CAR T Therapy Shows Some Significant Responses in High Grade Glioma
Fifty percent of patients had stable disease or better, with 2 complete responses and 2 partial responses.
Janice Chen, PhD, on Novel Approaches to Gene Editing
The cofounder and chief technology officer of Mammoth Biosciences discussed the importance of diverse approaches to gene editing to address a variety of indications.
Manipulation of the FOXO1 Gene Holds Potential to Enhance CAR T-Cell Fitness
Evan Weber, PhD, an assistant professor of pediatrics at Children's Hospital of Philadelphia, discussed his work on the role of the FOXO1 gene in T-cell persistence and exhaustion.
Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing Therapies
The cofounder and chief executive officer of Verve Therapeutics discussed the company’s pipeline.
B-VEC Eyedrops Improve Vision in Ocular DEB Case Study
B-VEC is approved in a gel formulation under the name Vyjuvek for skin wounds in dystrophic epidermolysis bullosa.
Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease
The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.
Around the Helix: Cell and Gene Therapy Company Updates – May 22, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Ocugen Gene Therapies Progress in Stargardt, Retinitis Pigmentosa, and Geographic Atrophy
Ocugen’s gene agnostic modifier gene therapies include OCU400 and OCU410.
John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1
The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.
Atara Submits BLA for Tab-Cel EBV+ PTLD T-Cell Therapy
Tab-Cel is approved under the name Ebvallo in Europe, Switzerland, and United Kingdom.
CRISPR-Editing EBT-101 Therapy Safe, Temporarily Suppresses HIV Infection
Excision is evaluating a higher dose in a second cohort as well as exploring alternative, potentially redosable, delivery methods.
Erika Fullwood Augustine, MD, MS, on Optimizing End Points for Rare Disease Clinical Trials
The associate chief science officer of the Kennedy Krieger Institute discussed an aspect of clinical trial design highly relevant to gene therapy development for rare diseases.
NKGen’s Autologous NK Therapy Proceeds to Phase 2 Trial for Alzheimer Disease
Most participants had improved or stable cognitive function in a proof-of-concept study.
Maria Escolar, MD, on Continued Positive Results With Gene Therapy and UCBT in Krabbe Disease
The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.
End-to-End Thinking is Key to Overcoming Friction in Gene Therapy Development
Abhishek Gupta, BS, the senior vice president of genetic medicines at Syneos Health, discussed common setbacks in gene therapy trials and how to overcome them.
Cell-Based Quadrivalent Influenza Vaccine Displays Superiority Over 3 Flu Seasons
A study of the flu seasons from 2017 to 2020 suggest that cell-based influenza vaccines outperformed egg-based vaccines in effectiveness.
A2 Bio’s Trial for Solid Tumor Tmod CAR-T A2B694 Doses First Patient
A2B694 is being evaluated for the treatment of patients with mesothelin-expressing solid tumors that have lost HLA-A*02 expression.
Casey Maguire, PhD, on Potential Advantages of Encapsulated AAVs
The associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.
Working to Enable Gene Therapy for Cardiovascular Disease
Jonathan W. Weinsaft, MD, chief of cardiology and professor of medicine at Weill Cornell Medical College, discussed research the center is doing in the space.
Faraz Ali, MBA, on Trends in Precision Medicine for Cardiovascular Disease
The chief executive officer of Tenaya Therapeutics discussed the growing interest in genomic medicines in cardiology.
Cell and Gene Therapy Trials Face Disproportionate Amount of Clinical Holds
Oncological trials of biologics also face particularly higher rates of clinical holds.
CGTLive®’s Weekly Rewind – May 17, 2024
Review top news and interview highlights from the week ending May 17, 2024.
Otoferlin-Related Hearing Loss Gene Therapy May Open the Door to Gene Therapies for Other Types of Hearing Loss
Lawrence R. Lustig, MD, discussed promising early results from the phase 1/2 CHORD trial evaluating Decibel Therapeutics and Regenerons’ DB-OTO.
Next ACGT Grant to Focus on Cell and Gene Therapy for Pancreatic Cancer
The invitation-only annual scientific summit, held on March 29, in New York City, brought together top scientists in cancer cell and gene therapy.
Verismo Therapeutics’ CAR-T SynKIR-310 Cleared for Trial in R/R B-NHL
Verismo intends to go forward with plans for the phase 1 CELESTIAL-301 clinical trial, which the company expects to initiate in the second half of this year.
Faraz Ali, MBA, on Adding Gene Editing Programs to Tenaya’s Cardiovascular Disease Pipeline
The chief executive officer of Tenaya Therapeutics discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.
Considerations for Gene Therapy Logistics and Implementation
Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.
Liso-Cel/Breyanzi Gets Accelerated Approval for Relapsed/Refractory Follicular Lymphoma
The CAR T-cell therapy was also recently approved for treating chronic lymphocytic leukemia or small lymphocytic lymphoma.
AADC Deficiency Gene Therapy up For FDA Priority Review
Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.
Around the Helix: Cell and Gene Therapy Company Updates – May 15, 2024
