The FDA fast-tracked an inhalable gene therapy for advanced lung cancer after an early 11-patient trial showed tumour shrinkage in three patients and disease stabilization in five. Larger studies involving about 250 patients are underway.
Two Louisiana parents published a children’s book to raise awareness of Infantile Neuroaxonal Dystrophy, a rare disorder affecting two of their daughters. They are also helping raise money for a gene therapy trial.
Sensorion appointed Fred Chereau chief executive effective June 1, 2026, while reporting six-month Audiogene trial data for SENS-501. The company said early hearing improvements in cohort 2 persisted at six months and no serious adverse events were reported.
Benitec Biopharma's gene therapy BB-301 improved swallowing ability in all four OPMD patients followed for at least one year in a Phase Ib/II trial. One patient showed continued improvement at two years. The company plans to meet with the FDA in mid-2026 to discuss a pivotal study.
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.
Phase 1 data in 17 adults with Friedreich's ataxia cardiomyopathy showed AAVrh.10hFXN (LX2006) was generally well tolerated and showed signs of biological activity. The gene therapy is being advanced into a larger trial.
Recent studies in retinitis pigmentosa showed mixed results for gene and optogenetic therapies. UGX-201 and MCO-010 were associated with visual acuity improvements, while AAV8.hPDE6A showed no significant functional gains at 1 year.
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.
India is expanding rare disease support under the 2021 policy, with aid up to Rs 50 lakh and 15 Centres of Excellence. Officials also called for local innovation as treatment costs can reach ₹16 crore.
The United Arab Emirates has unveiled two gene therapy projects targeting autism, epilepsy, and rare brain disorders in children. The initiatives focus on underlying genetic causes and earlier diagnosis.
