American Society of Gene & Cell Therapy (ASGCT)

Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa, discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.

Xandra Breakefield, PhD, an investigator of neurology at Mass General Research Institute, discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.

Lisa Nieland, a PhD candidate from Lieden University Medical Center and Breakefield Lab at Mass Gen, discussed her work presented at the ASGCT 2024 meeting.

Miloš Miljković, MD, the chief medical officer of Cartesian Therapeutics, discussed data presented at ASGCT 2024 from a phase 2a study.

David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed future applications for the company’s AI-guided discovery platform.

Scott Jeffers, PhD, the chief technology officer at GenSight Biologics, discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.

David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed findings from a patient treated for a TNP02 missense mutation.

David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed the development of an allele-selective ASO for a single patient with a de novo pathology.

The research scientist at Seattle Children’s Research Institute discussed findings from mouse research he presented at ASGCT’s 2024 conference.

Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio, discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.

David Suhy, PhD, the cofounder and chief scientific officer at Earli, discussed the company’s unique approach to cancer diagnosis.

Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics, discussed the company’s goal of bringing gene therapy to a much broader population of patients.

Janice Chen, PhD, the cofounder and chief technology officer of Mammoth Biosciences, discussed the importance of diverse approaches to gene editing to address a variety of indications.

The head of therapeutics at Epic Bio discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.

The chief scientific officer of Tome Biosciences also discussed the company’s pipeline for its new technology.

The chief scientific officer of Tome Biosciences discussed the company’s technologies in the context of integrative gene therapy and cell therapy.

The principal investigator at Seattle Children’s Research Institute discussed her lab’s work on using ultrasound mediated gene delivery to target hemophilia A.

The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research with UMGD for the treatment of hemophilia.

The cofounder and chief scientific officer of Mammoth Biosciences discussed the company’s mouse model research on treating hypertriglyceridemia.

The chief scientific officer of Caribou Biosciences discussed results from preclinical research evaluating the gene editing approach.

The chief scientific officer of Caribou Biosciences discussed the company’s platform for genome editing.

Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.

Faraz Ali, MBA, 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.

Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s research on capsids, promoters, and manufacturing improvements.

Erika Fullwood Augustine, MD, MS, 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.

The cofounder and chief scientific officer at Earli discussed the company’s unique approach to cancer diagnosis.

Sebastien Lefebvre, MSc, the vice president of R&D at Verdot, discussed the company's unique platform.

The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.

The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.

The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.