CGTLive’s Weekly Rewind – October 28, 2022
Review top news and interview highlights from the week ending October 28, 2022.
PR001 Hopes to Address Underlying Mutations in Neuronopathic Type 2 Gaucher Disease
CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
TJ Cradick, PhD, on A Potential Cure for HIV With EBT-101
The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.
JC Virus T-Cell Therapy to be Evaluated in Progressive Multifocal Leukoencephalopathy
The trial expects to enroll its first patient in the first half of 2023.
GSK, Lyell Immunopharma Terminate Agreement for Targeted Cell Therapies
The decision comes after strategic uncertainty regarding a review of preliminary clinical data from GSK’s lete-cel product candidate trial.
Around the Helix: Cell and Gene Therapy Company Updates – October 26, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Aimee Donald, MBChB, PhD, on Assessing PR001 in Children With Type 2 Gaucher Disease
The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.
New Process Could Drastically Reduce CAR-T Manufacturing Time
Cells produced by this method were also shown to be more potent in animal models than cells made with typical processes.
Cystinosis Gene Therapy Trial in Full Swing
The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.
Vinaya Murthy, MS, MPH, on Genetic Counseling Services for Rare Disorders
As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.
Celyad Discontinues CYAD-101 for Metastatic Colorectal Cancer
The decision comes after a previous clinical hold delayed key medical milestones in the KEYNOTE-B79 trial.
NIH Awards Grant for Engineered Mechanosensitive Channel Glaucoma Gene Therapy
The grant goes toward developing an innovative approach to gene-agnostically and autonomously regulate pressure.
Alice Zhou, MD, PhD, and Omar Butt, MD, PhD, on Investigating Associations With CAR T Toxicities
The medical oncologists at Washington University School of Medicine in St. Louis discussed background to their research on associations between neurofilament light and neurotoxicity.
GvHD Leads to Death in FCR001 Living Donor Kidney Transplant Trial
The DMC has recommended the FREEDOM-1 trial to continue without restrictions as the patient was treated before protocols were amended to mitigate GvHD risk.
CGTLive’s Weekly Rewind – October 21, 2022
Review top news and interview highlights from the week ending October 21, 2022.
Allogeneic CAR T Yields 15-Month CR in Follicular Lymphoma
The first 6 patients dosed in the ANTLER trial had a 50% CR rate at 6 months.
Improving Access to Rare Disease Care
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
CAR T Therapy to be Evaluated in Lupus Nephritis
KYV-101 was developed using the construct Kyverna licensed from the NIH in January 2022.
Around the Helix: Cell and Gene Therapy Company Updates – October 19, 2022
Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
Allogeneic Natural Killer Cell Therapy Given Go-Ahead for Solid Tumors
NKGen plans to initiate a phase 1 trial pd SNK02 in the first quarter of 2023.
Bridging the Gap: Education, Awareness Key to Translating Medical Innovation to Community Health Care in Rare Diseases
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
Clive Svendsen, PhD, on Unlocking the Potential of Astrocyte Cell Therapy
The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
T-Cell Therapy Recognized for Treating Pediatric Hepatoblastoma
The therapy is currently being evaluated in the phase 1/2 ARYA-1, ARYA-2, and ARYA-3 studies in adults and children.
Gracell Continues Progress in R/R B-Cell Acute Lymphoblastic Leukemia
The first patient has been dosed in a phase 2 study of the allogeneic CAR T-cell therapy GC007g.
Otoferlin-Related Hearing Loss Gene Therapy Gets IND Clearance
In a rodent model, durable auditory brainstem responses to sound were observed after DB-OTO administration.
Samir Parekh, MD, on Improving Survival in Multiple Myeloma With Sequential T-Cell Redirection Therapies
The director of translational research in myeloma at the Tisch Cancer Institute discussed positive findings, including a 30-month PFS in study participants.
Gene Therapy Restores Night Vision in 2 Patients With Leber Congenital Amaurosis
Future data from other participants with LCA enrolled in the phase 1/2 study will be announced at a later date.
Michel Michaelides, MD, FACP, on Gene Therapy's Potential in Retinitis Pigmentosa
The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.
CGTLive’s Weekly Rewind – October 14, 2022
Review top news and interview highlights from the week ending October 14, 2022.
