Safe Handling Guidelines Needed for Gene Therapies in the Clinic
Current handling guidelines do not align between the laboratory and clinical settings.
Anjali Pradhan, MS, on Using Single Cell Sequencing for Gene Editing Quality Control
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
Liso-Cel/Breyanzi Approved for Relapsed/Refractory Mantle Cell Lymphoma
The CAR T-cell therapy was also recently approved for treating follicular lymphoma, chronic lymphocytic leukemia, and small lymphocytic lymphoma.
LCA2 Gene Therapy Shows Visual, Retinal Improvements at Low Doses
No serious adverse events or significant retinal atrophy occurred.
Brian Kim, MBA, on the Importance of Single Cell Analysis to Cell and Gene Therapy
The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.
Around the Helix: Cell and Gene Therapy Company Updates – May 29, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Ethical Framework Needed for Gene Therapy Clinical Trials
An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.
Wugen Garners New FDA and EMA Regulatory Designations for T-Cell Malignancy CAR-T WU-CART-007
The company also announced that it will be reporting new data at the European Hematology Association 2024 Hybrid Congress.
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.
Anixa Biosciences Makes Progress in the Clinical Evaluation of CER-T Approach to Treating Ovarian Cancer
The company announced that the fifth patient was treated in its phase 1 clinical trial.
Aurion Biotech Evaluating Combination Cell Therapy Product AURN001 as Alternative to Corneal Transplant in Phase 1/2 ABA-1, CLARA Clinical Trial
In honor of Healthy Vision Month, CGTLive® took a closer look at the clinical trial design for this novel treatment.
Cartesian Therapeutics’ Myasthenia Gravis mRNA CAR-T Descartes-08 Garners FDA RMAT Designation
The CAR-T product is currently being evaluated in the phase 2b portion of a clinical trial (NCT04146051) for MG.
CAR TEAM Cells Show Dramatic, Short-Lived Responses in Glioblastoma
Transient responses in 2 of 3 participants may be due to limited persistence of CARv3-TEAM-E T-cells in the weeks after infusion.
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.
CGTLive®’s Weekly Rewind – May 24, 2024
Review top news and interview highlights from the week ending May 24, 2024.
Cabaletta Bio’s CAR-T CABA-201 Shows Safety for First Patients Dosed in Myositis and Lupus Trials
The data comes from the phase 1/2 RESET-Myositis clinical trial (NCT06154252) and the phase 1/2 RESET-SLE clinical trial (NCT06121297).
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
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.
