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NEW BOOK
LATEST NEWS
Capricor's Shares Drop After FDA Rejects Deramiocel Cell Therapy for Heart Disease in DMD
Capricor Therapeutics faced a significant setback on July 11, 2025, when the FDA rejected its cell therapy, Deramiocel , for treating...
2 days ago
FDA Rejects Ultragenyx's Gene Therapy for Rare Disease - Sanfilippo Syndrome
The FDA has declined to approve Ultragenyx Pharmaceutical's gene therapy, UX111, for treating Sanfilippo syndrome type A, a rare genetic...
2 days ago
Biogen's Salanersen Shows Promise for SMA Patients Post-Gene Therapy
Biogen’s experimental drug, Salanersen, is showing promising potential for treating children with spinal muscular atrophy (SMA), even...
Jul 1
GENE TECH TIMES
The Future Has Arrived
Light-Induced Gene Therapy Using Nanoparticles Targets and Destroys Cancer Cells' Mitochondria
A groundbreaking new approach in cancer treatment has emerged, utilizing light to disable the mitochondria, the energy-producing...
Feb 9
Gene Therapy Restores Enzyme Levels to Treat Hypophosphatasia - A More Permanent Treatment
Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones...
Feb 4
Gene and Cell Therapy Clinical Trials to Notice in 2025
Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below...
Jan 31
Five-Year Follow-Up Shows Sustained Visual Improvements in LHON Patients Treated with AAV Gene Therapy
A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide...
Dec 27, 2024
Gene Therapy Successfully Reverses Heart Failure in Minipig Study: A Big Step in Heart Failure Treatment
Heart failure, which affects over 64 million people worldwide, is currently considered irreversible without a heart transplant....
Dec 11, 2024
Serious Immune Reaction in a Young Patient Challenges Rett Syndrome Clinical Trial
A clinical trial for NGN-401, a gene therapy designed to treat Rett syndrome, recently faced a setback when a young participant...
Nov 22, 2024
Positive Phase 2 Results for Gene Therapy in Parkinson’s Disease
In exciting news for Parkinson’s disease research, MeiraGTx has announced positive results from a Phase 2 clinical bridging study of...
Oct 18, 2024
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....
Sep 4, 2024
New Gene Therapy Breakthrough Paves Way for Better Treatment of Brain Disorders
In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke...
Aug 20, 2024
FDA Expands Approval of ELEVIDYS: A Breakthrough Gene Therapy for Duchenne Muscular Dystrophy
On June 20th, 2024, the FDA expanded its approval of ELEVIDYS , an innovative AAV-based gene therapy developed by Sarepta Therapeutics ,...
Jun 20, 2024
AAV-Based Gene Therapy Clinical Trial Offers Hope for Children with Congenital Deafness
The AK-OTOF-101 clinical trial by Akouos, Inc., a subsidiary of Lilly & Co., brings renewed hope to children suffering from congenital...
Mar 30, 2024
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