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LATEST NEWS
FDA Proposes One Approval for Many Mutations in Personalized Gene Editing
A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.
2 days ago4 min read
Estuary Launches With $103 Million to Bring Gene Therapy to Polycystic Kidney Disease
Estuary Biotherapeutics launched with $103 million to develop one-time, kidney-directed AAV gene therapies for ADPKD, the most common inherited kidney disease. Here is how restoring polycystin proteins could stop, or even reverse, kidney cysts.
Oct 224 min read
Should Gene Therapy Be Used for Common Diseases? Two Cardiologists Propose Five Questions
One-time genetic treatments are moving from rare diseases to common ones like high cholesterol. In JAMA Internal Medicine, two University of Michigan cardiologists explain why permanence, rare side effects and cost call for a new framework.
Oct 224 min read
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GENE TECH TIMES
The Future Has Arrived
Gene Therapy: A New Era for Diabetes Treatment
The fight against diabetes is being revolutionized with an innovative approach from Genprex, with its gene therapy candidate, GPX-002. Genprex, a gene therapy company focused on developing therapies for cancer and diabetic patients, has announced its plan to launch a new subsidiary, NewCo, which would solely focus on developing and commercializing GPX-002. GPX-002 was designed to treat both Type 1 and Type 2 Diabetes. This gene therapy employs an engineered AAV vector to deli
Sep 4, 2024
FDA Approves BEQVEZ: A Breakthrough One-Time Gene Therapy for Adults with Hemophilia B
Gene therapy is transforming the landscape of treatment options, and the recent FDA approval of BEQVEZ marks a significant milestone for individuals living with hemophilia B. This innovative one-time gene therapy replaces the need for regular infusions of clotting factor, offering new hope for patients suffering from this genetic disorder. Understanding Hemophilia B Hemophilia B is a genetic condition caused by mutations in the human factor IX gene, leading to a deficiency i
May 1, 2024
Lenmeldy: The First FDA-Approved Gene Therapy for Metachromatic Leukodystrophy in Children
Lenmeldy, a groundbreaking gene therapy developed by Orchard Therapeutics, has recently received FDA approval, making it the first and only treatment available for children with Metachromatic Leukodystrophy (MLD), a rare genetic disorder that severely affects the brain. Metachromatic Leukodystrophy is a rare lysosomal storage disorder caused by mutations in the ARSA and/or PSAP genes. These mutations result in the accumulation of sulfatide, leading to the degeneration of esse
Apr 15, 2024
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