The Importance of Precise Reproducibility of AAVs
Scott Jeffers, PhD, the chief technology officer at GenSight Biologics, discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.
A Promising Case Study of Ultra-Rare, AI-Guided, ASO Development
David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed findings from a patient treated for a TNP02 missense mutation.
AI-Guided ASO Development for Ultra-Rare Diseases
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.
Chun-Yu Chen, PhD, on Addressing Hemophilia A With CRISPR/Cas9 mRNA LNP Gene Editing
The research scientist at Seattle Children’s Research Institute discussed findings from mouse research he presented at ASGCT’s 2024 conference.
Evaluating Epigenetic Gene Therapy for the Treatment of FSHD
Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio, discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.
Using DNA Nanoplasmids as a Diagnostic Tool in Cancer
David Suhy, PhD, the cofounder and chief scientific officer at Earli, discussed the company’s unique approach to cancer diagnosis.
Bringing Gene Therapy to Common Diseases
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.
As Gene Editing Technology Advances, Numerous Potential Modalities Emerge
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.
Alexandra Collin de l’Hortet, PhD, on Evaluating Epigenetic Gene Therapy for the Treatment of FSHD
The head of therapeutics at Epic Bio discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.
David Dimmock, MBBS, on Accelerating Therapy Discovery and Approval With AI
The chief medical officer at Creyon Bio discussed future applications for the company’s AI-guided discovery platform.
John Finn, PhD, on Using Programmable Genomic Integration to Make Edits Big and Small
The chief scientific officer of Tome Biosciences also discussed the company’s pipeline for its new technology.
David Dimmock, MBBS, on a Promising Case Study of Ultra-Rare, AI-Guided, ASO Development
The chief medical officer at Creyon Bio discussed findings from a treated patient with a TNP02 missense mutation.
Scott Jeffers, PhD, on The Importance of Precise Reproducibility of AAVs
The chief technology officer at GenSight Biologics discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.
Michael Severino on In Vivo Gene Editing With RNA Gene Writers
The chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.
Chris Wright, MD, PhD, on Anelloviruses, a Potential Alternative to AAV for Gene Therapy
The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.
John Finn, PhD, on the Potential of Programmable Genomic Integration
The chief scientific officer of Tome Biosciences discussed the company’s technologies in the context of integrative gene therapy and cell therapy.
Carol Miao, PhD, on Using UMGD to Target Liver Sinusoidal Endothelial Cells
The principal investigator at Seattle Children’s Research Institute discussed her lab’s work on using ultrasound mediated gene delivery to target hemophilia A.
Carol Miao, PhD, on the Potential of Ultrasound Mediated Gene Delivery
The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research with UMGD for the treatment of hemophilia.
Lucas Harrington, PhD, on Using an Ultracompact CRISPR System to Target Cells Outside the Liver
The cofounder and chief scientific officer of Mammoth Biosciences discussed the company’s mouse model research on treating hypertriglyceridemia.
Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a Pain
The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.
Miloš Miljković, MD, on mRNA-CAR-T Descartes-08's Potential for Treating Myasthenia Gravis
The chief medical officer of Cartesian Therapeutics discussed data presented at ASGCT 2024 from a phase 2a study.
Steve Kanner, PhD, on Evaluating chRDNA Technology for ATTR and Familial Hypercholesterolemia
The chief scientific officer of Caribou Biosciences discussed results from preclinical research evaluating the gene editing approach.
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
The medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.
Steve Kanner, PhD, on Caribou’s chRDNA Technology
The chief scientific officer of Caribou Biosciences discussed the company’s platform for genome editing.
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
The PhD candidate from Lieden University Medical Center and Breakefield Lab at Mass Gen discussed her work presented at the ASGCT 2024 meeting.
Interest in Genomic Medicine Research for Heart Disease Continues to Grow
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
The senior researcher at Seattle Children’s discussed advantages of engineered Tregs compared with natural Tregs.
Gene Transfer Versus Gene Editing Approaches in Cardiology
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.
