Susan Ruediger


The Power of Partnership in CMT Gene Therapy Research

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the origins of the CMTRF.

Ami J. Shah, MD


Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene-Edited Cell Therapy in PKD

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

Stanley Frankel, MD


Selecting Cell Therapy Targets in Cancer

The chief executive and medical officers of Cytovia Therapeutics discussed the company's therapeutic targets and cell therapy scaffold.

Vlad Hogenhuis, MD, MBA


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.

Kenneth Anderson, MD


Dr. Anderson on Addressing Unmet Needs in Myeloma With Descartes-11

Kenneth C. Anderson, MD, discusses potentially addressing unmet needs in patients with multiple myeloma through the use of the mRNA-based CAR T-cell therapy Descartes-11.

Jason Fontenot, PhD


Jason Fontenot, PhD, on Overcoming the Challenge of Delivery for Zinc Finger Epigenetic Repressors

The chief scientific officer of Sangamo Therapeutics discussed the company’s AAV capsid evolution research.

Katie Robinson


CARsgen Therapeutics’ CT041 Shows Promise in Advanced Gastric/Gastroesophageal Junction Adenocarcinoma

The chimeric antigen receptor-modified autologous T cells targeting CLDN18.2 had a manageable safety/tolerability profile and promising efficacy.

Eric Crombez, MD


Addressing Unmet Needs With Gene Therapy

Eric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.

Aimee Donald, MBChB, PhD


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.

Hamid Khoja, PhD


Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhD

The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.

Daniel Teper, PharmD, MBA


Selecting Cell Therapy Targets in Cancer

The chief executive and medical officers of Cytovia Therapeutics discussed the company's therapeutic targets and cell therapy scaffold.

Donald Kohn, MD


Donald Kohn, MD, on the Trajectory of Gene Therapy

The renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.

Jeffrey S. Chamberlain, PhD


Jeffrey S. Chamberlain, PhD, on Collaborating to Cure DMD

The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.

Neil Weinreb, MD


Challenges With Conducting Clinical Trials in Gaucher Disease: Neil Weinreb, MD

The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.

GeneTherapyLive Staff


CGTLive’s Weekly Rewind – April 8, 2022

Review top news and interview highlights from the week ending April 8, 2022.

Jan ter Meulen, MD, PhD


Developing Controllable Tumor Infiltrating Lymphocyte Therapies

Jan ter Meulen, MD, PhD, chief scientific officer, Obsidian Therapeutics, discussed the potential of the cytoDRIVE platform.

Breanna DiAndreth, PhD


Breanna DiAndreth, PhD, on Improving Selective Targeting of Tumor Cells

The scientist at A2 Biotherapeutics discussed the company’s dual-receptor Tmod technology.

James Hoffman, MD


Looking Forward in Relapsed/Refractory Multiple Myeloma Treatments

James Hoffman, MD, leads a discussion about recently approved therapies and exciting future treatment options for relapsed/refractory multiple myeloma.

Jennifer Kwon, PhD


Jennifer Kwon, PhD, on Reducing LDL Cholesterol in NHPs With Epigenome Editing

The senior scientist at Tune Therapeutics discussed preclinical research presented at the ASGCT 2023 meeting.

Scott Requadt


Tackling Deceased Kidney Transplant and Severe Scleroderma With Cell Therapy

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the phase 2 FREEDOM-3 trial and preclinical studies of the cell therapy FCR-001.

Colleen Hall


Gene Editing's Potential for COVID-19 and Other Indications

New human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.

Vishal Patel, MD


Vishal Patel, MD, on the Potential Advantages of a Gene Therapy Approach for Polycystic Kidney Disease

Patel discussed the potential advantages of his team’s gene therapy approach compared to an investigational PKD drug.

Olga Uspenskaya, MD, PhD


Working Toward a Single Dose Gene Therapy for Type 1 Gaucher Disease

Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.

Ilan Ganot


Unique Challenges With Gene Therapy in Duchenne Muscular Dystrophy

The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed challenges in developing gene therapies for DMD.

Tianhang Luo, MD


Weijia Fang, MD, and Tianhang Luo, MD, on EpCAM-Targeted CAR-T Therapy in Advanced Colorectal and Gastric Cancer

Fang recently presented at the European Society for Medical Oncology (ESMO) 2022 Congress.

Carol Miao, PhD


Carol Miao, PhD, on Delivering Gene Editing Tools With Nonviral Methods in Hemophilia A Models

The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research on nonviral delivery methods for gene editing tools in the context of treating hemophilia A.

Muhammad Bilal Abid, MD, MRCP


Optimizing COVID-19 Vaccines in Patients Receiving CAR T Therapy: Muhammad Bilal Abid, MD, MRCP

The assistant professor of medicine at the Medical College of Wisconsin discussed immune-compromising factors that are indigenous to CAR T-cell therapy recipients.

Gavin Macbeath, PhD


Gavin Macbeath, PhD, on Screening for Off-Target Effects

The chief scientific officer at TScan Therapeutics discussed the company’s SafetyScan technology.

José-Alain Sahel, MD


Gene Therapy Optogenetics Combination Efficacious in Retinitis Pigmentosa: José-Alain Sahel, MD

The professor from University of Pittsburgh School of Medicine discussed the combination of an optogenetics device and gene therapy in treatment of RP.

Rick Fair


Developing Safer, Next-Generation CAR T-Cell Therapies

Rick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s programs and future plans.

© 2023 MJH Life Sciences

All rights reserved.