Massachusetts
Poor efficacy data for two early-stage candidates for the rare disease alpha-1 antitrypsin deficiency have convinced Vertex Pharmaceuticals to terminate their development.
Moderna’s stock price plunged more than 20% in Thursday morning trading after the company lowered its 2024 revenue guidance due to weak demand for its coronavirus vaccine in the second quarter.
With an upfront $800 million payment and $325 million in potential milestones, Otsuka Pharmaceutical is acquiring Jnana Therapeutics’ drug discovery platform and rare disease candidate, the companies announced Thursday.
Sarepta has been hit with another patent infringement lawsuit, this time from Sanofi and its subsidiary Genzyme alleging that the biotech used protected technology related to AAV vectors.
The FDA will have six months to review Vertex Pharmaceuticals’ suzetrigine, potentially the first new class of drugs for acute pain in more than 20 years, according to the company.
The advantages of using circular RNAs—including increased durability, enhanced protein expression and substantially lower manufacturing costs compared to linear mRNAs—have driven a spate of investment in this technology.
The FDA has four big events in the coming two weeks, including an advisory panel meeting for an ultra-rare disease.
Biogen and Sage Therapeutics’ investigational neuroactive steroid did not significantly improve upper limb tremors in patients with essential tremor, the companies announced Wednesday.
Allarity Therapeutics announced Monday that the Securities and Exchange Commission has made a preliminary determination recommending an enforcement action against the company for alleged violations of federal securities laws.
The Department of Health and Human Services’ Office of the Inspector General found that bluebird bio’s fertility support program for its gene therapies could potentially violate federal anti-kickback statutes.
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