Videos

The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.

The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

The vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization also discussed the company’s planned presentation at ACR Convergence 2024.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the growing interest in gene therapy for attendees of the annual meeting.

The vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization discussed the Autoimmunity Cell Therapy Network.

The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.

The chief scientific officer and head of research, development, and Medical at Ocugen, discussed OCU400, the company’s gene-agnostic gene therapy for IRDs.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed several still-investigational drugs and therapies for DMD.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.

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

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.

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

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

The cofounder and chief science officer of Longeveron discussed working to show potential benefits in a follow-up phase 3 trial.

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

The chief medical officer at Creyon Bio discussed findings from a treated patient with a TNP02 missense mutation.

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.

The chief technology officer at GenSight Biologics discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.

The chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.

The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.

The cofounder and chief science officer of Longeveron discussed updated data from the phase 2a CLEAR MIND study.

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 Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.

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.