StemCell Therapy

Newswise CLEVELANDResearchers at Case Western Reserve University have used a unique method to safely deliver gene therapy to fight a rare, but irreversible, genetic eye disorder known as Stargardt disease. By using chemically modified lipidsinstead of the viruses most commonly used as carriersStargardt disease did not return in animal models for up to eight months after treatment, said lead researcher Zheng-Rong Lu, the M. Frank Rudy and MargaretDomiter Rudy Professor ofBiomedical Engineering at the Case School of Engineering, with a dual appointment at the School of Medicine.

A health ministry panel approved Wednesday the production and sale of a gene therapy for treating spinal muscular atrophy, a rare incurable disease that destroys muscle function through a decrease in motor neuron cells. The health minister is expected to formally approve the drug, Zolgensma, by the end of March, with public health insurance coverage seen starting this summer. The one-time-only gene therapy, developed by Swiss drug giant Novartis AG, will cover patients below 2 years old with abnormalities in their genes to make proteins necessary for maintaining motor nerves.

For the cell and gene therapy revolution to be fullyrealized, physicians, research scientists, biomanufacturing experts, advocacygroups, regulatory bodies like the Food & Drug Administration (FDA) andother key stakeholders have to Think Different, as Apple famously encouraged.

TrakCel and Ori Biotech have announced a new non-exclusive strategic partnership to "achieve supply chain orchestration within closed system manufacturing for the cell and gene therapy sector. The Ori platform enables therapy developers and contract manufacturers to achieve automated CGT manufacturing in a closed platform, and so the collaboration aims to enable users of the Ori Biotech manufacturing platform to benefit from live supply chain visibility through TrakCel software

The excitement about cell and gene therapies is almost tangible within the biotech and pharma industry. Over 950 companies are actively developing advanced therapies, which are expected to make exceptional improvements to peoples lives in the next decade. Although hopes are high, the industry still faces a number of challenges in cell and gene therapy manufacturing, mainly around being able to deliver these often difficult to make, complex treatments at the scale needed to meet patient demand.

Biomarin(BMRN) just recently announced that the FDA has accepted for Priority Review the BLA for ValRox (valoctocogene roxaparvovec) for adults with hemophilia A, notes biotech expertJohn McCamant, editor ofThe Medical Technology Stock Letter. This constructive action by the FDA marks the first marketing application accepted for a gene therapy (GT) product for any type of hemophilia in the United States.The PDUFA date is August 21, 2020. More from John McCamant: Alkermes Targets Bipolar Disorder and Schizophrenia For the past 2+ years, we have known that the FDA has been proactive with gene therapy companies with an emphasis on hemophilia

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BioMarins hemophilia A drug has been set an Aug. 21 PDUFA date as the California biotech looks to gain a speedy approval for the first-ever gene therapy for the bleeding disease

Supplies of Zolgensma, the gene therapy approved last year for spinal muscular atrophy, are tight.

For most parents, their child's second birthday is one of the many exciting milestones of toddlerhood - a chance to share a toast with friends and family and look forward to a future filled with possibilities. For Queensland parents Kellee and Jamie Clarkson, it's a date that fills them with dread. Their 18-month-old daughter Wynter has spinal muscular atrophy (SMA), a degenerative genetic disorder that attacks the motor neurons in her spine, progressively weakening her muscles and shutting down movement.

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all.

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all.

BOSTON and LONDON, Feb. 27, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global leader in gene therapy, today reported business highlights and financial results for the year ended December 31, 2019, as well as 2020 strategic priorities and upcoming milestones.

PARIS--(BUSINESS WIRE)--Regulatory News: Lysogene (Paris:LYS) (FR0013233475 LYS), a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). LYS-SAF302, a second-generation gene therapy, is designed to deliver a functional copy of the SGSH (N-sulfoglucosamine sulfohydrolase) gene to the brain through a one-time direct-to-CNS administration, and is being investigated in the international Phase 2/3 clinical trial AAVance (NCT03612869)

Two Emirati sisters were among the first patients worldwide to receive a new gene therapy to treat patients with a rare inherited eye disorder that causes blindness. The procedure, which injects a lab-created virus that helps the eye produce necessary proteins, has been recently introduced to Cleveland Clinic Abu Dhabi. The sisters Alia, 13, and Hessa, 11, are affected by retinal dystrophy - a rare genetic disorder that affects their ability to see, especially in dim light.

Alongside resolutions, the new year tends to come with biopharma acquisitions.

Image source: The Motley Fool.

DUBLIN--(BUSINESS WIRE)--The "2nd Annual Cell Therapy Bioprocessing Conference" conference has been added to ResearchAndMarkets.com's offering. Over the last decade, the field of cell therapy has rapidly grown, and it holds enormous promise for treating many diseases.

BY GRANT LANCASTER When 9-year-old Zach Becker rushes around his living room in Brooklyn, he is just as care-free as any kid his age, decked out in a Batman costume complete with a cape that swooshes behind him as he charges over to pester his 11-year-old brother Ben. His spirits are completely unaffected by the rare disease that he has, one that has likely already shortened his life. Zach has Fanconi anemia, an illness affecting 1 in 160,000 people worldwide that keeps his bone marrow from making blood cells properly, according to the National Institutes of Health.

Critical considerations in ensuring patient access to cell and gene therapies, and continued industry growth, include proper understanding of how to manage source material quality and supply chain continuity. The following areas will be covered: The importance of defining source material requirements for translation of allogeneic advanced therapies The value of a reliable and recallable donor network for consistent source material access The critical requirements for obtaining GMP-compliant source materials for commercial manufacture Best practice in handling, monitoring and delivering temperature-sensitive source materials The importance of early planning, coordination and communication with all stakeholders The webinar will take place on 12 March 2020 at 9:00 PDT / 12:00 EDT / 16:00 GMT / 17:00 CET