
The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

The assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.

The director of the leukodystrophy service at Mass Gen discussed the phase 1/2 study of AXO-AAV-GM2.

Principal investigator Barry Greenberg, MD, FHFSA, shares extended results from the phase 1 trial and also preliminary pediatric safety results in a presentation at ASGCT 2022.

At ASCGT 2022, Italian investigators report updated safety and efficacy data from a phase 1/2 trial, in which all evaluable subjects achieved platelet transfusion independence and reported improved median platelet counts.

Alexander I. Spira, MD, PhD, FACP, research institute director with Virginia Cancer Specialists Research Institute, discusses the examination of RTX-240 in a phase 1 trial (NCT04372706) with solid tumors, presented recently at the 2022 AACR Annual Meeting.

Tim Lu, MD, PhD, CEO and co-founder of gene circuit company Senti Bio, joins CGTL to discuss his company's approach to creating "smarter" therapies, including its CAR-NK platform.

Radek Špíšek, PhD, co-founder and chief executive officer of SOTIO Global, and Geoffrey Hodge, chief executive officer of SOTIO BioTech US, join CGTL to discuss its 3 technology platforms and how they are leveraging them to attack the solid tumor micro-environment.

Rami Elghandour, chairman and CEO of Arcellx, shares updates on the phase 1 trial of CART-ddBCMA for the treatment of subjects with relapsed and refractory multiple myeloma.

The CEO and managing director of Chimeric Therapeutics hopes to contribute to the narrative with a handful of offerings in development across 2 platforms: CORE-NK, which uses allogeneic natural killer cells, and T-cell derived autologous therapies.

Lexeo recently announced FDA clearance of its investigational new drug application for LX2006 for Friedreich's ataxia cardiomyopathy.

The real-world data support the recently published results from a phase 3 study, which showed most patients achieved transfusion independence.

In this late-breaking poster presented at the Tandem Meetings, investigators recommend that patients with primary or secondary central nervous system lymphoma be included in future clinical trials for CAR T-cell therapy.

Sebastian Wanless, MD, PhD, senior clinical director of TC BioPharm, joins CGTL to discuss the company's development of chimeric co-stimulatory receptor T cells, as well as its clinical programs targeting cancers and infectious diseases.

Ruhong Jiang, PhD, cofounder, president, and chief executive officer of ASC Therapeutics, joins CGTL to discuss the company's AAV8-based gene therapy for patients with hemophilia A, as well as other pipeline activity.

Matt Killeen, PhD, chief scientific officer at Renovacor, shares updates on the company's lead candidate, REN-001, an adeno-associated viral vector serotype 9-based gene therapy for the treatment of BAG3-associated dilated cardiomyopathy.

Carsten Brunn, PhD, president and chief executive officer of Selecta Biosciences, joins CGTL to discuss ImmTOR, an immune tolerance platform that could ultimately enable gene therapy redosing in certain cases.

David Apelian, MD, PhD, MBA, chief executive officer at BlueSphere Bio; and Warren Shlomchik, MD, cofounder and scientific advisory board chairman at BlueSphere Bio, join CGTL to discuss the company's leading T-cell therapy clinical program.

The chief executive officer of Graphite Bio discusses the phase 1/2 CEDAR trial of GPH101, an investigative Cas9-edited autologous CD34+ cell therapy for the treatment of sickle cell disease.

Erandi De Silva, PhD, co-founder and senior vice president of product development at Forge Biologics, discusses the dual approach of her company in manufacturing and also developing adeno-associated viral (AAV) -based gene therapies, including FBX-101 for Krabbe disease.

Peter J. McAllister, MD, FAAN, presented promising data from the phase 2 STEMTRA trial at AAN 2022.

The professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms.

Michael Singer, MD, PhD, cofounder and chief scientific officer of Cartesian Therapeutics, discussed the company’s lead candidate, Descartes-08.

The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.

Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.

The global head of research and development at Atara Biotherapeutics discussed the future of MS research.