Around the Helix: Cell and Gene Therapy Company Updates – October 25, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Brett Kopelan, MA, and Heather Fullmer on the Potential Impact of Gene Therapy on Epidermolysis Bullosa’s Treatment Landscape
In observance of Epidermolysis Bullosa Awareness Week, the debra of America representatives discussed unmet needs for the patient population and the approval of the first EB gene therapy.
Positive Preclinical Data Support FTD-GRN Gene Therapy Study Initiation
AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.
David Rawlings, MD, on the Potential of Engineered Treg Cells to Treat Autoimmune Diseases
The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.
Focal Epilepsy Cell Therapy NRTX-1001 Shows Efficacy Signals in First Patient Cohort
Neurona Therapeutics also reported that this first patient cohort has completed enrollment in addition to the early efficacy data from the first 2 patients treated.
Mark Walters, MD, on Long-Term Blood Cancer Risk in Children Treated With Gene-Edited Cell Therapy
The professor in residence of pediatrics at University of California San Francisco discussed a potential long-term consequence of gene therapy and myeloablative conditioning.
REGENXBIO’s ABBV-RGX-314 Trials Aim to Reduce Treatment Burden in Inherited Retinal Diseases
The company expects that pivotal ATMOSPHERE and ASCENT trials will support a BLA filing in 2024 for wet AMD, while the AAVIATE trial evaluates the therapy in diabetic retinopathy.
Verve’s Hypercholesterolemia Gene-Editing Therapy VERVE-101 Cleared for US Trial After Rocky Start
The US IND application, on hold since November 2022, was cleared with the help of data from a clinical trial being conducted in New Zealand.
Milind Desai, MD, MBA, on Clinically Evaluating MYBPC3–Associated Hypertrophic Cardiomyopathy Gene Therapy
The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed the design of the MyPeak-1 clinical trial for TN-201 and the associated MyCLIMB natural history study.
Efforts to Advance Cell and Gene Therapies for Breast Cancer
October is National Breast Cancer Awareness Month, and thus serves as an additional moment to point to the progress made in cell and gene therapy development, as well as the lingering challenges.
Trial for CytoAgents’ Drug for Management of CAR-T-Associated CRS Begins Enrolling Participants
The first patient will be treated with CTO1681 at UPMC Hillman Cancer Center.
CGTLive’s Weekly Rewind – October 20, 2023
Review top news and interview highlights from the week ending October 20, 2023.
BrainStorm Cell Therapeutics Pulls BLA for ALS Cell Therapy NurOwn
The company will meet with the FDA again to discuss the future of the investigational cell therapy.
bluebird's HGB-210 Trial Hopes to Support First SCD Gene Therapy Approval
Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.
Milind Desai, MD, MBA, on Addressing Unmet Needs in MYBPC3-associated Hypertrophic Cardiomyopathy
The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed TN-201, an investigational gene therapy for MYBPC3-associated HCM.
Canavan Gene Therapy Improves Developmental Measures Compared to Natural History
MSEL 5-mean domain, receptive language, and expressive language scores were significantly improved from baseline in treated participants.
Around the Helix: Cell and Gene Therapy Company Updates – October 18, 2023
Intellia’s CRISPR Therapy for ATTR-CM First to Enter Late-Stage Trials
Intellia Therapeutics plans to initiate the phase 3 trial of NTLA-2001 by the end of 2023.
NK Cell Therapy Shows Early Antitumor Response in B-NHL
The full data readout from the phase 1/2 clinical trial is expected in the first quarter of 2024.
David Rawlings, MD, on Research That Supports the Development of Engineered B-cell Therapies
The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed findings from several preclinical studies that could help bring engineered B-cell therapies to clinical trials.
Trial Evaluating Anixa’s CER-T Approach to Treating Ovarian Cancer Finishes Dosing First Cohort
The company noted that there have been no dose-limiting toxicities so far among the 3 patients in the cohort.
Engineered Plasma Cells Show Potential in Leukemia Mouse Model
Richard James, PhD, an associate professor at the University of Washington and a principal investigator at Seattle Children's Research Institute, discussed a potential alternative to T-cell therapy in cancer.
TCR-T Therapy Cleared for Lung, Gastric Cancer Trial
Biosyngen is also evaluating the CAR T-cell therapy BRG01 in a phase 1/2 trial for EBV-positive relapsed/metastatic nasopharyngeal carcinoma.
MSC Therapy Shows Improvements in Open-Label Extension Trial for Patients With Multiple Sclerosis
Among 15 tested patients, who received at least 2 IT injections of the MSC therapy, improvements of between 5% and 18% in 25 feet walking were observed.
Patient With Post-CAR–T Relapse Achieves Complete Response With multiTAA-specific Lymphoma T-cell Therapy
MT-601, in contrast to CAR-T therapies, is not genetically engineered.
Hold Lifted on ASO for Myotonic Dystrophy Type 1, Trial to Initiate in the US
The FDA previously placed the trial on clinical hold for undisclosed reasons.
CGTLive’s Weekly Rewind – October 13, 2023
Review top news and interview highlights from the week ending October 13, 2023.
Fractyl’s Single-Dose Obesity Gene Therapy GLP-1 PGTX Outcompetes Chronic Semaglutide in Rodent Study
The mechanism for weight loss was attributed to reduced food intake observed in both treatment groups.
Sharon Hesterlee, PhD, on Further Research Needs With Muscular Dystrophies
The chief research officer of the Muscular Dystrophy Association discussed research needs that remain in the field in light of recent progress and approvals.
Richard James, PhD, on the Potential of Engineered Plasma Cells in Cancer
The associate professor at the University of Washington, and principal investigator at Seattle Children's Research Institute discussed results from a leukemia mouse model.
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