Taysha Drops Gene Therapy Candidate as FDA Again Calls for Study Changes
Pictured: FDA sign in its headquarters/iStock, GrandbrothersTaysha Gene Therapies has decided to drop its lead experimental AAV-based gene therapy candidate after the FDA reiterated calls for the company to put the treatment through a randomized, double-blind...
FDA Accepts Orchard’s BLA for Rare Disease Gene Therapy Behind EU Approval
Pictured: Gene therapy illustration showing an injection targeting a specific DNA site/iStock, Ilya Lukichev The FDA on Monday accepted Orchard Therapeutics’ Biologics License Application for OTL-200, an investigational gene therapy for rare disease metachromatic...
Excision Biotherapeutics’s pre-clinical studies testing dual-guide CRISPR strategies for viral excision as part of the company’s efforts to develop a cure for HIV
Dr. Nadia Amrani, a scientist at Excision Biotherapeutics conducts pre-clinical tissue culture studies testing Excision's dual-guide CRISPR strategies for viral excision as part of the company's efforts to develop a cure for HIV. (Photo Credit: Dawn Wessman...
Moderna, Immatics Ink Potential $1.8B Oncology Deal including Cancer Vaccines
Pictured: Moderna sign on building in Cambridge/iStock, hapabapaVaccine giant Moderna and oncology biotech Immatics have inked a research partnership worth a little more than $1.8 billion in potential payments to the latter. The collaboration will combine Moderna’s...
A modified mRNA aids heart attack recovery in mouse and pig models
Jianyi "Jay" Zhang, M.D., Ph.D. Credit: UABSevere heart attacks often progress to end-stage heart failure because the dearth of heart muscle cells diminishes pumping power of the heart's left ventricle. The damaged heart, unable to repair itself, deteriorates and...
2seventy to Lay Off 40% of Workforce, CEO to Depart in Reorganization
Pictured: Illustration depicting large layoffs/iStock, Andrii Yalanskyi 2seventy bio on Tuesday kicked off a sweeping strategic restructuring initiative aimed at streamlining its business operations and R&D model—including advancing fewer pipeline programs—to generate...
Despite FDA doubts, Alnylam wins expert backing for Onpattro expansion into rare heart disease
While FDA panelists supported Alnylam Pharmaceuticals' medicine in transthyretin amyloidosis cardiomyopathy, they recommended a limited FDA approval that would severely restrict the med's addressable population. (FDA)After Alnylam Pharmaceuticals faced FDA doubts...
New Gene Therapy Drug for Inherited Retinal Dystrophy Released in Japan
In a significant development for patients with inherited retinal dystrophy, the gene therapy drug “Luxturna” by Novartis Pharma has been released in Japan. While the drug comes with a high price tag of approximately 100 million yen for both eyes, its potential to...
Engineers design more powerful RNA vaccines
High-throughput synthesis and screening of biodegradable cyclic ionizable lipids synthesized by Ugi-3CR. a, General reaction scheme of Ugi-3CR (top); structures of the three components of the ionizable lipids (cyclic amine head group, isocyanide/isocyano acetate...
Rocket Pharmaceuticals and FDA Converge on Phase 2 Gene Therapy Trial for Danon Disease, Fueling Investor Optimism
New Jersey's Rocket Pharmaceuticals has successfully aligned with the FDA on the design of a pivotal Phase 2 trial for a rare disease, potentially hastening the approval of their gene therapy. This open-label study will enlist 12 patients for treatment with RP-A501,...
North America’s Youngest Infant Receives Pioneering SMA Gene Therapy Treatment
The Montreal Children's Hospital has reported a significant breakthrough: a nine-day-old baby named Samuel has become the youngest patient in North America to receive gene therapy for a rare, degenerative disease called spinal muscular atrophy (SMA). SMA affects nerve...
Novartis Drops Eye Disease Gene Therapy from $800M Gyroscope Acquisition
Pictured: Novartis campus in Switzerland/iStock, Michael Derrer Fuchs Novartis is discontinuing the development of GT005, a mid-stage investigational gene therapy being developed for geographic atrophy secondary to dry age-related macular degeneration. The...
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