Curated news and analysis on clinical trials, drug approvals, and medical research.
Ralph Abraham resigned as CDC principal deputy director after less than two months, leaving the agency's top two positions vacant. The departure is part of broader HHS leadership changes under Secretary Robert F. Kennedy Jr.
The FDA has proposed new guidelines to create a pathway for approving bespoke therapies tested in small patient groups, specifically targeting rare genetic conditions and gene editing treatments that don't fit traditional approval systems.
The FDA accepted a new drug application for tirabrutinib to treat relapsed or refractory primary central nervous system lymphoma, with a decision expected December 18, 2026. The application is supported by phase 2 trial data showing a 67% overall response rate.
The FDA released draft guidance establishing a new approval framework for individualized gene-editing and RNA-based therapies targeting ultra-rare diseases, allowing approvals based on small studies when traditional trials are not feasible.
T-cell immunotherapies including CAR T-cell therapy and T-cell engagers are expanding cancer treatment options but present significant cardiovascular risks and operational complexities requiring coordinated multidisciplinary management.
Rocket Pharmaceuticals will participate in two investor conferences in March 2026, with the CEO taking part in a fireside chat and management hosting investor meetings at both events.
Federal regulators unveiled a new regulatory approach to accelerate approval of personalized genetic treatments for rare, life-threatening conditions, allowing smaller studies when traditional large-scale trials aren't practical while maintaining post-market safety oversight.
ImmunityBio announced $113 million in net product revenue for Anktiva in 2025, a 700% year-over-year increase, while expanding regulatory authorization to 33 countries and securing first approval for lung cancer treatment in Saudi Arabia.
The FDA proposed a new framework to accelerate approvals of personalized treatments for rare genetic diseases, allowing drugmakers to rely on small studies when traditional trials are not feasible while requiring post-approval evidence collection.
The global CRISPR and prime editing market is forecast to grow at 24.1% CAGR through 2031, driven by therapeutic advances and regulatory progress. Scribe Therapeutics achieved a second milestone with Eli Lilly, while new RNA production and gene editing technologies accelerate research.
The FDA has expanded approval of Wakix (pitolisant) to treat cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-controlled substance approved for both pediatric and adult narcolepsy patients with or without cataplexy.
A 24-year-old woman with refractory lupus achieved two successful pregnancies following dual-target CAR-T cell therapy, while bispecific T cell engagers showed efficacy in treatment-refractory antisynthetase syndrome and systemic sclerosis patients.
