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BridgeBio Pharma plans to file for approval in the third quarter. If granted, the oral drug could present a “highly differentiated” option over current achondroplasia therapies, according to Jefferies.
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.
THE LATEST
New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant setbacks after a patient died in a clinical trial for Sarepta’s investigational gene therapy.
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ziclumeran, though analysts at BMO Capital Markets at the time brushed it off as a “non-concern.”
The takeover of its competitor, announced Sunday, could also bring some attention to Dyne Therapeutics, which has a similar RNA-based pipeline in rare muscle diseases.
Lynkuet is the first FDA-approved therapy that blocks both the neurokinin 1 and neurokinin 3 receptors to treat hot flashes.
The BioSpace 40 Under 40 winner opens up about his very personal career transformation from wealth management to biotech—and what it’s like to develop a drug for amyotrophic lateral sclerosis and frontotemporal dementia as a potential patient himself.
Last month, “historic positive results” from uniQure’s gene therapy snapped the Huntington’s community out of years of failure. As the biotech prepares to submit for FDA approval, BioSpace looks at four more candidates on the near horizon.
Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only disappointment in recent years—but one expert worries that communication of the results is creating “false expectations.”
The deal, announced early Sunday afternoon, will see Novartis gain access to Avidity’s neuroscience assets, while the San Diego biotech spins out a new company to shepherd its early-stage precision cardiology programs.
The $70 million upfront deal adds to a portfolio of drugs Biogen has been growing in various immunological conditions since 2024.