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.
CGTLive’s Weekly Rewind – January 20, 2023
Review top news and interview highlights from the week ending January 20, 2023.
Late-Onset Pompe Disease Gene Therapy Cleared to Continue Trial
The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.
Diane Simeone, MD, on Addressing Unmet Needs in Colorectal Cancer, NSCLC, and Pancreatic Cancer
Simeone discussed the design of BASECAMP-1, an observational study for patients previously treated for solid tumors who are at a high risk of relapse.
Editas Sells off iNK Preclinical Cell Therapy Program, Gene-Editing Technologies to Shoreline
The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.
Gene Therapy Improves Visual Acuity in MT-ND4 LHON Compared to Natural History
Investigators analyzed data from 3 phase 3 studies and a long-term follow-up study.
Etranacogene Dezaparvovec “Transformative” for Patients With Hemophilia B
Steven Pipe, MD, CS Mott Children’s Hospital, discussed the latest follow-up data from the HOPE-B study of the approved therapy, Hemgenix.
Alan Trounson, PhD, MSc, on New Frontiers in Allogeneic Cell Therapy
The chief executive officer of Cartherics discussed the company’s development of NK cell, T-cell, and macrophage cell therapies for various solid tumor indications.
Around the Helix: Cell and Gene Therapy Company Updates – January 18, 2023
CAR-T for NMOSD Demonstrates Manageable Safety in Phase 1 Trial
It was previously announced that CT103A received FDA clearance of its investigational new drug application for the treatment of relapsed/refractory (r/r) multiple myeloma.
Neovascular AMD Gene Therapy Cleared for Trials
A phase 1 clinical trial of EXG102-031 is expected to initiate in the first quarter of 2023.
Akshay Sharma, MBBS, on Investigating CRISPR/Cas9 Edited Cells for Sickle Cell Disease
The assistant member of the bone marrow transplant department at St. Jude Children’s Research Hospital discussed advantages of OTQ923 in treating SCD.
Krabbe Disease Gene Therapy Receives EMA PRIME Designation
Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.
CAR-T in Fibrin Glue-based Carrier Shows Potential as Adjuvant to Solid Tumor Surgery
Of the 20 mice treated with the experimental approach, 19 achieved clearance of residual tumor cells.
