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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Patients With T1D Achieve Insulin Independence Following Implantation of Cell Pouch System and Islet Transplant
Five patients in the cohort achieved this milestone and Sernova noted a sixth patient in the cohort is still awaiting assessment of islet graft function.
Gene Therapy Modifies Disease Progression in Patients With Giant Axonal Neuropathy
Taysha also provided updates on its gene therapy program for Rett syndrome.
FDA Approves BioMarin’s Hemophilia A Gene Therapy Roctavian
The FDA’s decision to approve this first gene therapy in hemophilia A was based on data from the phase 3 GENEr8-1 clinical trial.
Silence and Replace Gene Therapy Cleared for Oculopharyngeal Muscular Dystrophy Clinical Trial
Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301.
Parkinson Disease Neuronal Cell Therapy Demonstrates Safety, Cell Survival
Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.
FDA Approves Lantidra Cell Therapy for Type 1 Diabetes
With an original PDUFA date in August 2021, the decision is a long time coming.
Around the Helix: Cell and Gene Therapy Company Updates – June 28, 2023
Dan Shelly, PhD, on Developing an Allogeneic, Adaptable CAR Platform
The vice president of business development and alliances at Prescient Therapeutics discussed advantages of the OmniCAR, SpyTag, and SpyCatcher technologies.
High Dose NK Cell Therapy Shows Efficacy in AML
Updated data are from a total of 36 patients treated in a phase 1/2 study as of June 2023.
Bella Neufeld, PhD, on Streamlining Buffer Selection for AAV Gene Therapy Manufacturing Process Development
The director of research and development at Teknova discussed the company’s new screening kit and its potential to speed up AAV gene therapy development.
FDA Accepts BLA Filing for Hemophilia B Gene Therapy Fidanacogene Elaparvovec
In addition to the BLA acceptance, Pfizer noted that a European MAA for the gene therapy has been accepted by the EMA.
