcells in the human body. Ortho-biologics are created from compounds naturallypresent in the body which are used to facilitate the recovery of fracturedbones which injured joints, ligaments, and tendons.

These involve bone graft, growth factors, stem cells,platelet-infused plasma, autologous blood, and autologous controlled serum. Themesenchymal stem cells (MSCs) contained in the bone marrow has been shown to besuccessful in the production of the appropriate tissues.

Result in Orthopedic Procedures

Recent advances in this area, including growth factor andstem cell therapies, may contribute to faster recovery. One breakthrough isdrug-free bone grafts, which may be used to cure conditions such as orthopedicsurgery. Clinical trials have demonstrated that growth factors can improve thehealing cycle.

Stem cells will continually self-regenerate and transforminto either form of cell, providing an unmatched source of regenerativemedicine technology. Definitions of musculoskeletal procedures utilizing stemcells are listed below.

Biotechnology firms began utilizing orthopedic stemcells. For starters, BioTime works on stem cell therapies for age-relateddegenerative diseases, IntelliCell BioSciences on adipose-derived stem cellsfor orthopedic conditions, and Bio-Tissues on Ortho-biological treatments forcartilage defects.

Orthopedic procedures using robots are less intrusive anddeliver reproducible accuracy, resulting in shorter hospital stays and quickerrecovery times. The Swiss clinic, La Source, recorded a decline in averagehospitalization from 10 to 6 days with the usage of surgical robots.Nevertheless, this technology is also costly to develop, so solid,evidence-based trials are required to prove that robotic technology contributesto improved outcomes.

The Da Vinci Surgical Method became the first U.S. Food andDrug Administration (FDA)-an authorized robotic surgery program in 2000. Morebusinesses are investing in this technology to enhance navigation duringservice or to receive 3-D scans that aid in the design of custom joints.

Organizations that are interested, in robotics areinclined towards the following technological masterpieces:

Several modern surgical techniques are enhancing theresults. These involve motion-preservation methods, minimally intrusive surgery,tissue-guided surgery, and cement-free joint repair.

Motion recovery strategies require partial or completedisk removal and the usage of active stability systems and interspinous spacersthat do not impair versatility.

Minimally intrusive procedures involve the use ofendoscopes, tubular retractors, and computer-aided guidance devices, allowingan incision of just 2 cm instead of 12 cm in conventional therapies. Minimallyinvasive treatments are gaining popularity in joint and hip replacement and spinalsurgery.

Smart devices provide built-in sensors to offer real-timetracking and post-operative evaluation details to surgeons for better patientsafety across the clinical process. Such implants have the ability to minimizeperiprosthetic infection, which is an increasing orthopedic issue.Sensor-enabled innovations also presented health care professionals with arange of innovative, cost-effective goods.

Companies working in this field include:

3-D orthopedic printing is gaining traction in themanufacture of personalized braces, surgical equipment, and orthotics from arange of materials. 3-D printing technology cuts operating times, saves energy,increases the long-term reliability of the implant, and enhances the healtheffects of surgical procedures. 3-D printing technologies of orthopedics areinclusive of:

Companies investing in 3-D Orthopedic Printing

Medical science has taken a huge turn with the introduction of technology. The orthopedic industry has also transformed to a huge extent making sure that the specialists and surgeons are able to treat and operate on their patients without any hassle. Almost all the orthopedic hospitals are equipped with high-end gadgets and tools to assist the doctor.

Even though the technology has evolved greatly since thefield of medicine was invented, it is important to understand that this is justa beginning and there are many more things to come in the future.

Read more from the original source:
The Latest Technological Innovations in Orthopedic Surgery 2020 Technology - IMC Grupo

While some technology companies have been using 3D printing, or additive manufacturing, to address the personal protective equipment shortage caused by the novel coronavirus, others are using 3D printing technology to create samples of human organs and tissues for coronavirus testing.

As public health officials and philanthropists race to find treatments for COVID-19 the respiratory disease caused by the novel coronavirus researchers at universities and companies, like Novoheart Holdings Inc., are trying to speed up traditional drug and vaccine development processes without compromising patient safety by using 3D bioprinting.

Like traditional 3D printing, bioprinting uses a device to construct a three-dimensional model created with a computer-aided design system. Instead of using plastic filaments, however, bioprinters use cells mixed with biocompatible materials, called bioinks, to create three-dimensional systems that act like human organs and tissues. These systems can then be deployed in tests by drug developers or, in some cases, implanted into the human body.

This process of creating human organs and tissues out of pre-existing cells is not a new concept; the Wake Forest Institute for Regenerative Medicine produced new organs by hand for years for patient transplants, said Anthony Atala, the institute's director.

A 3D-printed ear scaffold

Source: Wake Forest Institute for Regenerative Medicine

Mini organs, major research

But the 3D-printing machines let Atala and his team automate the regenerative medicine process, and a new area of bioprinting has emerged: miniature organs.

These 3D-printed samples of organs are placed within microchips that are connected with a system of microfluidics, Atala said, so that they act like actual human organs. With these organs, researchers can test drugs and therapies for toxicity in the human body without human or animal model testing.

Atala said that some drugs may show no toxicity in cell cultures, animal models and human clinical trials, but after a few years on the market, these drugs prove harmful to humans.

For example, in 2000, the U.S. Food and Drug Administration recalled the diabetes drug Rezulin after it was found to cause liver failure, and 63 people died.

However, Atala said that when his team tested Rezulin on their miniature organ systems, they discovered toxicity in the liver within two weeks.

"You metabolize drugs differently than I do, you have different genetics than I do, your weight is different than mine, your size is different than mine, how you eliminate your drugs is different than mine," Atala said. "And when you put in the chip, you're getting rid of all that noise. It's really the drug against the organ."

While these miniature organs have proven effective in drug testing, Atala does not expect human clinical trials to go away any time soon. The organs need to be standardized and validated, and from a regulatory standpoint, human trials are still required.

"The hope is to use these systems initially to help select the best therapies before they get to patients," Atala said.

Heart in a jar

Now researchers are using these miniature organs to select the best therapies to fight COVID-19.

Novoheart uses 3D bioprinting to create a human ventricular cardiac organoid chamber, referred to as a 3D "heart-in-a-jar."

Novoheart's 3D "heart-in-a-jar"

Source: Novoheart

At the moment, Novoheart is providing its miniature hearts to companies that want to test the effects of certain potential COVID-19 therapies, like hydroxychloroquine and azithromycin, on the heart.

This is especially important as the U.S. FDA recently cautioned patients against using hydroxychloroquine for COVID-19 use due to reports of serious heart rhythm problems often when taken in combination with azithromycin.

The miniature hearts enable researchers to observe the mechanisms by which these drugs cause arrhythmias in the heart without testing in humans, Roger Hajjar, Novoheart's chief medical officer, said in an interview with S&P Global Market Intelligence.

Another benefit is that they are also able to study the coronavirus's direct effects on the heart. Hajjar said that researchers at Novoheart, alongside researchers with an infectious disease laboratory in Hong Kong, have found that the virus can cause myocarditis, or inflammation of the heart muscle, in patients with COVID-19. Myocarditis can ultimately lead to heart failure or death if left untreated.

Similarly, Atala and his team, in collaboration with George Mason University, are injecting miniature organs directly with the coronavirus in order to see how each organ becomes infected. In the future, the Wake Forest Institute for Regenerative Medicine team aims to study COVID-19 drugs' effects on the organs.

As many companies speed up the production of coronavirus therapies and typical regulatory guidelines are set aside, 3D bioprinting may see greater use as researchers seek a quick way of testing the effects of these drugs, proponents said.

Read more here:
3D-printed mini-organs may provide safe testing of COVID-19 therapies - S&P Global

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--jCyte Inc., a biotech company dedicated to improving the lives of patients with rare degenerative retinal diseases, today announced it has entered into a licensing agreement with Santen Pharmaceutical to develop and commercialize its first-in-class, investigational therapy, jCell, outside the U.S., in regions including Europe, Asia and Japan.

Under the terms of the licensing agreement, jCyte will receive $50 million in upfront cash, $12 million in a convertible note offering, and $190 million in clinical and sales milestones based on regulatory approval and initial sales in Europe, Asia and Japan. The total deal is valued at up to $252 million. jCyte is also entitled to receive tiered, double-digit royalty payments on net sales of jCell therapy once commercialized outside the U.S.

We are thrilled to partner with Santen, a global market-leader in ophthalmology. By leveraging Santens large, existing global commercial and medical infrastructure in ophthalmology, as well as its commitment to commercializing cell and gene therapies, we help ensure that more patients with retinitis pigmentosa who live outside the U.S. will have access to this technology, said Paul Bresge, Chief Executive Officer, jCyte, Inc. We intend to use the proceeds from this transaction to continue development of our lead investigational therapy jCell, to improve the lives of patients with retinitis pigmentosa, as well as other degenerative retinal diseases.

jCell is a first-in-class investigational treatment for retinitis pigmentosa, an inherited retinal disease. The treatment is a minimally-invasive intravitreal injection, which can be performed in an ophthalmologists office with topical anesthetic. The entire procedure takes less than 30 minutes. The principal mechanism of action is the release of neurotrophic factors that may rescue diseased retinal cells. jCell therapy aims to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated. jCell has been developed with support from the California Institute for Regenerative Medicine (CIRM), which has funded previous preclinical development and ongoing clinical studies.

In the United States, the evaluable portion of a Phase 2b clinical trial of jCell for the treatment of retinitis pigmentosa has been completed, and the crossover portion continues. jCell therapy has been administered in over 100 patients. The U.S. Food and Drug Administration (FDA) has granted jCyte Regenerative Medicine Advanced Therapy (RMAT) designation based on early clinical data, making jCell potentially eligible for BLA priority review. In addition to RMAT, jCell has received Orphan Drug designation from the FDA and the European Medicines Agency (EMA).

About Retinitis Pigmentosa (RP)

Retinitis pigmentosa is a rare, genetic condition that progressively destroys the rod and cone photoreceptors in the retina. It often strikes people in their teens, with many patients rendered legally blind by middle age. Worldwide, an estimated 1.9 million people suffer from the disease, including approximately 100,000 people in the U.S., making it the leading cause of inheritable blindness.

About jCyte, Inc.

jCyte, Inc. is a clinical-stage biotech company focused on developing jCell therapy for retinitis pigmentosa (RP) and other degenerative retinal disorders. jCell is a first-in-class investigational treatment for retinitis pigmentosa, an inherited retinal disease. The treatment is minimally-invasive and given as an intravitreal injection. There are currently no FDA approved therapies for RP. The company is pioneering a new era of regenerative therapies to treat patients with unmet medical need. For more information, visit http://www.jcyte.com.

About Santen

As a specialized company dedicated to ophthalmology, Santen carries out research, development, marketing, and sales of pharmaceuticals, over-the-counter products, and medical devices. Santen is the market leader for prescription ophthalmic pharmaceuticals in Japan and its products now reach patients in over 60 countries. With scientific knowledge and organizational capabilities nurtured over a nearly 130-year history, Santen provides products and services to contribute to the well-being of patients, their loved ones and consequently to society. For more information, please visit Santens website (www.santen.com).

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jCyte Inc. Enters into Ex-US Licensing and Commercialization Agreement for jCell Therapy with Global Ophthalmology Leader Santen Pharmaceutical -...

-One-Year Results from Randomized, Double-Blind, Placebo-Controlled Study Demonstrate Improved Performance of Upper Limb (PUL) 2.0 (p=0.05)-

-First Ever Study in DMD that Correlates Stabilization in Cardiac Function with a Reduction in a Biomarker of Cell Damage-

-Company Planning to Meet with FDA to Discuss Pathway to Approval-

-To Host Conference Call and Webcast Today at 8AM ET-

LOS ANGELES, May 13, 2020 (GLOBE NEWSWIRE) -- Capricor Therapeutics (Capricor) (NASDAQ: CAPR), a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment and prevention of diseases, announced today positive top-line 12-month results of the HOPE-2 clinical trial using CAP-1002 to treat patients in advanced stages of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Boys and young men typically lose their ability to walk in their teens and generally die of cardiac or respiratory complications by the 3rd decade of life. The datashowed improvements in upper limb, cardiac and respiratory function with p-values less than p=0.05 in multiple measures.

The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002 treated patients (p=0.05) with a mean change of 2.4 points over placebo patients. With the exception of steroids, preservation of function in DMD is uncommon. The placebo patients declined consistent with natural history, but in the treated group, most patients were stable or improved throughout the one-year treatment period.

The performance of the upper limb (PUL) is a clinically validated measure that evaluates upper limb (shoulder, arm, hand) strength in patients who are generally non-ambulant. Retention of upper limb function is important for self-care and preservation of human dignity and has become a focus for physicians and advocates to find treatments to help these later stage patients. The FDA has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application (BLA).

Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of the Department of Physical Medicine and Rehabilitation commented, I am incredibly pleased with the outcome of the HOPE-2 trial which demonstrated clinically relevant benefits of CAP-1002 which resulted in measurable improvements in upper limb, cardiac and respiratory function. This is the first clinical trial which shows benefit to patients in advanced stages of DMD for which treatment options are limited.

The data also showed global improvements in cardiac function as measured by ejection fraction (p=0.004) and indexed volumes (LVESV, p=0.01, LVEDV p=0.07). These are surrogate measures of cardiac function and are considered the gold standard in terms of relevance to long term outcomes. Remarkably, there is also a reduction in the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage. In normal human subjects, there is typically no CK-MB measurable in the blood. It is well accepted that continuous muscle cell damage in DMD leads to pathologically high enzyme levels associated with cardiac muscle cell loss. HOPE-2 demonstrated a reduction in CK-MB levels as compared to placebo (p=0.006). This is the first ever study in DMD that correlates cardiac functional stabilization with reduction of a biomarker of cell damage.

Linda Marbn, president and CEO of Capricor said, To date, there are no therapies to treat the cardiomyopathy associated with DMD. Based on the statistically and clinically meaningful improvements in these and other measures of skeletal, cardiac and respiratory performance, we have requested an End-of-Phase 2 meeting with FDA to discuss next steps and a pathway to approval of a Biologics License Application for CAP-1002 in DMD.

Phase II HOPE-2 Study Design

HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the Companys lead investigational therapy, CAP-1002, in boys and young men who are in advanced stages of DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point in the intent to treat (ITT) population. Approximately 80% of the patients were non-ambulant and all patients were on a stable regimen of steroids. Demographic and baseline characteristics were similar between the two treatment groups.

Study Results

Top-Line Efficacy Data:

Mean Change from baseline to 12 months (standard deviation) shown.ITT (intent to treat) population shown P-values are nominal values unadjusted for multiple testing Mixed model repeated measures analysis

Dr. Marban continued, We are delighted with the final data from HOPE-2. It has met our expectations in terms of clinical meaningfulness in this population of patients where treatment options are extremely limited. In HOPE-2, CAP-1002 was delivered by intravenous infusions given quarterly. The data suggests that CAP-1002 could delay the progression of DMD. We are excited to share this data with FDA and discuss next steps towards commercialization. We have had tremendous support from the DMD advocacy community and we are grateful to the patients and families who participated in this study so that we could reveal the impact of CAP-1002 in treating DMD.

Safety Update

CAP-1002 was generally safe and well tolerated throughout the study. With the exception of hypersensitivity reactions which were mitigated with a common pre-medication regimen, no safety signals were identified in the HOPE-2 trial.

The FDA has granted Capricors CAP-1002 RMAT and Orphan Drug Designation, and the FDA has also granted a Rare Pediatric Disease Designation to CAP-1002 for DMD. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted, covers the broad treatment of DMD. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher. This is the second clinical trial investigating CAP-1002 showing similar results in DMD. Capricor completed the HOPE-Duchenne (Phase I/II) trial published in Neurology, the medical journal of the American Academy of Neurology in 2019.

The Company has initiated a technology transfer with a leading global CMO to prepare for commercial manufacturing of CAP-1002.

Conference Call and Webcast Details

Capricor will host a conference call and webcast with slides today, May 13, 2020, at 8:00 a.m. ET to discuss the top-line results of the HOPE-2 study. To participate in the conference call, please dial 855-327-6838 (domestic) or 604-235-2082 (international) and reference the access code: 10009621.

To participate via a webcast, please visit: http://public.viavid.com/index.php?id=139843 to view the slides. The webcast will be archived for approximately 30 days and will be available at http://capricor.com/news/events/.

Financial Update for the First Quarter of 2020

The Company reported a net loss of approximately $2.1 million, or $0.30 per share, for the first quarter of 2020, compared to a net loss of approximately $2.5 million, or $0.75 per share, for the first quarter of 2019.

As of March 31, 2020, the Companys cash, cash equivalents and marketable securities totaled approximately $13.2 million, compared to approximately $9.9 million on December 31, 2019. As of May 12, 2020, the Company has 12,464,006 shares issued and outstanding.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment and prevention of diseases. Capricors lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also investigating the field of extracellular vesicles and exploring the potential of exosome-based candidates to treat or prevent a variety of disorders. The HOPE-Duchenne trial was funded in part by the California Institute for Regenerative Medicine. For more information,visit http://www.capricor.com and follow the Company on Facebook, Instagram and Twitter.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002

CAP-1002 consists of allogeneic off-the-shelf cardiosphere-derived cells, or CDCs, a type of cardiac cell therapy that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity. It is being investigated for its potential to modify the immune systems activity to encourage cellular regeneration. The cells function by releasing exosomes that are taken up largely by macrophages and T-cells and begin a cycle of repair. CDCs have been the subject of over 100 peer-reviewed scientific publications and administered to approximately 150 human subjects across several clinical trials.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities and Exchange Commission on March 27, 2020. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricors exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Media Contact:Caitlin KasunichKCSA Strategic Communicationsckasunich@kcsa.com212.896.1241

Investor Contact:Joyce Allaire LifeSci Advisors, LLCjallaire@lifesciadvisors.com617.435.6602

Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com310.358.3200

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Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients with Duchenne Muscular Dystrophy Treated with Lead Candidate CAP-1002...

ISPE drives industry-wide clarity of new regulations and guidance, advising on potential impact and facilitating practical solutions, seeking harmonization of regulatory expectations where desired and possible.

NORTH BETHESDA, Md. (PRWEB) May 13, 2020

The International Society for Pharmaceutical Engineering (ISPE) announced featured presenters representing the Food and Drug Administration (FDA) at the 2020 ISPE Biopharmaceutical Manufacturing Virtual Conference. Taking place 12 June 2020, this first of its kind fully interactive digital event features timely and topical presentations and panel discussions from 40+ global experts representing FDA, GSK, Kite Pharma, Novartis, Roche, and more.

Featured FDA presenters will provide updates on regulatory expectations, considerations, and perspectives on biotechnology and biological products.

ISPE drives industry-wide clarity of new regulations and guidance, advising on potential impact and facilitating practical solutions, seeking harmonization of regulatory expectations where desired and possible, said Tim Howard, PE, CPIP, President & CEO, ISPE. It's vital that the biopharmaceutical industry pay close attention to what the regulators have to say and that we continue to develop constructive relationships.

FDA Presenters:

Wilson Bryan, MD, Director, Office of Tissues and Advanced Therapies, FDA/CBERDr. Bryans keynote presentation will provide insights into current developments in regenerative medicine, future trends, and challenges.

Patricia Hughes, PhD, Branch Chief, Division of Microbiology Assessment, FDA/CDERDr. Hughes will share regulatory considerations of innovations in sterile manufacturing and will also join the closing Fireside Chat.

Richard Friedman, Deputy Director, Science & Regulatory Policy, FDA/CDERFriedman will provide updates on sterile products and change management.

Raj Puri, MD, PhD, Director, Division of Cellular & Gene Therapies, FDA/CBERDr. Puri will participate in the closing Fireside Chat featuring global regulatory and industry leaders who are working to frame the strategy for the era ahead.

In addition to regulatory insights, industry experts will share their knowledge and lessons learned on critical topics, including:

To explore the educational agenda and to register, please visit ISPE.org/Bio20.

About ISPEThe International Society for Pharmaceutical Engineering (ISPE) is the worlds largest not-for-profit association serving its members through leading scientific, technical, and regulatory advancement across the entire pharmaceutical lifecycle. The 18,500 members of ISPE are building solutions in the development and manufacture of safe, effective pharmaceutical and biologic medicines, and medical delivery devices in more than 90 countries around the world. Founded in 1980, ISPE has its worldwide headquarters and training center in North Bethesda, Maryland USA, and its operations center in Tampa, Florida USA. Visit ISPE.org for more information.

For more information, contact: Amy HenryMarketing Communications ManagerInternational Society for Pharmaceutical Engineering (ISPE)Email: ahenry@ispe.org ISPE.org

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FDA Leaders to Speak at the 2020 ISPE Biopharmaceutical Manufacturing Virtual Conference - PR Web

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Life Science, and many more. Trade barriers are further restraining the demand- supply outlook.

Extracellular Matrix Market accounted to US$ 24.30 Mn in 2018 and is expected to grow at a CAGR of 8.2% during the forecast period 2019 2027, to account to US$ 47.46Mn by 2027.

The growth is driven by that countries such as China, Japan, and India which are engaged in conducting several studies. For instance, Australia is also involved in the several studies and also it has held various conferences for the tissue engineering and other application of the extracellular matrix.

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The potential of regenerative medicine that facilitates tissue regeneration in the affected area reduced the requirement of tissue transplant. The extracellular matrix is derived from a readily available tissue source, it can stimulate the growth of tissue in vivo with minimal inflammation, and it is available off the shelf. These advantages of the extracellular matrix enables in the ideal soft tissue replacement treatment procedures to repair contour defects. Therefore, there is increase in the vascular reconstruction or the plastic surgeries are anticipated to grow the extracellular matrix during the forecast period.

The studies for the molecular cell biology of extracellular matrix (ECM) are being conducted across the world. The cell adhesion molecules (CAMs), other properties and advantages of the extracellular matrix are turning out to be beneficial area of discovery with several interesting factors for disease and disorders in animals and humans. These advantages are also include treating of some cancers such as breast cancer, pancreatic cancer, liver cancer and colon cancer.

The globalextracellular matrix marketby raw material segments was led by porcine segment. In 2018, the porcine segment held a largest market share of 41.09% of the extracellular matrix market, by raw material. However, the bovine segment is expected to be the fastest growing segments of the market in 2027 owing to more beneficial properties to treat various conditions which is expected to become the major factor for the growth of the extracellular matrix market.

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Extracellular Matrix Market to Reap Over US$ 47.46Mn in Revenues by End of 2027 - Cole of Duty