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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
SPG50 Gene Therapy Warrants Further Study for Spastic Paraplegia
MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.
Travis Drow, BS, on Evaluating Engineered Tregs for Potential in Treating Multiple Sclerosis
The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.
Patient With DMD Dies in Pfizer’s Phase 2 Gene Therapy Trial
In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.
TDT/SCD Lentiviral Therapy Boasts Quick, Well-Tolerated Engraftment in Patients
KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.
Holly Peay, PhD, on Patient Preference Research as a Means of Patient Expression
The senior research scientist at RTI International discussed how patient preference studies can help patients with rare diseases have their voices heard.
First Patient Set to Receive bluebird’s Approved Sickle Cell Gene Therapy, Lyfgenia
Lovo-cel was approved as Lyfgenia in December 2023.
Carlos A. Ramos, MD, on Alternatives to T-Cell Therapy
The professor of medicine at Baylor College of Medicine discussed research with NK-T cells and alternatives to αβ T-cells.
Intellia’s Phase 3 Clinical Trial for CRISPR-Based Gene Therapy Enrolls First Patient
The first patient in the trial, which is evaluating NTLA-2001 for the treatment of ATTR Amyloidosis, was enrolled at MedStar Washington Hospital Center.
Prime Medicine’s Gene-Edited Stem Cell Product PM359 Cleared for US Trial in Chronic Granulomatous Disease
The company anticipates that initial data from the study will be announced next year.
Advances in CRISPR/Cas9 Gene Editing Will Be Highly Represented in ASGCT Presentations This Year
Terence R. Flotte, MD, the vice president of ASGCT, discussed what attendees can expect at the ASGCT Annual Meeting this year.
Holly Peay, PhD, on Keeping Patient Preference in Focus
The senior research scientist at RTI International discussed the progress that has taken place in recent years with regard to patient preference research.
CGTLive®’s Weekly Rewind – May 3, 2024
Review top news and interview highlights from the week ending May 3, 2024.
Terence R. Flotte, MD, on the Upcoming ASGCT Annual Meeting and Recent Trends in Gene Therapy
The vice president of ASGCT discussed important research that will be represented at the upcoming conference and challenges for the field to address in the future.
ODAC Supports MRD Endpoint in Accelerated Approval for Multiple Myeloma
The ODAC unanimously voted in favor of the use of minimal residual disease negativity.
Labcorp’s Companion Diagnostic Assay for Beqvez Receives FDA Approval
Testing for the presence of neutralizing antibodies is an essential step before patients can receive Beqvez.
