A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Wafik S. El-Deiry highlighted p53 research and the August 2025 approval of dordaviprone/modeyso, the first treatment for diffuse gliomas with H3K27M mutations. Separate posts also described glioblastoma precision oncology discussions and a lifetime achievement award in Mumbai.
New AI-powered platforms enable same-day cancer treatment decisions and streamline clinical trial matching. University of Utah's μPharma chip predicts drug responses in under four hours, while City of Hope's HopeLLM system matches patients to trials across its national network.
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
Recent publications and collaborations advance glioblastoma research through blood-based microRNA diagnostics, combination immunotherapy trials, and identification of MOV10 as a prognostic biomarker and therapeutic target.
Five intra-articular gene therapy platforms for knee osteoarthritis are in human trials in the US, while India prepares regulatory guidelines for mRNA, gene and cell therapies to accelerate innovation.
The FDA has proposed a new "plausible mechanism" pathway to approve customized treatments for rare diseases after testing in only a handful of patients, aiming to remove barriers for gene editing and other bespoke therapies that don't fit traditional approval systems.
The FDA approved acalabrutinib combined with venetoclax for chronic lymphocytic leukemia and small lymphocytic leukemia based on phase III trial results. Separately, Yale researchers identified CD25 protein complex as a new drug target for aggressive leukemias.
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.
Researchers have developed modified CAR T cells that overcome prostaglandin E2 suppression in solid tumors and a selective therapy targeting IGHV4-34 that spares healthy immune cells, addressing key limitations in current treatments.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07602270 |
Effects of Ketogenic Diet on Alcohol Intoxication |
RECRUITING | NA |
| NCT07594067 |
TCR1188-ABC Cells in KRAS-mutated Cancers |
NOT_YET_RECRUITING | PHASE1 |
| NCT07593456 |
NST-SPARK Open-Label Phase 1 STTR (IGNITE) |
NOT_YET_RECRUITING | NA |
| NCT07581639 |
Comparison of Live and Virtual Music-based Interventions in Mitigating Stress |
COMPLETED | NA |
| NCT07568080 |
SnapChole: An International, Time-Bound Prospective Platform Study of Management Strategies and Outcomes in Acute Calculous Cholecystitis |
NOT_YET_RECRUITING | |
| NCT07565207 |
Effect of aiTBS on Intrinsic Spectral Dynamics and Task Performance |
NOT_YET_RECRUITING | NA |
| NCT07565194 |
Arousal-related Memory Following Theta Burst Stimulation. |
NOT_YET_RECRUITING | NA |
| NCT07564193 |
The Coach Mpilo Study: Evaluation of a Peer-led Intervention to Promote Engagement in HIV Care for Men Living With HIV |
NOT_YET_RECRUITING | NA |
| NCT07561307 |
Optimizing Stimulation Parameters for Electroconvulsive Therapy |
NOT_YET_RECRUITING | NA |
| NCT07558148 |
OCD LIFU Target Engagement |
RECRUITING | NA |
