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Category Archives: Gene Medicine

Cell and Gene Therapies Shatter Prior Records, with Continued Growth Expected – BioSpace

Cell and gene therapies achieved record growth in 2020, surpassing prior high points in terms of financings and approvals. Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), painted a rosy picture despite a few challenges at the 2021 Cell & Gene State of the Industry Briefing during this years Biotech Showcase, held virtually January 11-15.

2020 was a record-shattering year for financing, with $19.9 billion raised in 2020, up from the $9.8 billion raised in 2019 and the $13.3 billion raised in 2018 the previous record, she said.

Follow-on financings, at $6.8 billion, accounted for the largest portion of funding, followed by venture capital at $5.6 billion and IPOs at $3.7 billion. Upfront payments from partnerships totaled $3 billion and private placements totaled $1.2 billion.

The number of large financings exceeding $200 million grew noticeably. Sana Bios $700 million private financing, announced last June, topped the field. Iovance and bluebird bio gained the most from follow-on financing, with fundings of $604 million and $575 million, respectively. Legend Bio led the list of IPOs, raising $487 million last May.

Large pharma continued to buy-in to biotechs for research, development, commercialization, and licensing agreements focuses largely on oncology and CNS disorders, Lambert said. She cited Sangamo, with two major collaborations with Biogen ($350 million) and Novartis ($75 million), though adding, Bayer is especially active.

In the stock market, Regenerative medicine companies outperformed the NASDAQ Biotech Index, she continued. Although stock prices for virtually everything plummeted in mid-March, biotech stocks rebounded. Cell based immuno-oncology (IO) share prices increased 80% from their January 1 levels. Gene therapy was close behind, with a 70% increase, and all publicly traded regenerative medicine companies saw a 50% increase.

Given the overall environment, it seems safe to predict that 2021 will be another good year for regenerative therapies.

Currently, there are approximately 1,100 gene, cell and tissue-based therapeutic developers throughout the world. Of those, the overwhelming majority are in North America, with 543. We saw a lot of growth in China, Japan, and Korea, she said, with 295 companies. Europe boasts 209.

Globally, 1,220 clinical trials are underway for regenerative medicine.

More than 100 clinical trials commended in the fourth quarter alone, Lambert said. Roughly, they are divided evenly among cell, gene, and IO. With 152 trials in phase III and nearly half of those in gene therapy, this offers strong support for predictions by both the FDA and EMA that there will be 10 to 20 advanced therapies approved each year through 2025.

What people often forget, Lambert said, quoting Amy Price, a mother of two gene therapy recipients, is that cell and gene therapies arent some fantastical futuristic thing. Two of the Price children received gene therapy in a clinical trial 10 years ago for metachromatic leukodystrophy (MLD), a historically fatal disease. That drug, Libmeldy, by Orchard Therapeutics, was approved in Europe in 2020, making it one of the most significant milestones of the year.

The benefits of cell and gene therapy have expanded beyond experimental treatments.

Patients are continuing to benefit from innovative therapies, Lambert said. We saw a significant number of gene therapy approvals in 2020. In addition to Libmeldy, she cited approvals of Zolgensma (by Novartis Gene Therapies) in Europe, Japan, and Canada; Tecartus (by Kite, a Gilead company) in the U.S.; and Luxturna (by Spark/Roche) in Canada.

Looking forward, oncology, and particularly IO, dominates the regenerative medicine landscape. Some 554 oncology trials are underway.

Investors have invested heavily in this space for some time, and IO comprises 50% of Phase I trials in cell and gene therapy, Lambert noted. Focus is increasing on allogeneic therapies as well as gene editing.

Central nervous system therapeutics are the second most popular therapeutic indication for regenerative therapies for the second year in a row, with 94 trials. 2020 saw promising data from the first-ever attempt iPSC therapy for Parkinsons disease.

Gene editing continues to advance in the clinic, she added. For the first time, a patient was treated with CRISPR therapy in vivo and, later, systemically with CRISPR. CRISPR Therapeutics and Vertex Pharmaceuticals shared data (during J.P. Morgan week) from a sickle cell trial of 20 patients.

Despite these scientific advances and investor enthusiasm, cell and gene therapies face challenges in terms of dosing and delivery, and chemistry, manufacturing and controls (CMC), Lambert admitted. Gene therapy represents almost half of the Phase III pipeline, so we expect to learn a lot quite soon.

While it goes without saying that 2020 was a challenging year, many of the repercussions of the COVID-19-related disruptions remain to be seen. They extend not only to operational and clinical disruptions but also to regulatory backlogs and the politicization of diagnostics and therapeutics, all set against the usual challenges of fast-moving science.

None-the-less, Lambert pointed out, There were some positive developments.

As she said, Medicare approved a new diagnosis related group (DRG) for CAR T cell therapy and promulgated a new rule for outcomes-based therapies, thus enabling a new payment model that ARM deems essential for cell and gene therapies. In Europe, the European Commissions pharmaceutical strategy now recognizes the importance of cell and gene therapy. We are encouraged that we can build on that starting point with the Commission, Lambert said. ARM also is expecting progress on n-of-one therapies for ultra-orphan indications this year.

Looking ahead to 2021, Lambert identified six regenerative therapies on the FDAs docket from Mallinckrodt, bluebird, BMS, PTC Bio, and Gensight Bio. All indications are that 2021 will be a fantastic year of scientific, technological, and clinical progress in this sector, Lambert predicted.

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Cell and Gene Therapies Shatter Prior Records, with Continued Growth Expected - BioSpace

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Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics – GlobeNewswire

-- Alliance will assess the use of Sareptas proprietary gene editing technology and Genevants proprietary LNP delivery platform for multiple neuromuscular targets --

-- Sarepta to have options for an exclusive license to Genevants LNP technology for four neuromuscular indications --

-- Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future milestones and royalties --

CAMBRIDGE, Mass., VANCOUVER, British Columbia, and BASEL, Switzerland, Jan. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industrys most robust and expansive lipid nanoparticle (LNP) patent estate,today announced a research collaboration and option agreement for the delivery of LNP-gene editing therapeutics in Sareptas pipeline for neuromuscular diseases. LNPs offer the potential for a non-viral approach to gene editing and can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues.

Gene editing has the potential to revolutionize the treatment of diseases caused by genetic mutations - including rare neuromuscular diseases - by permanently altering genes that lead to disease. Sarepta is pursuing a variety of approaches to genetic medicine including exon skipping, gene therapies and gene editing in pursuit of cures for rare diseases.

Under the terms of the agreement, Genevant will design and collaborate with Sarepta in the development of muscle targeted LNPs to be applied to gene editing targets in early stage development. Sarepta will have rights to an exclusive license to Genevants LNP technology for up to four neuromuscular indications, including Duchenne muscular dystrophy. Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future development, regulatory and commercial milestones and tiered royalties ranging from the mid-single to low-double digits on future product sales.

As Sarepta works to advance precision genetic medicine across multiple modalities, weve invested in partnering and research efforts focused on improving the utility and benefit of gene-based medicines and providing the greatest possible outcome to patients. This includes advancing our pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat a broad range of neuromuscular diseases, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics.Genevants established leadership and proven LNP technology offers the potential to deliver gene editing machinery to targeted tissue through a non-viral delivery approach. Applying this science to neuromuscular diseases fits squarely within our mission to translate scientific breakthroughs into meaningful advances for patients whose lives have been impacted by rare disease.

Genevant scientists have been at the forefront of LNP delivery of nucleic acids for over 20 years. Our platform is the most clinically validated in the space and is the delivery technology behind the first nucleic acid-LNP product to have achieved FDA approval, said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation. Efficient, optimized delivery is often the difference between successful and unsuccessful nucleic acid drug development, and we are excited to bring our experience to Sareptas gene editing programs in neuromuscular disease where new options and new approaches are desperately needed.

About Genevant SciencesGenevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Companys scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Companys LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. Genevant Sciences is committed to transforming the future of human health. For more information, please visitwww.genevant.com.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties obligations and responsibilities under the agreement, potential payments and fees and Sareptas right to an exclusive license to Genevants LNP technology for up to four neuromuscular indications; the potential benefits of LNPs, including offering a non-viral approach to gene editing that can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues; the potential for gene editing to revolutionize the treatment of diseases caused by genetic mutations including rare neuromuscular diseases by permanently altering genes that lead to disease; the goal of Genevant to design and collaborate with Sarepta in the development of muscle-targeted LNPs that can be applied to gene editing targets in early stage development; and Sareptas goal to advance its pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat a broad range of neuromuscular diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Genevant may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in the discovery of any new therapeutic compounds or any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information by Sarepta

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Contacts:

Sarepta Therapeutics, Inc.Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

Genevant SciencesPete Zorn, pete.zorn@genevant.com

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Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics - GlobeNewswire

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Ixaka (formerly Rexgenero) Launches as an Integrated Cell and Gene Therapy Company – Business Wire

LONDON--(BUSINESS WIRE)--Ixaka Ltd, an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The Companys shareholders have funded the business with over 40 million in financing.

Previously Rexgenero Ltd, a UK-based company pioneering the development of cell therapies to treat serious diseases such as cancer and chronic limb-threatening ischaemia (CLTI), the launch of Ixaka follows integration of its nanoparticle gene therapy business in France and a shareholder restructuring.

The new business will continue to develop Ixakas proprietary technologies concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics. Ixakas technologies enhance the naturally therapeutic power of cells by targeting curative cells at the site of disease, or by directly modifying cells within the body to improve disease targeting and boost their restorative function.

Joe Dupere, CEO of Ixaka, commented: Ixakas broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies. Our focus is now on accelerating progress to help realise the potential for durable and curative cell and gene therapies. By exploring multiple therapies across oncology and cardiovascular, genetic, neurological and autoimmune diseases, we are well positioned to bring life-changing treatments to multiple patient populations with critical unmet needs.

REX-001, Ixakas lead MCT product, is an autologous cell-based product in clinical development for the treatment of CLTI. REX-001 is currently being evaluated in the pivotal Phase III SALAMANDER clinical trial at multiple sites across Europe.

Ixakas polymeric nanoparticle platform can be used to perform genetic modifications directly inside a patients body. The platform enables in vivo targeting and transduction of T cells, and is currently being applied to generate chimeric antigen receptor (CAR) T-cell therapies in vivo for haematological malignancies. Modifications of the components will allow the technology to target a broad range of serious diseases, including cancers and genetic, neurological and autoimmune diseases.

A total of $15.4 billion was raised in the first half of 2020 for the development of cell and gene therapies, with 1,078 regenerative medicine and advanced therapy clinical trials ongoing worldwide1.

References1. https://alliancerm.org/sector-report/h1-2020-report/

ENDS

About Ixaka

Ixaka is a cell and gene therapy company focused on using the natural powers of the body to cure disease.

Ixakas proprietary technologies enhance the naturally therapeutic power of cells by increasing the presence of curative cells at the site of disease, or by directly modifying cells within the body to improve disease targeting and boost their restorative effect.

Ixakas technologies concentrated multi-cell therapies and nanoparticle therapeutics demonstrate potential for the treatment of a broad range of serious diseases across oncology, cardiovascular, neurological and ocular diseases, and genetic disorders.

Ixaka has offices in London, UK with R&D and manufacturing operations in Seville, Spain and Paris, France and additional manufacturing capability in Frankfurt, Germany.

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

Connect with us: Twitter: https://twitter.com/ixaka_Ltd; LinkedIn: https://www.linkedin.com/company/ixaka-limited/

About Ixakas multi-cell therapies

Multi-cell therapies (MCT) are derived from natural tissue extracts which are selected for the most active cells, removing components (such as red blood cells and platelets) that potentially reduce the activity of therapeutic cells. Our first MCT is REX-001, which is currently in a multi-site Phase 3 clinical trial for chronic limb-threatening ischemia (CLTI).

Ixakas REX-001 MCT consists of a combination of progenitor cells and immune cells (lymphocytes, monocytes and granulocytes) which are selected and concentrated from a patients own bone marrow and administered directly to the site of occluded blood vessels in the lower leg. Locally administered REX-001 acts to regenerate blood vessels (through both direct and indirect paracrine mechanisms), modulate immune responses, improve blood flow, improve tissue oxygenation, and promote wound healing. These effects lead to a significant improvement in clinical outcomes and quality of life through complete ulcer healing and alleviation of chronic ischemic rest pain.

About Ixakas in vivo gene delivery technology

Ixakas targeted nanoparticle (TNP) therapeutic is a platform which enables therapeutic cells to be targeted and genetic modifications to be performed directly inside the body. The first application is in the generation of chimeric antigen receptor (CAR) T-cell therapies for haematological malignancies. Modifications of the components however allows the technology to target a broad range of therapeutic cells for the treatment of many serious diseases including cancers, genetic disorders, neurological, autoimmune and ocular diseases.

The TNP in vivo gene delivery approach enables targeting of specific cells and expression of the gene of interest directly in the patient. The technology is also targeted and controllable offering potentially improved efficacy and safety. Generation of enhanced therapeutic cells through genetic modification inside the body also enables more standardized manufacturing which is less expensive as it does not require costly dedicated manufacturing sites needed to expand cells before use (as is required for ex vivo cell therapies).

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Ixaka (formerly Rexgenero) Launches as an Integrated Cell and Gene Therapy Company - Business Wire

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Taysha Gene Therapies and UT Southwestern Medical Center Launch Innovation Fund to Accelerate Advancement of AAV Gene Therapies for Monogenic Diseases…

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, and UT Southwestern Medical Center (UTSW) today announced the launch of an innovation fund to discover and develop novel gene therapy candidates and next-generation technologies for monogenic diseases of the CNS. This expanded partnership will support UTSWs discovery efforts to facilitate the translation of promising discoveries from bench to clinic. Taysha will have an exclusive option on new programs and intellectual property associated with, and arising from, the research conducted under this agreement.

A team of researchers from the gene therapy program at UT Southwestern will explore novel gene therapy targets in new disease areas and create next-generation gene therapy technology platforms to address some of the current limitations of this modality.

We are excited to expand our alliance with UTSW to accelerate the discovery and development of novel gene therapy candidates and next-generation technologies for patients with monogenic CNS diseases, said RA Session II, President, Founder and CEO of Taysha. We believe that the combination of UTSWs translational research expertise in gene therapy and strong track record of innovation and our experience in drug development and GMP manufacturing will create opportunities to reach more patients with unmet medical needs. Our relationship with the UTSW gene therapy program has produced over 18 novel product candidates, including TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1, which are currently in clinical development. We are pleased by the significant progress our partnership has achieved and are excited to build on that foundation and momentum to bring additional compelling innovation to the clinic.

About The University of Texas Southwestern Medical Center

UT Southwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes and includes 23 members of the National Academy of Sciences, 17 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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Taysha Gene Therapies and UT Southwestern Medical Center Launch Innovation Fund to Accelerate Advancement of AAV Gene Therapies for Monogenic Diseases...

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Parent Project Muscular Dystrophy Invests $1 Million in Satellos Bioscience to Support New Regenerative Medicine Technology – PRNewswire

HACKENSACK, N.J., Jan. 19, 2021 /PRNewswire/ --Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced a $1 million programmatic investment in Satellos Bioscience Inc. (Satellos) to support the development of a new regenerative medicine for the therapeutic treatment of Duchenne.

Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the dystrophin gene. In people living with Duchenne, it was discovered by Dr. Michael Rudnicki, the scientific founder of Satellos, that muscle stem cells are severely compromised in their ability to create muscle progenitor cells which repair injured muscle. As a result, people with Duchenne are unable to keep up with the continuous damage to their muscles throughout life.

Satellos's technology is based on this ground-breaking discovery into what controls muscle stem cell differentiation, and the significant impact its dysregulation has on the progressive nature of Duchenne. The company is developing a means to correct this problem through a novel therapeutic approach involving the administration of a small molecule drug, which mobilizes the body's own muscle stem cells, also known as satellite cells, to repair and regenerate muscles.

Such a small molecule drug could be used to treat all people living with Duchenne and furthermore, could be used throughout life to boost the continuous repair and regeneration of skeletal muscle ravaged by this disease, thereby enhancing and extending the lives of people with Duchenne.

"For over 25 years, PPMD has been committed to exploring and supporting every single therapeutic possibility. With this programmatic investment in Satellos, we continue our cutting-edge approach to accelerate finding treatments that have the potential to end Duchenne for every single person impacted by the disease," said Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation. "There is compelling proof of concept data showing that the Satellos technology can improve muscle quality and restore function in the mdxmouse model of Duchenne. This investment from PPMD will enable the Satellos team to build on their proof of concept by amplifying their efforts to invent and refine a drug development candidate suitable for testing in humans."

"We are delighted to be working with PPMD and proud to have the therapeutic potential of our novel science recognized by such a leading entity in the fight against Duchenne", said Frank Gleeson, Satellos founder and CEO. "Our profound discoveries into how muscle stem cells repair and regenerate muscle offers a new avenue for addressing a root cause of the progressive debilitation characteristic of Duchenne. Support from PPMD will enable us to accelerate the development of our new treatment approach which offers the promise of helping Duchenne patients of all ages and stages of disease."

To learn more about PPMD's robust Research Strategy, funding initiatives, and strategies for accelerating drug development,click here.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY:

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength.Parent Project Muscular Dystrophy (PPMD)fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won four FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne atEndDuchenne.org.Follow PPMD onFacebook,Twitter, Instagram, andYouTube.

ABOUT SATELLOS BIOSCIENCE INC.:

Satellos is a regenerative medicine company dedicated to developing novel therapeutics that stimulate or restore muscle regeneration in life threatening disorders. Our founding scientist, Dr. Michael Rudnickidiscovered that the dysregulation of stem-cell polarity a process that balances replenishment of stem cells and production of specialized tissue cells, including muscle can lead to the inability of the body to properly repair and regenerate muscle throughout life.

Satellos is initially applying these discoveries to our lead program to develop a new therapeutic treatment which restores muscle regeneration in Duchenne muscular dystrophy, a lethal degenerative disease. Defects in muscle regeneration are also causative in many chronic conditions which we plan to pursue including sarcopenia (muscle wasting with age), cachexia (muscle wasting from chemotherapy or smoking) and various dystrophies. Founded in 2018, Satellos is headquartered in Canada.For more information about Satellos' discovery platform and development programs please visit Satellos.com.

SOURCE Parent Project Muscular Dystrophy (PPMD)

Join the fight. End Duchenne.

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Parent Project Muscular Dystrophy Invests $1 Million in Satellos Bioscience to Support New Regenerative Medicine Technology - PRNewswire

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Bone Therapeutics, Rigenerand Ink Cell Therapy Deal – Contract Pharma

Bone Therapeutics, a cell therapy company addressing unmet medical needs in orthopedics and other diseases, and Rigenerand SRL, a biotech company that both develops and manufactures medicinal products for cell therapy applications, primarily for regenerative medicine and oncology, have signed an agreement for a process development partnership.Allogeneic mesenchymal stem cell (MSC) therapies are currently being developed at a fast pace and are evaluated in numerous clinical studies covering diverse therapeutic areas such as bone and cartilage conditions, liver, cardiovascular and autoimmune diseases in which MSCs could have a significant positive effect.Advances in process development to scale up these therapies could have major impacts for both their approval and commercial viability. This will be essential to bring these therapies to market to benefit patients as quickly as possible, said Miguel Forte, chief executive officer, Bone Therapeutics. While Bone Therapeutics is driving on its existing clinical development programs, we have signed a first formal agreement with Rigenerand as a fellow MSC-based organization. This will result in both companies sharing extensive expertise in the process development and manufacturing of MSCs and cell and gene therapy medicinal products. Bone Therapeutics also selected Rigenerand to partner with for their additional experience with wider process development of advanced therapy medicinal products (ATMPs), including the conditioning and editing of MSCs.The scope of collaborations between Bone Therapeutics and Rigenerand aims to focus on different aspects of product and process development for Bone Therapeutics expanding therapeutic portfolio. Rigenerand will contribute to improving the processes involved in the development and manufacture of Bone Therapeutics MSC based allogeneic differentiated cell therapy products as they advance towards patients. The first collaboration between the two organizations will initially focus on augmented professional bone-forming cellscells that are differentiated and programmed for a specific task. There is also potential for Bone Therapeutics to broaden its therapeutic targets and explore new mechanisms of action with potential gene modifications for its therapeutic portfolio.In addition to Rigenerands MSC expertise, Bone Therapeutics also selected Rigenerand as a partner for Rigenerands GMP manufacturing facility. This facility, situated in Modena, Italy, has been designed to host a number of types of development processes for ATMPs. These include somatic, tissue engineered and gene therapy processes. These multiple areas of Rigenerand capabilities enable critical development of new processes and implementation of the gene modification of existing processes. In addition, Rigenerand has built considerable experience in cGMP manufacturing of MSC-based medicinal products, including those that are genetically modified.Process development and manufacturing is a key part of the development for ATMPs internationally. Navigating these therapies through the clinical development phase and into the market requires a carefully considered process development pathway, said Massimo Dominici, scientific founder, Rigenerand, professor of medical oncology, and former president of the International Society for Cell & Gene Therapy (ISCT). This pathway needs to be flexible, as both the market and materials of these therapies continues to evolve alongside an improved clinical efficacy.Giorgio Mari, chief executive officer, Rigenerand, said, Rigenerand will offer considerable input from its experience of MSC-based therapies to enable Bone Therapeutics to keep and further accelerate the pace in development of the product processes of its MSC based allogeneic differentiated cell therapy as they advance towards patients. We will continue to use our MSC expertise in the development of Rigenerands own products, as well as in process development and manufacturing cell and gene therapies for partner organizations across the globe.

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Bone Therapeutics, Rigenerand Ink Cell Therapy Deal - Contract Pharma

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