GlobalData on MSN
Apertura and NIH institutes to test gene therapy for NPC1
The Ara Parseghian Medical Research Fund will provide financial backing for the multi-year research effort.
Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today ...
Completion of enrollment anticipated within 12 monthsGALILEO-3 is intended to support the registration of FLT201 in the adult GD1 ...
A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.
A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered ...
A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious and common complication of the condition, has shown promising results in a ...
Morning Overview on MSN
The FDA approved the first gene therapy for sickle cell in children as young as two
Regulators have opened a one-time gene therapy to some of the youngest patients with sickle cell disease. On July 1, 2026, ...
AUSTIN, Texas, April 28, 2026 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for ...
Fractyl Health, Inc. announced promising preclinical results for its gene therapy RJVA-001 at the 2025 ASGCT Annual Meeting, showing that a single dose leads to substantial metabolic improvements ...
Proprietary dual-vector platform delivers full-length ABCA4, enabled by Atsena's laterally spreading AAV.SPR capsid for delivery to the central ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
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