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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
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
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.
