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The hold was placed earlier this year when the FDA asked for more preclinical data, but the agency was slow to respond due to ‘strain’ on its capacity, according to Neurizon.

AstraZeneca has invested heavily in AI, primarily through collaborations, including an up to $5.3 billion partnership with China’s CSPC Pharmaceutical in June.

This latest FDA program aims to provide speedier reviews for generic drugmakers who produce their products in the U.S.

Rocket Pharmaceuticals’ strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia.

The centerpiece of the collaboration is the gene editor ABO-101, being developed for primary hyperoxaluria type 1, a rare disease that leads to severe kidney stones.

As industry leaders gather at the annual event in Phoenix, the cell and gene therapy space remains in a state of flux, with M&A activity and regulatory support signaling momentum while commercialization challenges continue to hinder broader investor interest.

Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.

While Bruton’s tyrosine kinase inhibitors are often hailed as the next big breakthrough in multiple sclerosis, Immunic Therapeutics and others are leveraging neuroprotective targets and remyelination to keep the disease at bay.

Jeanne Marrazzo, former director of the National Institutes of Allergy and Infectious Disease, was formally terminated Thursday after months on administrative leave, after filing a whistleblower report.

Expanded exemptions for orphan drugs could mean prolonged protections for top-selling drugs like Merck’s Keytruda, which was initially approved under this designation in 2014.

Findings from the Phase III VESALIUS-CV study reinforce the cholesterol-lowering benefit of Repatha in high-risk patients without prior cardiovascular disease, for which the drug was approved in August 2024.

Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.

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