CRISPR Therapy Shows Proof-of-Concept in Preventing Antibiotic-Resistant E. Coli Infection in Patients With Cancer
The proof-of-concept trial was in healthy volunteers, and additional studies are being planned in patients with blood cancers undergoing hematopoietic stem cell transplants.
Around the Helix: Cell and Gene Therapy Company Updates – July 12, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Carol Miao, PhD, on Bringing Together Novel Gene Editing Tools and Delivery Methods
The principal investigator at Seattle Children’s Research Institute discussed the potential of new delivery methods and gene editing to overcome the limitations of AAV vector-based gene therapies.
Enrollment Nears Completion in Cell Therapy Hemorrhagic Trauma Trial
Invimestrocel is being investigated for multiple indications, including ischemic stroke.
Sensorion Submits UK Clinical Trial Application for OTOF Hearing Loss Gene Therapy
Pending clearance of the CTA for OTOF-GT, the company plans to initiate the phase 1/2 Audiogene clinical trial.
HEMO-CAR-T IND Gets Clinical Hold for Manufacturing Issue
Hemogenyx is currently remanufacturing the therapy without the identified splicing issue.
Sung-Yun Pai, MD, on Further Research With Lentiviral Gene Therapy for X-SCID
The senior investigator at the National Cancer Institute Center for Cancer Research discussed unmet needs that remain and further research to be done.
RNA Therapy Shows No Benefit for ALS or FTD
Wave Life Sciences is discontinuing WVE-004.
Jacob Appelbaum, MD, PhD, on Investigating SC-DARIC33 and IL-15 for AML
The senior research fellow at Fred Hutch Cancer Center discussed research trends at the 2023 ASGCT meeting.
First Patient With NHL Receives γδ T-Cell Therapy in Phase 1 Trial
ACE1831 is Acepodia’s first γδ T-cell therapy to enter clinical trials.
Mary Jane "MJ" Mulcahey, PhD, OTR/L, CPPC, CLCP, FASIA, on Reevaluating Outcome Measures for Spinal Cord Injury Clinical Trials
The director of the Center for Outcomes and Measurement in the Jefferson College of Rehabilitation Sciences at Thomas Jefferson University discussed the research she presented at the first Annual SCI Investor Symposium.
Collaboration Needed to Accelerate the Development of Cell Therapies for Spinal Cord Injury
Brian Culley, MS, the chief executive officer of Lineage Cell Therapeutics, discussed advancements made with SCI cell therapy OPC1 at the first Annual SCI Investor Symposium.
NurOwn Cell Therapy Reduces NfL in Patients With ALS
Reductions in NfL were also associated with changes on ALSFRS-R scores.
Farah Sheikh, PhD, on Developing Gene Therapy for Arrhythmogenic Right Ventricular Cardiomyopathy
The professor of medicine at University of California San Diego discussed preclinical data on the LX2020 gene therapy.
CGTLive’s Weekly Rewind – July 7, 2023
Review top news and interview highlights from the week ending July 7, 2023.
Motor Function Improvements Observed Among Patients With SMA Treated With Nusinersen After Zolgensma
Among 29 patients who were treated with nusinersen in the RESPOND trial and evaluable for efficacy, most demonstrated an increase in mean total HINE-2 score from baseline.
AMD Gene Therapy Cleared for US Trial
A trial in China was initiated in December 2022 for patients with neovascular and wet age-related macular degeneration.
Duchenne Muscular Dystrophy Cell Therapy CAP-1002 Demonstrates Improvements in LVEF
In terms of safety, Capricor Therapeutics’ CAP-1002 was reported to be well-tolerated.
Gene Therapy Yields Vision Improvements in Patients With LHON
Data from patients treated in early access programs were presented at the 9th EAN Congress.
Krystof Bankiewicz, MD, PhD, on GDNF Gene Therapy for Parkinson’s and AADC Deficiency
The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.
Around the Helix: Cell and Gene Therapy Company Updates – July 5, 2023
Hope Remains for CAR-T Approaches in Ovarian Cancer
Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed past and current clinical trials for investigational CAR-T therapies in ovarian cancer.
Jonathan Yen, PhD, on Continuing Research With Prime Editing for Sickle Cell Disease
The director of therapeutic genome engineering, St. Jude Children’s Research Hospital discussed challenges with performing prime editing in cells.
Brian Culley, MS, on Sharing Knowledge From the Development of Spinal Cord Injury Cell Therapy
The chief executive officer of Lineage Cell Therapeutics discussed findings related to its cell therapy OPC1 that the company presented at the first Annual SCI Investor Symposium.
Raphaël Ognar on Adding New Options to the Oncologic Cell Therapy Arsenal
The cofounder and chief executive officer of NKILT Therapeutics discussed unique advantages of the CIR platform.
Evidence of Disease Reduction Observed Among Patients Treated With Ovarian Cancer CAR-T PRGN-3005
Mary “Nora” Disis, MD, director of University of Washington Medicine’s Cancer Vaccine institute, discussed research she coauthored that was presented at ASCO’s 2023 conference.
Insulin-Producing Islet Cell Therapy VX-880 Meets Primary Efficacy End Point in Patients With Type 1 Diabetes
The 2 patients were among 6 who have received treatment in Vertex’s trial so far and were the only 2 evaluable for the primary efficacy end point.
Steven Katz, MD, on Assessing New Delivery Methods for CAR T-Cell Therapy
The chief medical officer of Trisalus Life Sciences discussed studies evaluating the company’s PEDD system.
CGTLive’s Weekly Rewind – June 30, 2023
Review top news and interview highlights from the week ending June 30, 2023.
Bella Neufeld, PhD, on Addressing Pain Points in AAV Gene Therapy Development
The director of research and development at Teknova discussed the company’s recent announcement of a new line of products intended to streamline process development in AAV gene therapy manufacturing.
