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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
First Patient Receives Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.
Novartis Drops Development of Geographic Atrophy Gene Therapy GT005
Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.
Sharon Hesterlee, PhD, on Unprecedented Progress in Muscular Dystrophies
The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.
First Patient With MSLN-Solid Tumors Receives SynKIR-110 CAR T-Cell Therapy
SynKIR-110 is based off of the KIR-CAR platform developed at the University of Pennsylvania.
Juan Francisco Cabello, MD, on the History and Current State of Newborn Screening
The head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile discussed positive impact of newborn screening so far and work that still needs to be done.
Multi-Base–Edited CAR T-Cell Therapy Trial Doses First Patient With R/R T-ALL/T-LL
Beam Therapeutics also expects to announce initial data from its BEACON trial in sickle cell in 2024.
BioCardia Likely to Miss the Mark in Phase 3 CardiAMP Heart Failure Cell Therapy Trial
The company also announced its plans for a modified study protocol that may allow a roadway to approval.
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.
RNAi Therapeutic Shows Dose-Dependent Reductions in Systolic Blood Pressure
Zilebesiran is being evaluated as a monotherapy in the KARDIA-1 study and as a combination therapy in KARDIA-2, which will report topline data in early 2024.
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.
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