Videos

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.

The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed potential effects of cilta-cel's approval.

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed the impact of CAR T-cell therapies in patients with lymphoma.

The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day.

Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform.

The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.

David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.

The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.