CGTLive’s Weekly Rewind – November 11, 2022
Review top news and interview highlights from the week ending November 11, 2022.
CRISPR-CAR T Therapy Yields First Allogeneic Complete Response in R/R Solid Tumors
Data from the COBALT-RCC study in renal cell carcinoma were presented at SITC 2022.
Susan Foy, PhD, on Evaluating Personalized, Neoantigen-Specific T Cell Therapy in Solid Tumors
The CRIPSR-edited neoantigen-specific T cell therapy demonstrated safety and feasibility in the first-in-human PACT-0101 study presented at SITC 2022.
Gene Expression Profiles Characterize CAR T-Cells in Solid and Hematological Tumors
Further analysis will be performed to elucidate which characteristics correlate with optimal cell therapy behavior.
ALS Gene Therapy Momentum Continues With New Orphan Drug Designation
The FDA action follows a slew of recent cell and gene therapy updates in the field of amyotrophic lateral sclerosis.
Around the Helix: Cell and Gene Therapy Company Updates – November 9, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Type 1 Diabetes Cell Pouch System Clinical Trial Cleared to Begin Enrolling Second Cohort
Several of the first cohort patients have had their Cell Pouch implants for over 3 years and no patients have elected to have their implants removed.
Samir Parekh, MD, on Surprising Efficacy of Sequential T-Cell Redirection Therapy in Multiple Myeloma
The director of translational research in myeloma at the Tisch Cancer Institute discussed the relevance of the new research as more CAR T therapies come to market.
BioMarin Prepares for PDUFA Delay for Val-Rox Hemophilia Gene Therapy
Recent communication with the FDA suggests that the agency's decision might be delayed past Q1 2023.
ICER Suggests $2.9 Million Cap for Hemophilia Gene Therapies
A final evidence report suggests a $1.9 million cap for val-rox and a $2.9 million cap for EtranaDez.
OTOF-Mediated Hearing Loss Gene Therapy Gets FDA Rare Pediatric Disease Designation
Preclinical research in an OTOF-knockout mouse model demonstrated reversal of deafness.
FDA Places Gene-Editing Therapy on Hold for Hypercholesterolemia
No treatment-related adverse events have been reported and Verve expects a letter detailing the hold within 30 days.
DMD Patient Dies in CRISPR Gene Therapy Trial Led By Nonprofit Biotech Cure Rare Disease
The trial was launched through a partnership between Cure Rare Disease and UMass Chan Medical School.
Barry Paul, MD, on Improving CAR T-Cell Therapy Efficacy in Multiple Myeloma
The assistant professor of Hematologic Oncology and Blood Disorders at Atrium Health discussed integrating CAR T therapy into treatment paradigms.
Serum-free, Suspension-based Lentiviral Vector Platform May Allow for Accelerated Production
The platform is capable of producing lentiviral vectors for preclinical, clinical, and commercial scales with high titers ranging from 1x10^8 TU/ml to 1x10^9 TU/ml.
Robert Wenham, MD, MS, FACS, On Assessing FSHR-CAR-T Therapy in Ovarian Cancer
The chair of gynecologic oncology at Moffitt Cancer Center gave an overview of a phase 1 trial being conducted at Moffitt Cancer Center.
CGTLive’s Weekly Rewind – November 4, 2022
Review top news and interview highlights from the week ending November 4, 2022.
Type 1 Diabetes Cell Therapy Cleared to Begin Clinical Trial
AlloStem will be evaluated in the CELZ-201 clinical trial for patients with newly diagnosed type 1 diabetes.
RDEB Cell Therapy Improves Wound Healing and Reduces Pain
Abeona has announced topline results from its phase 3 VIITAL study of EB-101.
REGENXBIO’s Gene Therapy Improves Diabetic Retinopathy Disease Severity
Updated data from the ALTITUDE trial were presented at the 55th ARS meeting in Pasadena, CA.
MB-106, A CD20-CAR T, Yields 100% ORR in Waldenstrom Macroglobulinemia
Data from the first 2 patients were presented at the IWWM 2022 meeting.
Encapsulated Cell Therapy Slows Rate of Progression of Macular Telangiectasia Type 2
Neurotech has announced updated data from protocol A and B phase 3 studies.
Around the Helix: Cell and Gene Therapy Company Updates – November 2, 2022
Robert Wenham, MD, MS, FACS, On Advantages of FSHR-CER T for Ovarian Cancer
The chair of gynecologic oncology at Moffitt Cancer Center discussed advantages of targeting follicle stimulating hormone receptor with cell therapy for ovarian cancer.
uniQure Resumes Enrollment in Huntington Disease Gene Therapy Trial
The DSMB has recommended the trial to continue with additional risk mitigation procedures.
Alice Zhou, MD, PhD, and Omar Butt, MD, PhD, on Surprising Associations With Neurotoxicity and Neurofilament Light
The medical oncologists at Washington University School of Medicine in St. Louis discussed surprising findings from their retrospective study.
Researchers Highlight Potential of Gene Editing in Inherited Retinal Diseases
Studies have demonstrated positive data that suggest in vivo gene editing will be the future treatment paradigm for retinal diseases.
New TIL Therapy to be Assessed in NSCLC
Cellular Biomedicine group developed the therapy after efficacy was seen with Iovance’s lifilecuel in solid tumor studies.
Aimee Donald, MBChB, PhD, on Making Progress With Gene Therapy in Lysosomal Storage Diseases
The pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.
Vlad Hogenhuis, MD, MBA, on Developing Controllable CAR Ts for Solid Tumors
The chief executive officer of Chimera Bioengineering discussed CBIO-007 and its planned IND submission for colorectal and triple negative breast cancer.
