Research Needs in Pompe Disease: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.
Gene Therapy for Rare Diseases: Latest Updates
Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.
Improving Diagnosis in Rare Diseases: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.
Improving Representation in Clinical Trials: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.
Addressing AAV Antibodies in Hemophilia B Gene Therapy: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.
Gene Therapy Potential in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.
Epstein-Barr Virus and Autoimmune Disease: Jakob Dupont, MD
The global head of research and development at Atara Biotherapeutics discussed the future of MS research.
Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD
The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD
Improving Access to CAR T-Cell Therapies: Thomas G. Martin, MD
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed potential effects of cilta-cel's approval.
Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD
The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.
Cautious Optimism for GM1 Gangliosidosis Gene Therapy: David Weinstein, MD
The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.
Improving Access to CAR T-Cell Therapies in Lymphoma: Nilanjan Ghosh, MD, PhD
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed the impact of CAR T-cell therapies in patients with lymphoma.
Global Awareness, Diagnosis a Priority for Rare Diseases: Michael Parini, JD
The chief executive officer and director of Freeline Therapeutics discussed the importance of Rare Disease Day.
Advantages of Invariant Natural Killer T Cell Therapies
Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform.
Working to Find a Cure for Charcot-Marie-Tooth: Keith Fargo, PhD
The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.
New Findings May Help Bolster CRISPR/Cas9 Accuracy
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.
Improving Rare Disease Awareness: Bruce Dezube, MD
The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.
Determining Order of CAR T Therapy: Elias Jabbour, MD; Jae Park, MD
The oncologists from MD Anderson and Memorial Sloan Kettering Cancer Centers discuss sequencing CAR T-cell therapies and other key therapies in patients with B-cell acute lymphoblastic leukemia.
Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.
A Positive Outlook for Rare Disease Research: Michael Singer, MD, PhD
The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.
Improving Rare Disease Awareness: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.
Developing Therapies for Rare Diseases: Kinnari Patel, PharmD
The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.
Improving Rare Disease Awareness: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.
Ongoing Gene Therapy Trials in Stargardt Disease : Prof. Carel Hoyng
The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
Launching PDGENEration to Advance Genetic Testing in Parkinson Disease: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.
Managing Safety in Fitusiran Treatments of Hemophilia: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.
Unmet Needs in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.
Assessing Safety of Fibroblast Cell Therapy in Multiple Sclerosis: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.
Risk Alleles in Age-Related Macular Degeneration: Steffen Schmitz-Valckenberg, MD
The ophthalmologist from John A. Moran Eye Center discussed the close association between disease progression and uncovered risk alleles.
Targeting Unmet Needs in Rare Dermatologic Diseases With Gene Therapy
Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.
