News
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
FEATURED STORIES
Dual and even triple or quadruple track processes have come roaring back in 2026 thanks to a glut of M&A that has refilled investors’ wallets. Big Pharma is being put on notice that time is critical if they want to acquire.
Policymaking at FDA has been anything but business as usual under the Trump administration, but former regulators cite the agency’s new investigational new drug pilot program as a sign of normalcy.
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
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Read our takes on the biggest stories happening in the industry.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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This discussion features DEIB leaders from California Life Sciences, MassBio, Eli Lilly and Takeda. We explore changing attitudes in the life sciences workforce, if organizations are adjusting either their DEIB or communication strategies, and how evolving philosophies around DEIB are impacting human resources and talent acquisition activities.
We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system
In a fireside chat at the American Society of Gene & Cell Therapy conference, CBER Director Peter Marks spoke with Takeda’s Kristin Van Goor about how the regulator is approaching the exploding gene therapy space.
While Teva missed Wall Street expectations in the first quarter of 2024, it reported Wednesday a 5% increase in Q1 revenues while reporting that the company’s schizophrenia candidate scored a late-stage trial victory
Regeneron Pharmaceuticals on Wednesday revealed that its investigational gene therapy DB-OTO restored hearing in two young children, according to an oral presentation at the American Society of Gene & Cell Therapy annual meeting.
Amylyx’s recent decision to withdraw its ALS drug Relyvrio from the market highlights an important business decision for companies: when to continue marketing or investigating a drug that has failed a pivotal or confirmatory study.
AstraZeneca announced that it will voluntarily pull Vaxzevria from the global market amid a sharp decline in demand and following the company’s recent admission that its vaccine is linked with a rare side effect.
As interest in psychedelic therapies ramp up, Lykos Therapeutics will go in front of the FDA’s Psychopharmacologic Drugs Advisory Committee on June 4 to present its investigational treatment for post-traumatic stress disorder.
Pfizer’s investigational Duchenne muscular dystrophy gene therapy fordadistrogene movaparvovec has been hit with another patient death, forcing the pharma to pause dosing in its Phase III study.
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.