FDA Addresses Zolgensma Gene Therapy Data Manipulation
The agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.
Gene Therapy for CLN6 Batten Disease Shows Positive Interim Results
Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.
PMZ-1620 Enters Phase 3 Trial As a Potential Treatment For Cerebral Ischemic Stroke
The investigational therapy has the potential to be a first-in-class neuronal progenitor cell therapeutic with anti-apoptotic activity that improves cerebral blood flow and neurological outcome in stroke.
Parkinson Disease Gene Therapy PR001 Granted Fast Track Designation
A phase 1/2 clinical trial is expected to be initiated in the second half of 2019.
FDA Grants Fast Track Designation to ABO-202 AAV9 Gene Therapy for Batten Disease
Abeona Therapeutics is currently planning a phase 1/2 clinical trial to evaluate ABO-202 in Batten disease.
Huntington Disease Gene Therapy AMT-130 Wins FDA Fast Track Designation
The planned phase 1/2 trial of the recombinant AAV5 vector treatment, the first one-time administered AAV gene therapy to enter clinical testing for Huntington disease, is expected to begin dosing patients in the second half of 2019.
ATA190 Demonstrates Promise in Treating Progressive MS
The trial results indicate that EBV-specific adoptive T cell therapy is well tolerated and further back this approach in efficacy trials.
AT132 Granted RMAT Designation for X-Linked Myotubular Myopathy
The designation is backed by positive interim data from ASPIRO, which has demonstrated significant improvements in neuromuscular and respiratory function at week 24.
CD34+ Cell Therapy Receives Regenerative Medicine Advanced Therapy Designation by FDA for Refractory Angina
Clinical data demonstrate that CD34+ cell therapy improves exercise capacity, angina frequency and reduces mortality in no-option refractory angina.
FDA Accepts IND for Gene-Edited Cell Therapy BIVV003 for Treatment of Sickle Cell Disease
The investigational new drug application allows the initiation of phase 1/2 clinical trial to assess the safety of BIVV003 in adults.
Amir Kashani, MD, PhD: Stem-Cell Based Therapy for Dry Age-Related Macular Degeneration
A novel subretinal implantation of a stem cell-based bioengineered patch may restore vision or prevent further vision loss.
First FDA-Approved Gene Therapy Procedure for Inherited Disease Performed
This is the first and only FDA-approved gene therapy for treatment for an inherited disease.
FDA Approves Spark Therapeutics' Voretigene Neparvovec-rzyl
This is the first gene therapy approved in the US targeting mutations in a specific gene.
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