Treating Autoimmune Diseases With Smarter Cell Therapies
Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.
Around the Helix: Cell and Gene Therapy Company Updates – October 5, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CAR T Therapy Yields Remission in Systemic Lupus Erythematosus
Five patients with lupus treated with anti-CD19 CAR T-cell therapy are now in remission.
B-NHL Cell Therapy Cleared for Trials
Oncternal will soon initiate a phase 1/2 dose-escalation study of ONCT-808.
Christine Coughlin, MD, PhD, on TRACK-NK Technology in Non-Small Cell Lung Cancer
The chief executive officer of Cytoimmune discussed the company’s technology platforms and programs.
Gene Therapy Yields Improvements in Danon Disease
Rocket Pharma presented updated data from adult and pediatric patients at the 2022 HFSA meeting.
Sid Kerkar, MD, on Developing CAR T Therapies Without Lymphodepletion Chemotherapy
The vice president of oncology and research and development at Exuma Biotech discussed the company’s platforms, including CAR-TaNK cells.
Solid Bio Adds Friedreich Ataxia to Pipeline With Aavanti Acquisition
Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.
Wet AMD Gene Therapy Reduces Anti-VEGF Treatment Burden
Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.
Advancing Neonatal Cell Therapy Research
Newborn Cell Therapies Group at Monash University has received a $50,000 grant from Lions Cord Blood foundation.
Omidubicel Improves Survival in Hematologic Malignancies With HSCT
Disease-free survival was 56% in 105 participants over 3 years.
CGTLive’s Weekly Rewind – September 30, 2022
Review top news and interview highlights from the week ending September 30, 2022.
Modified Immune Cell Therapy Induces Kidney-Donor-Specific Immunosuppression
Patients treated with the highest dose of MIC-Lx had lymphocyte reactivity against third-party cells but not stimulatory donor blood cells.
Working With the Community to Improve Patient Access to Trials, Therapies
Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.
KIR-CAR T-Cell Therapy Recognized for Mesothelioma Treatment
Verismo plans to initiate a phase 1, first-in-human trial of SynKIR-110 in the first quarter of 2023.
Sarepta’s DMD Gene Therapy Goes In Front of FDA With BLA Submission
SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.
Cellenkos to Try T Regulatory Cell Approach in ALS
A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.
Allogeneic CAR T Recognized for Cutaneous T-Cell Lymphomas
CTX-130 is being investigated in phase 1 studies for relapsed/refractory T or B-cell malignancies and renal cell carcinoma.
Around the Helix: Cell and Gene Therapy Company Updates – September 28, 2022
Leaping the Next Hurdle in CAR T-Cell Therapies
Phil Cyr, Senior Vice President, Precision Value & Health, discussed challenges to tackle with CAR T-cell therapies.
Vertex Continues Gene Therapy Momentum in Blood Disorders With Exa-Cel Submission
Bluebird bio’s ZYNTEGLO was the first gene therapy to be approved in the space in August 2022.
Repeated Injections of ALS Gene Therapy Well-Tolerated
Although efficacy and HGF expression were assessed as outcome measures, comparisons were not able to be made between placebo and Engensis groups.
DMD Cell Therapy Shows Promise in Early Clinical Trial Data
The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.
Optimizing CAR T Therapies for Gastric Cancer
Hong Ma, senior vice president, clinical development, cancer immunotherapy, CARSgen, discussed data from a phase 1 trial of CT041.
University of Miami Researcher Awarded $3.5 Million for Research on Gene Therapy for Usher Syndrome
Xue Zhong Liu, MD, PhD, and research partner Zheng-Yi Chen, PhD, will investigate gene editing and CRISPR/Cas9 systems to potentially treat Usher syndrome.
First Patient Dosed in Limb-Girdle Muscular Dystrophy Gene Therapy Trial
The 2-stage, multicenter clinical trial will recruit approximately 39 patients who have been diagnosed with LGMDR9.
Deyaa Adib, MD, on Safety Advantages of TAC T-Cell Therapy in Solid Tumors
The chief medical officer of Triumvira Immunologics discussed how TAC01-HER2 could address unmet needs in treating solid tumors.
First-in-Human Trial to Assess KIR-CAR T-Cell Therapy in MSLN+ Solid Tumors
Verismo plans to begin enrollment in the STAR-101 trial in the first quarter of 2023.
Target for Polycystic Kidney Disease Gene Therapy Might Lie in MicroRNA Binding Sites
Elimination of the miR-17 binding site on PKD1 and PKD2 mRNA alleviated cyst growth in preclinical models.
Jeffrey Miller, MD, on Emerging Research With Natural Killer Cells
The deputy director of the Masonic Cancer Center discussed trends of presentations from ESMO 2022.
