Cell and Gene Therapy

Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts.

Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.

Among the 55 novel drugs that crossed the regulatory finish line last year were notable new mechanisms of action, coming particularly in the oncology and neurosciences spaces.

In this short teaser, BioSpace’s Head of Insights Lori Ellis talks to CBER Director Peter Marks and Tom Whitehead, Co-Founder of the Emily Whitehead Foundation about anticipated discussions at the upcoming GenScript Biotech Forum.

In a deal expected to close in Q1 2025, Roche will gain access to Poseida’s off-the-shelf CAR T candidates.

By mid-2025, the biotech will split into two entities: a new, as-yet-unnamed innovative medicines specialist and a cell therapy company, the latter of which will inherit the Galapagos name.

BioSpace presents 25 noteworthy biopharma startups in ’25; analysts forecast stronger M&A as the J.P. Morgan Healthcare Conference kicks off next week; GLP-1s continue to expand their reach as Novo, Lilly fight against compounders; and a look ahead to five key FDA decisions in Q1.

From ADCs and radiopharmaceuticals to cell and gene therapies, eager young startups are betting on advances in biopharma’s most competitive therapeutic spaces—and attracting dollars from Big Pharma.

Sangamo is on course to run out of money within months and has now lost access to up to $220 million in milestone payments from Pfizer.

The approval concludes what has been a difficult regulatory path for Ryoncil, which suffered FDA rejections in 2020 and 2023.