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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.

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

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.

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.

Expanded sweetener molecule development program aims to broaden access to plant-based sweetening solutions tailored to diverse customer food and beverage categories.

• $5.5 million financing that closed on April 2, 2026 together with $5.7 million in capital raised in Q1-2026, expected to provide Company with cash runway into 2027 • $4.5 million of accrued VAT refunds and R&D refundable tax credits from UK subsidiary begin maturing, including $1.9 million VAT cash refund expected in June 2026 • Successful EMA and Australia TGA (ATGA) meetings aligned around use of existing biomarker and pending release of 3-year overall survival data to support Conditional Marketing Authorisations (CMAs) for OST-HER2 in prevention or delay of recurrence in fully-resected, pulmonary metastatic osteosarcoma (“Metastatic Osteosarcoma”) • 2.5-year overall survival data expected to be released during ASCO 2026 • EMA and ATGA have aligned on key design aspects of confirmatory Phase 3 Metastatic Osteosarcoma protocol required to have commenced prior to grant of CMAs or FDA BLA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint • Upcoming U.S. FDA Type C meeting and U.K. MHRA SAM meeting to align with EMA & ATGA prior to Australia Clinical Trial Notification submission for Phase 3, including alignment on 3-year overall survival as a primary clinical efficacy endpoint. U.S. FDA (“FDA”) Pre-BLA meeting to confirm alignment on surrogate and primary clinical efficacy endpoints with EMA/ATGA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint, and seek decisions on rolling review acceptance and RMAT designation following the EMA and U.K. MHRA granting ATMP designation (EMA/MHRA RMAT equivalent) for OST-HER2 in Metastatic Osteosarcoma • OS Animal Health S-1 filing for proposed ‘Go-Public’ transaction and receipt of SEC comments