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Algae proteins partially restore blind man s vision, study says

Algae proteins partially restore blind man’s vision, study says By Catherine Park FILE - A doctor examines the eye of a patient. Researchers believe algae could be the solution to fixing blindness after one patient who received an experimental treatment had his vision partially restored, according to a recent study.  Optogenetics may enable mutation-independent, circuit-specific restoration of neuronal function in neurological diseases, according to the study, published in Nature Medicine this month.  For the study, doctors were treating a patient with retinitis pigmentosa, which impacts more than two million people worldwide, the study’s authors said.  Retinitis pigmentosa is a neurodegenerative eye disease where loss of photoreceptors can lead to complete blindness, the study said. 

AskBio Announces IND for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Cleared to Proceed by U S FDA

Share this article Share this article RESEARCH TRIANGLE PARK, N.C., May 25, 2021 /PRNewswire/  Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced that the U.S. Food & Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LION-101 to proceed in a Phase 1/2 clinical study. LION-101 is a novel recombinant adeno-associated virus (rAAV) based vector being developed as a one-time intravenous infusion for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). LION-101 will be evaluated in a Phase 1/2 multicenter study to evaluate a single intravenous (IV) infusion in adult and adolescent subjects with genotypically confirmed LGMD2I/R9. AskBio plans to initiate dosing for the LION-101 Phase 1/2 clinical study in the first half of 2022.

Children s Hospital of Philadelphia President and CEO Releases Special Podcast Series

Children s Hospital of Philadelphia President and CEO Releases Special Podcast Series - Breaking Through with Madeline Bell s Pioneered at CHOP series highlights stories about some of CHOP s amazing breakthroughs, told by the people who made them - News provided by Share this article Share this article PHILADELPHIA, May 5, 2021 /PRNewswire/  Doctors and scientists at Children s Hospital of Philadelphia (CHOP) are making breakthroughs that are transforming children s lives – and changing the world. In Pioneered at CHOP, a five-episode special series of her popular podcast Breaking Through with Madeline Bell, CHOP President and CEO Madeline Bell speaks with five CHOP doctors and scientists about the stories behind some of their groundbreaking work.

Chutes & Ladders—GSK vaccine exec Reichman splits to take biotech CEO post

(Pixabay)                  Welcome to this week s Chutes & Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good word or the bad from your shop to Fraiser Kansteiner, and we will feature it here at the end of each week. Another GSK vaccine exec jumps ship for biotech CEO role GlaxoSmithKline Amir Reichman is stepping down. Reichman is leaving his post as head of global vaccines engineering core technology at GSK to eventually head up operations at BiondVax Pharmaceuticals. Reichman will split duties with BiondVax’s founder and CEO Ron Babecoff until March 2 as he wraps his time at GSK. Reichman joined GSK in 2015 as part of a multibillion-dollar deal that saw GSK trade off its oncology assets for Novartis’ vaccines unit. Reichman held various positions at Novartis in its vaccine supply chain unit before becoming director of GSK’s global vaccines supply chain and then head of global vaccines engineering

How 2 scientific pioneers teamed up to run AskBio, Bayer s new gene therapy division

Bayer AG Published Jan. 21, 2021 Share it Large pharmaceutical companies have made gene therapy a priority with a series of acquisitions over the past several years, a stamp of validation for a field that s pushed through decades of ups and downs. One of the latest buyers is German healthcare conglomerate Bayer, which in October inked a $2 billion deal for North Carolina gene therapy developer Asklepios Biopharmaceuticals, also known as AskBio. For Bayer, the acquisition is part of a broader effort to build a gene and cell therapy division. But the deal is just as noteworthy for AskBio, an unusually large, privately held biotech based on the work of one of gene therapy s pioneers, Jude Samulski.

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