HEMO-CAR-T IND Gets Clinical Hold for Manufacturing Issue
Hemogenyx is currently remanufacturing the therapy without the identified splicing issue.
RNA Therapy Shows No Benefit for ALS or FTD
Wave Life Sciences is discontinuing WVE-004.
First Patient With NHL Receives γδ T-Cell Therapy in Phase 1 Trial
ACE1831 is Acepodia’s first γδ T-cell therapy to enter clinical trials.
NurOwn Cell Therapy Reduces NfL in Patients With ALS
Reductions in NfL were also associated with changes on ALSFRS-R scores.
AMD Gene Therapy Cleared for US Trial
A trial in China was initiated in December 2022 for patients with neovascular and wet age-related macular degeneration.
Gene Therapy Yields Vision Improvements in Patients With LHON
Data from patients treated in early access programs were presented at the 9th EAN Congress.
Gene Therapy Modifies Disease Progression in Patients With Giant Axonal Neuropathy
Taysha also provided updates on its gene therapy program for Rett syndrome.
Parkinson Disease Neuronal Cell Therapy Demonstrates Safety, Cell Survival
Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.
FDA Approves Lantidra Cell Therapy for Type 1 Diabetes
With an original PDUFA date in August 2021, the decision is a long time coming.
High Dose NK Cell Therapy Shows Efficacy in AML
Updated data are from a total of 36 patients treated in a phase 1/2 study as of June 2023.
Assessing GPRC5D-Targeted CAR T Therapy for R/R Multiple Myeloma
Susan Bal, MD, assistant professor of medicine at University of Alabama – Birmingham, discussed new data on BMS-986393.
Mesenchymal Stromal Cell Therapy Shows Potential in Affecting Crohn Disease Features
The new analysis is from a phase 1 trial of Celularity’s legacy MLASC therapy.
FDA Approves Sarepta's Landmark DMD Gene Therapy Elevidys
Delandistrogene moxaparvovec treatment will reportedly cost $3.2 million.
Inhibitory Interneuron Cell Therapy Reduces Seizures in First 2 Patients With MTLE
Data from a phase 1/2 clinical trial of NRTX-1001 were presented at the ISSCR 2023 Annual Meeting.
Patient Death Prompts Hold on Arcellx’s CART-ddBCMA for R/R Multiple Myeloma
The company believes that limitations on bridging therapies were a contributing factor to the death.
2023 World Sickle Cell Day: The Home Stretch for Gene Therapy in SCD?
More work remains to be done to improve accessibility to populations and countries that have the highest unmet need with SCD treatment.
MDA, CureDuchenne, PPMD Collaborate to Reduce AAV Antibodies to Enable Gene Therapy
The advocacy groups are funding a 1-year study to be led by Barry Byrne, MD, PhD, at University of Florida.
Genetic Medicine in DMD: End Points, Assessment, and Approvals
CGTLive takes a look at challenges that have beset the first gene therapy coming close to market for treating Duchenne muscular dystrophy in 2023.
Point-of-Care CAR T-Cell Therapy Yields 100% ORR in R/R CLL With or Without Richter’s Transformation
The phase 1/2 Euplagia-1 trial is currently ongoing in Europe.
Base-Edited CD7-CAR T Cells Show Early Efficacy in T-ALL
New data from 3 patients were published in the New England Journal of Medicine.
EDIT-301 Increases Total and Fetal Hemoglobin in Patents with Sickle Cell Disease
The therapy seems to have a similar benefit in the first patient with TDT in the EdiTHAL trial.
Investigating CRB1 Mutations in Retinitis Pigmentosa 12
Sang Yoon Moon, PhD, postdoctoral Research Associate, Lions Eye Institute, discussed research with CRISPRa in human fibroblast cultures.
Lentiviral Gene Therapy Stabilizes Patients With MPS1H
Follow-up data up to 3 years showed supraphysiologic levels of blood IDUA and decrease of GAGs.
FDA Accepts Exa-cel BLA for Sickle Cell and Transfusion-Dependent β-Thalassemia
CRISPR Therapeutics and Vertex Pharmaceuticals completed their rolling submissions in April 2023.
Legend Biotech Seeks Expanded Use for Cilta-cel in Lenalidoimide-Refractory Multiple Myeloma
The most recent data from CARTITUDE-4 were presented at the 2023 ASCO Meeting.
TCR-T Therapy Shows Antitumor Activity in HBV-Hepatocellular Carcinoma
Updated data were presented at the ISCT 2023 Meeting.
Study Assessing Vibecotamab in MRD+ AML and MDS/CMML After Hypomethylating Agents
The study has enrolled 17 patients as of May 2023 and enrollment is expected to complete by the end of 2024.
New Survival Data Cements Axi-Cel's Place as a Second-Line SOC for LBCL
New overall survival analysis data on Yescarta from ZUMA-7 were presented at the ASCO 2023 meeting.
Cilta-cel Beats SOC in New Phase 3 Data for Lenalidomide-Refractory Multiple Myeloma
Safety findings were also improved when compared with the previous CARTITUDE-1 study, suggesting the therapy may be better tolerated in earlier lines of therapy.
START-001 is Evaluating TCR-Targeted Bifunctional Antibody in Patients With Solid Tumors
The study began enrollment in January 2023.
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