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The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
The FDA has introduced models intended to accelerate rare disease drug development, but recent reversals of guidance from the agency speak to a lack of clarity in its implementation. AI can help focus this process.
Just a few days after FDA Commissioner Makary resigned, ally Tracy Beth Høeg is also leaving the agency. Her departure comes amid reports of tension over a commissioner’s voucher for Sanofi’s diabetes drug.
Follow along as BioSpace tracks job cuts and restructuring initiatives.
California life sciences jobs declined 1.8% last year, according to the new California Life Sciences sector report. While National Institutes of Health funding and venture capital investment rose, their growth slowed from the previous year.
Rina-S is the last candidate standing from Genmab’s $1.8 billion ProfoundBio acquisition two years ago, with the Danish drugmaker ending development of another clinical program stemming from the buyout.
A batch of a chemotherapy product made at a Sun facility with a history of quality and compliance issues is being withdrawn from the U.S. market.
If Biogen has shown that tau can impact cognition, Denali’s technology—validated with an FDA approval in Hunter syndrome—could ensure the medicine gets where it needs to be for the greatest therapeutic impact, analysts said.
Aardvark Therapeutics had previously voluntarily suspended studies of ARD-101—and a related asset called ARD-201—after detecting anomalous echocardiographic readings in healthy volunteers that could indicate reduced heart efficiency.
CREATE Medicines is working on a clinical-stage pipeline for cancer, while its autoimmune programs are still in preclinical testing.
Renewed pharma interest in GPCR biology and radioligand therapies is drawing attention to functional peptide screening platforms.
Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.
PRESS RELEASES
Registration link below for Wednesday March 18, 2026 at 4 PM EDT Phio invites individual and institutional investors, as well as advisors and analysts with an interest in the life sciences sector, to attend this virtual event
Collaboration validates integrated RNA, protein, and morphology workflows and expands access to next generation optical pooled screening on the AVITI24™