Verismo Therapeutics’ CAR-T SynKIR-310 Cleared for Trial in R/R B-NHL
Verismo intends to go forward with plans for the phase 1 CELESTIAL-301 clinical trial, which the company expects to initiate in the second half of this year.
Faraz Ali, MBA, on Adding Gene Editing Programs to Tenaya’s Cardiovascular Disease Pipeline
The chief executive officer of Tenaya Therapeutics discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.
Considerations for Gene Therapy Logistics and Implementation
Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.
Liso-Cel/Breyanzi Gets Accelerated Approval for Relapsed/Refractory Follicular Lymphoma
The CAR T-cell therapy was also recently approved for treating chronic lymphocytic leukemia or small lymphocytic lymphoma.
AADC Deficiency Gene Therapy up For FDA Priority Review
Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.
Around the Helix: Cell and Gene Therapy Company Updates – May 15, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Evan Weber, PhD, on Improving CAR T-Cell Fitness
The assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.
Highlights From ACGT Summit 2024 Scientific Meeting
The invitation-only annual scientific summit, held on March 29, in New York City, brought together top scientists in cancer cell and gene therapy.
Verve Therapeutics Doses First Patient in Phase 1b Trial for Gene Therapy VERVE-102
VERVE-102 is intended to reduce LDL-C in patients with heterozygous familial hypercholesterolemia or premature coronary artery disease.
Faraz Ali, MBA, on Continued Innovation With Cardiovascular-Targeted AAV Gene Therapy
The chief executive officer of Tenaya Therapeutics discussed the company’s research on capsids, promoters, and manufacturing improvements.
Multicharacteristic Opsin Gene Therapy Improves BCVA, MLSDT in Retinitis Pigmentosa
High dose MCO-010 yielded statistically significant improvements in BCVA and MLSDT at week 52.
Shankar Ramaswamy, MD, on Bringing Gene Therapy to Common Diseases
The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.
ALS REGALS Trial Continues to Evaluate Cellenkos’ CK0803 Treg Therapy
The trial is set to dose its second cohort following a positive DSMB recommendation in March.
Kevin Campbell, PhD, on the Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy
The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.
Data Roundup: April 2024 Features Updates from AACR, AAN, in Muscular Dystrophy and Cancer
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Orchard Therapeutics’ Arsa-Cel Continues to Show Safety in Late Juvenile MLD
Updated data including a sixth patient and longer follow-up was presented at ASGCT’s 2024 Meeting.
Debora Mazzetti, MS, on Multitargeting MicroRNA in Glioblastoma
The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.
Abhishek Gupta, BS, on Addressing Friction Points in the Commercialization of Gene Therapies
The senior vice president of genetic medicines at Syneos Health discussed the importance of end-to-end thinking and collaboration when bringing gene therapies from development to the market.
Tumor Agnostic Deltarex-G+ Gene Therapy Demonstrates Activity in Breast, Pancreatic, Sarcoma Cancers
All 139 tumors screened had enhanced expression of CCNG1, a seldom-recorded gene on NGS.
Francesca Del Bufalo, MD, PhD, on Evaluating GD2-Directed CAR-T in Pediatric Neuroblastoma
The medical doctor and scientist at Bambino Gesù Chidren’s Hospital discussed phase 1/2 clinical trial data she presented at ASGCT’s 2024 Meeting.
CGTLive®’s Weekly Rewind – May 10, 2024
Review top news and interview highlights from the week ending May 10, 2024.
World Lupus Day 2024: Looking Back at a Year of New Cell Therapy IND Clearances
In observance of World Lupus Day, held annually on May 10, we took a look back at a year of progress in bringing CAR-T to this autoimmune disease.
Elevidys Shows Slight Statistical Improvements on Time to Rise, 10-Meter Walk/Run Despite NSAA Fail
The gene therapy is up for priority review with a PDUFA date of June 21, 2024.
NGGT Biotechnology’s Gene Therapy NGGT001 Improves Vision in Patients With Bietti’s Crystalline Dystrophy
In terms of safety, NGGT001 was deemed well-tolerated.
Luke Roberts, MBBS, PhD, on Early Clinical Data on Congestive Heart Failure Gene Therapy
The medical director of clinical development at AskBio discussed safety and efficacy data from a phase 1 trial of AB-1002.
Rod-Cone Dystrophy Gene Therapy Demonstrates Acceptable Safety, Study to Continue
The DSMB recommended proceeding to dose the highest dose cohort in the phase 1/2 PRODYGY study.
Lawrence R. Lustig, MD, on Evaluating Gene Therapy for the Treatment of Otoferlin-Related Hearing Loss
The chair of the Department of Otolaryngology—Head and Neck Surgery at Columbia University College of Physicians discussed the phase 1/2 CHORD clinical trial evaluating DB-OTO.
Casey Maguire, PhD, on Combining Viral-Like Particles and AAV
The associate investigator of neurology at Massachusetts General Hospital discussed research on extracellular vesicle-associated AAVs presented at ASGCT.
Around the Helix: Cell and Gene Therapy Company Updates – May 8, 2024
SPG50 Gene Therapy Warrants Further Study for Spastic Paraplegia
MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.
