Giant Axonal Neuropathy Gene Therapy Needs Double-blind Trial Before BLA Submission
The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.
Remestemcel-L Cell Therapy Makes Comeback With Resubmitted BLA for Pediatric GvHD
Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.
NK Cell Therapy/Rituximab Combination Fast Tracked for B-cell Non-Hodgkin Lymphoma
AB-101 is also being investigated with the innate cell engager AFM13 for CD30 lymphomas.
Working Toward a Single Dose Gene Therapy for Type 1 Gaucher Disease
Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.
Senti Bio Prioritizes Logic Gated Cell Therapies
Positive preclinical data related to SENTI-202 and SENTI-401 were presented at conferences last year.
Multi-characteristic Opsin Gene Therapy Gets Second Fast Track for Retinal Degenerative Diseases
MCO-010 is in phase 2 clinical trials for treating retinitis pigmentosa and Stargardt disease.
Rajni Agarwal-Hashmi, MD, on Safety Advantages of Gene Therapy in Fanconi Anemia Over SOC
The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.
Solid Tumor Cell Therapy Combination Trial Cleared to Continue
There were no deaths or safety signals identified by the DSMB for the 3 treated patients in cohort 1.
Allogeneic CAR T Cleared for B-cell Malignancy Trials
Sana Biotechnology also plans to submit another IND for the same indications for a CD22-targeted CAR T later in 2022.
MSC Therapy Shows Promise in Diabetic Kidney Disease
No adverse events related to the infusions of stromal cells were reported.
Advanced Colorectal Cancer CAR T Cleared for Trial
The phase 1 portion of the trial assessing CNA3103 will start enrollment in Australia in the first half of 2023; the phase 2 portion will expand to the US.
Cilta-cel CARTITUDE-4 Trial to be Unblinded After Meeting Primary End Point
Cilta-cel demonstrated a significant improvement in progression-free survival over standards of care.
CGTLive’s Weekly Rewind – January 27, 2023
Review top news and interview highlights from the week ending January 27, 2023.
Matt Angel, PhD, on Addressing Unmet Needs in Acute Myeloid Leukemia and Solid Tumors
The chief executive officer and president of Eterna Therapeutics discussed the company’s new collaboration with the University of Texas MD Anderson Cancer Center.
Glioblastoma CAR-T Trial Completes Initial Follow-up of Third Dose Cohort
No dose-limiting toxicities were observed at 28 days of follow-up.
Discogenic Progenitor Cell Therapy Shows Improvements in Lumbar Degenerative Disc Disease, Recognized by FDA
The DGX-A01 study met its primary safety and efficacy endpoints in its highest dose cohort of IDCT.
Refractory Angina Gene Therapy Demonstrates Safety and Efficacy in Phase 2 Trial
Nearly half of participants were able to engage in normal physical activity without angina at 6 months post-treatment.
New Data Supports Breyanzi’s Expansion to Chronic Lymphocytic Leukemia
Breyanzi is currently approved for the second-line or later treatment of large B-cell lymphomas.
DMD Cell Therapy Continues to Demonstrate Efficacy After 18-month Follow-up
Patients continued to show statistically significant upper limb performance improvements.
Around the Helix: Cell and Gene Therapy Company Updates – January 25, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Logic-gated CAR T Therapy Cleared for Lymphoma Trials
The CD19/CD20 dual-targeted CAR is currently under investigation in an investigator-initiated study in UCLA.
Allogeneic T-cell Progenitor Therapy Trial Doses First Patient With Leukemia
The dosing of the first patient took place in December 2022 and no adverse events related to the therapy have been reported.
TScan Set to Evaluate Multiplex TCR T Therapies for Solid Tumors
The FAD has granted primary IND clearance to the company’s T-Plex program and 2 initial TCR-T therapies.
Rett Syndrome Gene Therapy Cleared for Clinical Trial
NGN-401 is Neurogene’s second investigational gene therapy to enter clinical trials.
COVID-19 Cell Therapy Shows Promise in Proof-of-Concept Clinical Trial
No patients treated with Tevogen Bio’s TVGN 489 showed progression of their COVID-19 infections.
10 Cell and Gene Therapies to Watch in 2023
With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.
Duchenne Muscular Dystrophy Gene Therapy Trial Begins Recruiting Patients
REGENXBIO has also initiated recruitment for AFFINITY BEYOND, an observational study assessing the prevalence of AAV8 antibodies in male patients with DMD.
Diane Simeone, MD, on Screening Patients for CAR-T Eligibility Before Solid Tumor Relapse
Simeone discussed the progress and challenges seen so far in the BASECAMP-1 observational study.
Afami-cel Achieves Durable Responses and Acceptable Safety Profile in Synovial Sarcoma
The overall response rate was 44% among 16 patients with synovial sarcoma.
Adam Cuker, MD, on Higher Efficacy of Gene Therapy Over SOC in Hemophilia B
The associate professor of medicine at University of Pennsylvania discussed new data from the BENEGENE-2 study of fidanacogene elaparvovec.
