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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
First Patient Dosed With LCA5 Gene Therapy
Opus Genetics plans to add a pediatric cohort to the phase 1/2 trial once safety is established in adults.
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.
Anixa Makes Progress in CER-T Trial for Ovarian Cancer
The trial has dosed its third patient, and, safety validation pending, plans to dose a second cohort beginning at the end of 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.
Artiva NK Cell Therapy AlloNK/ Affimed Innate Cell Engager Combo Gets Fast Track Designation
The companies plan to evaluate the combination therapy in patients with HL in the phase 2 LuminICE-203 clinical trial.
Michael Kelly, PhD, on Continuing Progress With Gene Therapy in Muscular Dystrophy
The chief scientific officer of CureDuchenne discussed progress in the field so far and upcoming milestones.
Rocket Pharmaceuticals Confirms Phase 2 Trial Plans for Danon Disease Gene Therapy With FDA
The study will utilize a pediatric safety run-in that will include 2 patients.
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.
Sebastian Michels, MD, on the Role of Alternative Polyadenylation in ALS Disease Pathogenesis
The postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed findings he presented at ANA’s 2023 conference that may have implications for future RNA therapy development.
Hemogenyx Clears a Path for AML CAR-T Therapy
The FDA has accepted the company’s plan to address its current IND clinical hold on HEMO-CAR-T.
CGTLive’s Weekly Rewind – September 15, 2023
Review top news and interview highlights from the week ending September 15, 2023.
Thomas McCauley, PhD, on Potential Advantages of Epigenetic Therapy Over Small Molecule, Gene Therapy
The chief scientific officer at Omega Therapeutics discussed the issues with other modes of therapies that OEC therapy may address.
Kyverna’s Lupus Nephritis CAR-T Generally Well-Tolerated in First Patient Treated in the US
Kyverna stated that the patient, who received KYV-101 in July at the University of Colorado Anschutz Medical Campus, did not experience any ICANS.
Addition of Quizartinib FLT3 Inhibitor to Cell Transplant Improves Survival in AML
Patients were newly diagnosed and had acute myeloid leukemia positive for FLT3-iTD mutations.
Juan Francisco Cabello, MD, on How Gene Therapy Fits Into the Global Initiative for Newborn Screening
The head of the Pediatric Neurology Fellowship Program at the University of Valparaiso in Chile discussed how a lack of cost-effectiveness may hold back the potential of newborn screening in gene therapy.
MRD Negativity Predicts DOR With Cilta-Cel in Patients With Multiple Myeloma
A final analysis of data from CARTITUDE-1 was also presented at SOHO 2023, which revealed a PFS of 24.9 months in treated patients.
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.
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