Around the Helix: Cell and Gene Therapy Company Updates – March 23, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CAR T-Cell Therapy Promising in R/R Chronic Lymphocytic Leukemia
Seven patients from the trial continued to be followed-up as of the data cut-off date.
Combining RNA and CAR T-Cell Therapy for Myasthenia Gravis
Michael Singer, MD, PhD, cofounder and chief scientific officer of Cartesian Therapeutics, discussed the company’s lead candidate, Descartes-08.
Lineage Cell Therapeutics to Develop Cell Therapy for Hearing Loss
The company’s lead cell therapy candidate, OpRegen, has demonstrated efficacy in dry AMD.
Val-Rox Gene Therapy Restores Factor VIII Production and Reduces Bleeding in Hemophilia A
Complete results from the phase 3 GENEr8 study were published in the New England Journal of Medicine.
Research Needs in Pompe Disease: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.
Huntington Disease Gene Therapy AMT-130 Completes Trial Enrollment
A sister study of AMT-130 in Europe dosed its first patients in February.
Gene Therapy for Rare Diseases: Latest Updates
Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.
SQZ Biotech Awarded $2 Million to Develop Parkinson Disease Cell Therapy
The company recently received IND clearance for a solid tumor cell therapy.
Neutralizing Antibody Versus Total Antibody Assays in Gene Therapy Development: Key Considerations for Assessing Immunogenicity
Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.
Improving Diagnosis in Rare Diseases: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.
Adjuvant Olaparib Approved for BRCA+, High-Risk Early Breast Cancer
Olaparib is now the first early-line therapy targeting BRCA-mutations in breast cancer.
Improving Representation in Clinical Trials: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.
T-Cell Therapy Gets Orphan Drug Designation for AML
Half the patients in the high-dose cohort of a phase 1a/2b trial achieved complete responses.
CGTLive’s Weekly Rewind – March 18, 2022
Review top news and interview highlights from the week ending March 18, 2022.
Addressing AAV Antibodies in Hemophilia B Gene Therapy: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.
Mesenchymal Stem Cell Therapy IND Cleared for Crohn Disease Fistulas
Another MSC therapy being developed by Mesoblast demonstrated efficacy earlier in March.
Mesenchymal Stem Cell Therapy Improves Joint Function in Rheumatoid Arthritis
Significant decreases were seen in swollen and tender joint measures.
$12 Million Grant Awarded for Hemophilia A Gene Therapy Research
The grant, given to Indiana University School of Medicine, will fund 3 major projects to improve different aspects of hemophilia gene therapy.
Proposed FDA Gene Editing Guidance Emphasizes Long-Term Safety Evaluations
The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.
Around the Helix: Cell and Gene Therapy Company Updates – March 16, 2022
2-year Data for Duchenne Muscular Dystrophy Gene Therapy Shows Durable Improvements
Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.
EMBARK Study Evaluates DMD Gene Therapy
Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.
Adrenomyeloneuropathy Gene Therapy Gets Orphan Drug Designation
The new designation follows an IND clearance and fast track review in February 2022.
First Patient Dosed in T-Cell Malignancy CAR T-Cell Therapy Trial
WU-CART-007 is the first clinical-stage allogeneic CAR therapy for T-cell malignancies in the US.
FDA Recognizes Canavan Disease Gene Therapy With Multiple Designations
Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.
Presymptomatic SMA Treatment With Zolgensma Associated With Normal Development
New data presented at MDA 2022 showcased positive motor and bulbar function data.
Gene Therapy Potential in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.
Urovant Gene Therapy Yields Improvements in Overactive Bladder
Further results will be presented at the American Urological Association Annual Meeting, May 13-16, 2022.
Epstein-Barr Virus and Autoimmune Disease: Jakob Dupont, MD
The global head of research and development at Atara Biotherapeutics discussed the future of MS research.
