September 29th 2023
Review top news and interview highlights from the week ending September 29, 2023.
September 27th 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Abeona has submitted a BLA for its EB-101 cell therapy.
September 22nd 2023
Review top news and interview highlights from the week ending September 22, 2023.
September 20th 2023
Orchard Therapeutics’ BLA for Metachromatic Leukodystrophy Cell Therapy Arsa-Cel Accepted With Priority Review
The PDUFA data has been set for March 18, 2024.
Around the Helix: Cell and Gene Therapy Company Updates – September 13, 2023
CGTLive’s Weekly Rewind – September 8, 2023
Review top news and interview highlights from the week ending September 8, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – September 6, 2023
Arsa-cel Continues to Show Benefit Up To 12 Years of Follow-up in Metachromatic Leukodystrophy
The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.
Kyverna Makes Progress Evaluating Lupus Nephritis Cell Therapy in US, Germany
Phase 1/2 trials have been cleared in Germany and dosed first patients in the US.
CGTLive’s Weekly Rewind – September 1, 2023
Review top news and interview highlights from the week ending September 1, 2023.
Abeona’s Sending Off RDEB Cell Therapy For BLA Submission
In recent data from the phase 3 VITAL trial, EB-101 decreased pain and improved wound healing.
Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023
CGTLive’s Weekly Rewind – August 25, 2023
Review top news and interview highlights from the week ending August 25, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – August 23, 2023
Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation
Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.
Gaucher Disease Type 1 Gene Therapy Trial Proceeds to Dose Second Cohort
The company announced that it had completed dosing in the first cohort of the GALILEO-1 trial of FLT201.
Ornithine Transcarbamylase Deficiency Gene Therapy Gets FDA Orphan Drug Designation
BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.
Neurogene’s Clinical Trial Seeks to Assess CLN5 Batten Disease Gene Therapy NGN-101
NGN-101 will be delivered to each participant via both intracerebroventricular and intravitreal routes on the same day in the phase 1/2 trial, with the therapy assessed for safety and efficacy.
Around the Helix: Cell and Gene Therapy Company Updates – August 16, 2023
Gene Therapy Shows Evidence of Dose-Dependent Effects on GM1 Gangliosidosis Biomarkers
At dose level 2, 3 of 4 participants had increases of β-Gal activity to normal levels for up to 12 months.
CGTLive’s Weekly Rewind – August 4, 2023
Review top news and interview highlights from the week ending August 4, 2023.
BLA Submission Gives Libmeldy a Shot at US Review for Metachromatic Leukodystrophy
The gene-edited cell therapy OTL-200 was approved in Europe in 2020.
Around the Helix: Cell and Gene Therapy Company Updates – August 2, 2023
PKP2 Gene Therapy Cleared for Cardiomyopathy Trial
LEXEO also recently completed dosing in the first cohort of a trial for Friedreich’s ataxia cardiomyopathy.
Exstrophy-Epispadias Complex Cell Therapy Gets Orphan Drug, Rare Pediatric Disease Designations
The first-in-human MuST trial is being conducted in several centers in Germany.
Gene Therapy After Umbilical Cord Blood Transplantation May Help Prevent Immune Response in Patients With Krabbe Disease
Maria Escolar, MD, the chief medical officer of Forge Biologics, discussed the atypical approach being used in the company’s trial for AAV gene therapy FBX-101.
Around the Helix: Cell and Gene Therapy Company Updates – July 26, 2023
CGTLive’s Weekly Rewind – July 21, 2023
Review top news and interview highlights from the week ending July 21, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – July 19, 2023
PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy Nets Fast Track, Orphan Drug Designations
Rocket Pharma is working to soon initiate a phase 1 trial of RP-A601.
Aerosol Cystic Fibrosis Gene Therapy Well-Tolerated, Shows Signs of Efficacy
Data on cohort 2 of the AEROW trial are expected in November 2023.
CGTLive’s Weekly Rewind – July 14, 2023
Review top news and interview highlights from the week ending July 14, 2023.
FDA Releases Guidance on CGT Manufacturing Changes, Good Cause for PMR Noncompliance
The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.