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Jul 17, 2026
Data presented at EHA show that sickle cell disease patients have high interest in gene therapy, with 71% eager to learn more, despite limited awareness and concerns about safety and cost. Early clinical data from approved CRISPR-based therapies demonstrate significant reductions in vaso-occlusive crises and hospital admissions.
Jul 12, 2026
Savara resubmits BLA with near-term FDA decision expected; Sarepta reports $331M in Q1 net product revenue but Elevidys sales continue to decline; AstraZeneca stock falls ahead of July 2026 earnings release.
Jun 29, 2026
All 12 Rett syndrome patients treated with Taysha's TSHA-102 gene therapy in the REVEAL Phase 1/2 trial achieved at least one developmental milestone within one year, with functional gains continuing up to 30 months. The company has completed dosing in the Phase 3 portion and expects top-line data and FDA feedback in early 2027.
Jun 22, 2026
ChemT Biotechnology closed a $4 million seed round led by Wavemaker Ventures to advance its AI-driven biomanufacturing platform. The company's CelMo™ platform demonstrated a 50% increase in antibody output in CHO cells. ChemT plans to expand its technology to stem cells, NK cells, and HEK cells.
Jun 17, 2026
The FDA agreed uniQure's existing trial data could support an accelerated approval filing for Huntington's therapy AMT-130, a reversal from earlier positions. Meanwhile, UK-based Harness Therapeutics selected HRN001 as its lead experimental candidate, targeting the FAN1 protein to address somatic expansion of Huntington's-causing genetic repeats.
Jun 15, 2026
The FDA has granted accelerated approval to Denali Therapeutics' Avlayah (tividenofusp alfa-eknm), the first therapy targeting neurological symptoms of Hunter syndrome. The approval was based on a surrogate endpoint measuring heparan sulfate reduction in cerebrospinal fluid, with confirmatory study results required for full approval. The global Hunter syndrome treatment market is projected to reach $2.6 billion by 2033.
Jun 09, 2026
Biotech stocks Ocugen, Kodiak Sciences, and CytoDyn are experiencing market movements driven by clinical data and regulatory milestones, particularly Biologics License Application (BLA) filings. Ocugen saw a surge in short interest, Kodiak reached a 52-week high on positive trial results, and CytoDyn's stock remains volatile as it advances its monoclonal antibody pipeline.
Jun 08, 2026
Sangamo Therapeutics has retained Raymond James to evaluate strategic alternatives to advance its pipeline and maximize stakeholder value. Key assets include the BLA-ready Fabry disease gene therapy ST-920, the STAC-BBB capsid platform generating $88M in fees to date, and multiple neurology programs. No transaction has been agreed and no timetable has been set.
May 20, 2026
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.
May 20, 2026
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.
May 19, 2026
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.
May 16, 2026
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.
May 14, 2026
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.
May 12, 2026
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.
May 12, 2026
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.
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.
May 05, 2026
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.
May 05, 2026
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.
May 01, 2026
Ray Therapeutics received EMA PRIME designation for its RTx-015 optogenetic gene therapy for retinitis pigmentosa, following recent FDA RMAT status. The company also appointed Michael Murtagh as Chief Regulatory Officer to support global development.
Apr 21, 2026
The FDA said uniQure’s Phase I/II data for Huntington’s disease gene therapy AMT-130 are not adequate for a marketing application. The agency recommended a new randomised, double-blind, sham surgery-controlled study.
