September 29th 2023
Review top news and interview highlights from the week ending September 29, 2023.
Unforeseen complications may arise when treating older patients with gene therapy.
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the history and current state of gene therapy research for neurological indications.
September 28th 2023
Atamyo has received $8.6 million in nondilutive financing from France 2030 for ATA-200's development.
September 27th 2023
Committee members were overwhelmingly against the available data supporting NurOwn as an effective treatment for mild to moderate ALS, with 17 no votes, 1 yes vote, and 1 abstention.
Around the Helix: Cell and Gene Therapy Company Updates – September 27, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapy and the Global Initiative for Newborn Screening
Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed factors holding back more widespread adoption of newborn screening for indications treatable with gene therapy.
Alternative Polyadenylation May Play a Role in ALS Disease Pathogenesis
Sebastian Michels, MD, a postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed how his lab’s findings may open the door to developing RNA therapeutics for ALS.
CGTLive’s Weekly Rewind – September 22, 2023
Review top news and interview highlights from the week ending September 22, 2023.
Taysha Gene Therapies Drops Development of Giant Axonal Neuropathy Gene Therapy TSHA-120
The company is now prioritizing its gene therapy program for Rett syndrome.
Around the Helix: Cell and Gene Therapy Company Updates – September 20, 2023
Catching Up on Newborn Screening to Improve Global Health Equity
Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed the current state of newborn screening and what needs to change.
Orchard Therapeutics’ BLA for Metachromatic Leukodystrophy Cell Therapy Arsa-Cel Accepted With Priority Review
The PDUFA data has been set for March 18, 2024.
Identifying Causative Somatic Mutations in Mesial Temporal Lobe Epilepsy May Open the Door to Targeted Therapies
Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, spoke about how his lab’s findings may imply a need to rethink epilepsy treatment development.
CGTLive’s Weekly Rewind – September 15, 2023
Review top news and interview highlights from the week ending September 15, 2023.
Around the Helix: Cell and Gene Therapy Company Updates – September 13, 2023
Somatic Mutations in the Ras-MAP Kinase Pathway May Play a Role in Mesial Temporal Lobe Epilepsy
Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, discussed his nonclinical research on disease pathogenesis in MTLE.
Blinded Cell Therapy Trial Reveals Significant Treatment Effect in Study Arms
Study author Mya C. Schiess, MD, commented on trends seen in the blinded findings so far.
Atamyo Therapeutics’ Limb-Girdle Muscular Dystrophy Gene Therapy ATA-100 Gets FDA IND Clearance
ATA-100 is currently being evaluated in a multicenter phase 1/2 clinical trial (NCT05224505) in Denmark, France, and United Kingdom.
CGTLive’s Weekly Rewind – September 8, 2023
Review top news and interview highlights from the week ending September 8, 2023.
Bemdaneprocel Moves to Phase 2 Study in Parkinson Disease After Positive Phase 1 Data
Significant improvements in ON time without troublesome dyskinesia were reported with the cell therapy after 1 year, with more apparent effects observed in the high-dose group.
uniQure's Mesial Temporal Lobe Epilepsy Gene Therapy AMT-260 Cleared for US Trial
In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.
Nusinersen Show Benefits in Other SMA Populations
New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.
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.
CGTLive’s Weekly Rewind – September 1, 2023
Review top news and interview highlights from the week ending September 1, 2023.
Design Therapeutics' DT-216 Displays Positive Phase 1 Results in Friedreich Ataxia
Data from a multiple-ascending dose study suggest the investigational agent developed by Design Therapeutics was generally well tolerated with dose-related increases in frataxin levels.
Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023
TSHA-102 Gets FDA Fast Track Designation in Rett Syndrome
TSHA-102 has previously received orphan drug and rare pediatric disease designations from the agency and is being investigated in the REVEAL phase 1/2 trial (NCT05606614).
BlueRock Therapeutics' Parkinson Disease Cell Therapy Bemdaneprocel Shows Initial Efficacy in Phase 1 Trial
In light of the promising results, BlueRock is preparing for a phase 2 trial.
Arrowhead Seeks Clinical Trials for ALS, Muscular Dystrophy RNAi Therapies
Pending approval, ARO-DUX4 will be evaluated in patients with facioscapulohumeral muscular dystrophy and ARO-SOD1 will be evaluated in patients with amyotrophic lateral sclerosis.
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.
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.
Richard Fessler, MD, on OPC1 Improving Quality of Life With Spinal Cord Injury
The professor of neurosurgery at Rush University Medical School discussed follow-up in the phase 1/2 trial of LCTOPC1 and further research to be done.
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