Brian Van Tine, MD, PhD, on Looking Ahead on Cell Therapy for Sarcomas
The professor of medicine and pediatrics at Washington University in St. Louis discussed future challenges and exciting research in the sarcoma field.
News and Expert Insights in Rare Cancer Cell Therapy for Rare Cancer Day
In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news and expert insights related to cell therapies in development for these indications.
CGTLive’s Weekly Rewind – September 29, 2023
Review top news and interview highlights from the week ending September 29, 2023.
Dose-Limiting AAV Toxicity Responsible for Cure Rare Disease’s DMD Trial Death
Unforeseen complications may arise when treating older patients with gene therapy.
Despite the Long History of Gene Therapy Research, Crossing the Blood-Brain Barrier Remains a Challenge
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the history and current state of gene therapy research for neurological indications.
Atamyo Therapeutics Submits CTA in Europe for Limb-Girdle Muscular Dystrophy Type 2C/R5 Gene Therapy
Atamyo has received $8.6 million in nondilutive financing from France 2030 for ATA-200's development.
Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314
The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.
Axi-Cel Shows Efficacy in Patients With R/R LBCL Ineligible for ASCT
Around half of patients with R/R large B-cell lymphoma are considered ineligible for autologous stem cell transplant and are thus ineligible for the approved indication of Yescarta.
FDA AdComm Votes Down BrainStorm Cell Therapeutics’ ALS Cell Therapy NurOwn
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.
Deborah Phippard, PhD, on the Future of Gene Therapy in Neurology
The chief scientific officer of Precision for Medicine discussed what lies on the horizon for gene therapies directed at neurological indications, such as Alzheimer disease and Parkinson disease.
Abeona's RDEB Cell Therapy Up for Review
Abeona has submitted a BLA for its EB-101 cell therapy.
Patients Receiving Autologous Stem Cell Transplant After Chemical Burns Show Corneal Improvements
The phase 1/2 trial is opening its second phase of recruitment after being well-tolerated in the first 4 patients and demonstrating some vision improvements.
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.
J. Andrew Livingston, MD, on Forging Forward With Novel Sarcoma Trials
The associate professor at MD Anderson Cancer Center discussed the upcoming trial of NY-ESO-1 TCR/IL-15 NK cell therapy for SS and MRCLS.
Precigen Trying Its Hand at CAR-T for Ovarian Cancer in Phase 1/1b Trial
Notably, the trial is assessing intravenous administration of PRGN-3005 against intraperitoneal administration in 2 separate cohorts.
Optic Atrophy Gene Therapy Cleared for Trials in Australia
NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.
Deborah Phippard, PhD, on Designing Clinical Trials for Gene Therapies in Neurological Indications
The chief scientific officer of Precision for Medicine discussed the unique aspects of clinical trials for gene therapies that need to be considered before initiation.
Krystal Biotech’s Type 1 Alpha-1 Antitrypsin Deficiency Gene Therapy KB408 Cleared for US Trial
Krystal also noted that earlier this month the gene therapy received orphan drug designation from the FDA.
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.
Shankar Musunuri, PhD, on Gene Modifier Therapy vs Gene Replacement Therapy for IRDs
The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.
CGTLive’s Weekly Rewind – September 22, 2023
Review top news and interview highlights from the week ending September 22, 2023.
Mesoblast Gears Up for Third Push for Remestemcel-L's Approval in GvHD
The FDA issued a CRL for the therapy, to be marketed as Ryoncil, in August 2023.
BASECAMP-1 Trial Identifies Patients With Solid Tumors to Assess Novel CAR T-Cell Therapy
J. Randolph Hecht, MD, discusses the challenges researchers have faced in the development of cellular therapy for patients with solid tumors, and details how A2B530 could potentially overcome some of these obstacles.
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.
Deborah Phippard, PhD, on the History and Current State of Gene Therapy for Neurological Indications
The chief scientific officer of Precision for Medicine discussed the previous and current delivery methods used for gene therapies and new innovations on the horizon.
Research Identifies Cell Signatures of CAR T-Cells in Long Remission
Cells from patients with ALL treated in the CARPALL study were analyzed and sequenced.
Around the Helix: Cell and Gene Therapy Company Updates – September 20, 2023
Pat Furlong, BSN, RN, on the State of Gene Therapy in Muscular Dystrophy
The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.
Ocugen’s Gene-Agnostic Gene Therapy OCU400 Demonstrates Improvement or Stabilization in Retinitis Pigmentosa, Leber Congenital Amaurosis
Among 12 patients evaluable for efficacy, 10 patients experienced stabilization or improvement from baseline in BCVA in their treated eyes.
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