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.
Ofra-Vec Continues to Show No Benefit in Platinum-Resistant Ovarian Cancer
Follow-up data were presented from the discontinued phase 3 OVAL study.
Assessing TCR-T Therapy in KRAS, EGFR, and TP53-Expressing Tumors
Maria Pia Morelli, MD, PhD, assistant professor at MD Anderson Cancer Center, discussed the first-in-human study of Sleeping Beauty TCR-T cells.
PepGen’s Myotonic Dystrophy Oligonucleotide Trial Stalls
In more positive news, the company’s phase 2 trial for treating DMD will enroll patients in the second half of 2023.
CRISPR-Edited/Oncolytic Virus Cell Therapy Combo Shows Preclinical Efficacy in Melanoma Brain Metastases
The promising preclinical data, observed in an accurate model of brain metastases, support initiation of a phase 1 trial.
Cleaving AAV Antibodies to Improve Gene Therapy Accessibility
Lena Winstedt, PhD, Hansa Biopharma, discussed imlifidase, which is being investigated in combination with gene therapies from Sarepta and Genethon.
Danon Disease Gene Therapy Improves Disease Biomarkers
All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.
Gene Therapy Improves Quality of Life in Patients With Glycogen Storage Disease Type 1a
All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.
High Dose Fordadistrogene Movaparvovec Improves NSAA, Muscle Volume in Patients With DMD
The trial compared data from external controls including a recent domagrozumab null study and a propensity score weighted comparison.
FDA Approves Epkinly T-Cell Engaging Bispecific Antibody for R/R DLBCL
The approval was based on data from the phase 1/2 EPCORE NHL-1 clinical trial.
CSF Infusion of Gene Therapy Transduces Cochlear Cells
The most robust transduction in nonhuman primates was seen with ICV delivery of pooled PM4.AAV.mRFP and AAV2.mNG gene therapy,
Dual Injections of 2-Vector Gene Therapy May Stabilize Disease in GM2 Gangliosidosis
Data trends suggesting stabilization of disease were seen in patients with Tay-Sachs, but more research is needed, especially for those with Sandoff disease.
Multi-Characteristic Opsin Gene Therapy for Retinitis Pigmentosa Improves Multiple Measures of Vision
Updated data from the RESTORE study on MCO-010, Nanoscope Therapeutics' investigational gene therapy, were presented at the 2023 ASGCT meeting.
Trial to Assess Selecta’s Gene Therapy/Immunomodulatory Combo in Patients With MMA
ImmTOR induces immune tolerance and may reduce or eliminate Nab formation, allowing potential redosing of gene therapy.
Intraarticular Gene Therapy Well-Tolerated, Improves Pain in Knee Osteoarthritis
Updated data from a phase 1 study of PCXR201, formerly known as Flexion’s FX201, were presented at the 2023 ASCGT Annual Meeting.
FDA AdComm Leans Yes on Sarepta DMD Gene Therapy
Committee members were mostly split, ultimately voting 8–6 in favor of the benefit-risk profile of SRP-9001 supporting accelerated approval.
TCR-Based T-Cell Therapy Cleared for Trials
The neoantigen-specific TCR therapy is the first to enter clinical trials out of a collaboration with Genentech.
Sangamo Loses Collaborators on Neurological, Neurodevelopmental Gene Therapy Programs
Biogen and Novartis terminated collaborations with the company that first initiated in 2020.
MSLN-Targeted KIR-CAR Therapy Trial Initiates at UPenn
SynKIR-110 was also granted fast track designation by the FDA in April 2023.
First Patient With ALS Dosed With Treg Cell Therapy
CK0803 is being evaluated for safety and tolerability in the REGALS study.
ICER Reports Sickle Cell Gene Therapy Cost Effective at $1.9 Million
The draft report was released shortly after the BLA submission of exa-cel was completed and shortly before submission of lovo-cel.
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