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Archive for the ‘Gene therapy’ Category

Scientists successfully test new way to deliver gene therapy – Newswise

Thursday, February 27th, 2020

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.

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Japanese government panel OKs expensive gene therapy for rare disease – The Japan Times

Thursday, February 27th, 2020

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.

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This Startup is on a Mission to Decentralize Cell and Gene Therapy Clinical Trials – BioBuzz

Thursday, February 27th, 2020

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.

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TrakCel, Ori Biotech ink supply chain deal for cell and gene therapy – PharmaTimes

Thursday, February 27th, 2020

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

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Tackling the Challenges in Cell and Gene Therapy… – Labiotech.eu

Thursday, February 27th, 2020

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.

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Biomarin- Gene Therapy and Hemophilia – Yahoo Finance

Thursday, February 27th, 2020

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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Roche confirms departure of gene therapy pioneer Kathy High – STAT

Thursday, February 27th, 2020

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free! STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis.Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

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BioMarin gene therapy won’t need an AdComm as it nabs speedy FDA review – FierceBiotech

Thursday, February 27th, 2020

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

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With supply tight, Novartis readies gene therapy plant for production – BioPharma Dive

Thursday, February 27th, 2020

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

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Parents’ fight to access life-changing $3.2m gene therapy for their daughter – 9News

Thursday, February 27th, 2020

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.

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‘The headlines are the headlines, but’: BioMarin talks up potential savings as hemophilia gene therapy launch looms – Endpoints News

Thursday, February 27th, 2020

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.

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Biogen touts new evidence from the gene therapy company it wagered $800M on – Endpoints News

Thursday, February 27th, 2020

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.

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Orchard Therapeutics Reports 2019 Financial Results and Reviews Key Strategic Priorities for 2020 – BioSpace

Thursday, February 27th, 2020

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.

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Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA – Business Wire

Thursday, February 27th, 2020

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)

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Abu Dhabi hospital uses gene therapy to save Emirati sisters from blindness – The National

Thursday, February 27th, 2020

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.

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Pharmacquired: Where are all the deals? – BioPharma Dive

Thursday, February 27th, 2020

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

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Orchard Therapeutics (ORTX) Q4 2019 Earnings Call Transcript – Motley Fool

Thursday, February 27th, 2020

Image source: The Motley Fool.

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2020 Cell Therapy Bioprocessing Conference (Boston, MA, United States – June 25-26, 2020) – ResearchAndMarkets.com – Business Wire

Thursday, February 27th, 2020

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.

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Brooklyn boy with rare blood disease to speak at awareness event on Feb. 29 – amNY

Thursday, February 27th, 2020

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.

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Biocair participates in cell and gene webinar alongside HemaCare and Beam Therapeutics – Cambridge Network

Thursday, February 27th, 2020

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

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