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AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.

Analysts at financial firm Cantor Fitzgerald are urging President Donald Trump to rethink his appointment of Robert F. Kennedy Jr. as Secretary of Health and Human Services.

The layoffs will take place throughout 2025 and will mostly affect Tenaya’s research and manufacturing operations. The company is continuing to test its hypertrophic cardiomyopathy gene therapy.

Despite a $400 million impairment charge, analysts say the removal of a drug-device combo from its portfolio is not a huge loss for Vertex given that the company has a more advanced type 1 diabetes candidate in zimislecel.

The stock market—and biotech insiders—reacted negatively to the allegedly forced resignation of CBER Director Peter Marks, who said RFK Jr. does not seek “truth and transparency” but rather “subservient confirmation of his misinformation and lies.”

Unlike other hemophilia therapies, Qfitlia is indicated for both hemophilia A and B, and can be given in patients regardless of the presence of neutralizing antibodies against clotting factor VIII or IX.

BMO Capital Markets analysts said the results potentially position lepodisiran as “one of the most durable assets in development to date” in the competitive Lp(a) space, where drugs are designed to lower the risk of cardiovascular events such as heart attack and stroke.

Ayrmid’s offer is 50% higher than bluebird’s previously announced deal with Carlyle and SK Capital Partners.

According to BMO Capital Markets, Rybelsus’ outcomes in SOUL were “inconsistent,” failing to significantly lower cardiovascular death and nonfatal stroke.

Industry updates and investment insights—including investor behavior and fundraising advice—were among the topics experts explored at Charles River Laboratories’ 2025 Cell & Gene Therapy Summit.

AI offers tremendous potential but there are critical and time-consuming flaws in black box AI predictions.

Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.

66.6% of OST-HER2 treated patients achieved 2-year overall survival compared with 40% in the historical control group (p = 0.0046) FDA issues Biologics Licensing Application (BLA) number for OST-HER2 in preparation for anticipated BLA filing for the prevention or delay of recurrent, fully resected, pulmonary metastatic osteosarcoma following August 27, 2025 End of Phase 2 Meeting Company responds to FDA correspondences seeking to align approval metrics for Regenerative Medicine Advanced Therapy (RMAT) designation, Breakthrough Therapy designation (BTD) and Biologics Licensing Application via Accelerated Approval Program Company’s NYSE American listing included in the Russell Microcap, Russell Microcap Value and Russell Microcap Growth indexes

MHRA seeks to harmonize UK & US regulatory review via Project Orbis EMA Rapporteur Scientific Advice Meeting scheduled for October 2025

Investment accelerates Maxwell’s path toward FDA trials and global market readiness