Muscular Dystrophy Association

Comments on the agency’s guidance document include requests that it extend the designation to platforms that have yet to yield an approved drug

CBER Director Marks met with biopharma companies, industry-funded advocates while considering gene therapy application

If successful, a clinical trial funded by CureDuchenne, MDA and PPMD could have industry-wide consequences for gene therapy

Pharma extends rare disease franchise, paying well-funded biotech $150M in cash and equity investment to embrace substrate reduction approach

Plus: Keytruda approved for adjuvant NSCLC and updates for AZ’s Evusheld, BMS’s Breyanzi, Magenta, Theonys and more

Plus: Icosavax’s antibody titers disappoint and updates from BMS, Ascendis and more

Patient groups that are an essential part of the life sciences ecosystem are suffering from increased demands and decreased funding

With $107 million in fresh series A cash, gene therapy company AavantiBio will develop a program for Friedreich’s ataxia with leadership joining from Sarepta, which joined the A round.