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May 10, 2026
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
Apr 23, 2026
Nature Communications findings showed NR2F6 deletion revived exhausted CAR-T cells and boosted activity against solid tumors. In murine models, modified cells drove tumor regression, prolonged survival and resistance to rechallenge.
Mar 21, 2026
The FDA has issued draft guidance creating a "Plausible Mechanism Framework" to accelerate approval of individualized therapies for ultra-rare diseases where traditional clinical trials are not feasible. The guidance focuses on gene editing and RNA-based treatments targeting specific genetic abnormalities.
Mar 12, 2026
Researchers discovered that adding three common amino acids to lipid nanoparticles dramatically improves mRNA and CRISPR gene editing delivery, boosting efficiency up to 20-fold and raising gene editing success rates from 25% to nearly 90%.
Mar 11, 2026
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.
Mar 04, 2026
Ocugen will host a conference call on March 4, 2026 at 8:30 a.m. ET to discuss fourth quarter and full year 2025 financial results and provide a business update, with a pre-market earnings announcement the same day.
Mar 02, 2026
The pharmaceutical industry is experiencing recovery driven by innovation in drug development, aggressive M&A activity, and expansion of drug development services. The sector faces both opportunities and headwinds including pipeline setbacks and regulatory pressures.
Feb 26, 2026
Three new market reports project significant expansion in pharmaceutical R&D outsourcing, the U.S. pharmaceutical market, and biotechnology services outsourcing, driven by innovation, aging populations, and cost efficiency needs through 2035.
Feb 26, 2026
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.
Feb 24, 2026
The FDA issued draft guidance establishing a plausible mechanism framework for approving individualized therapies targeting ultra-rare diseases when randomized controlled trials are not feasible due to small patient populations.
Feb 24, 2026
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.
Feb 24, 2026
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.
