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Conference Archive

James Beck, PhD, chief scientific officer of the Parkinson’s Foundation, discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

Expanding Access to Whole Genome Sequencing in Parkinson Research

American Academy of Neurology (AAN)

The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.

Michel Michaelides, MD, FACP, on Gene Therapy's Potential in Retinitis Pigmentosa

American Academy of Ophthalmology (AAO)

The best response was very good partial response in 3 of 5 participants. 

Allogeneic CAR-T Yields Some Responses in Multiple Myeloma After Anti-BCMA Therapy 

American Association for Cancer Research (AACR)

American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM)

Mind Moments®, a podcast from our sister site NeurologyLive®, held an exclusive interview with Jonathan Parker, MD, PhD.

The Promise of Cell Therapy Approaches in Epilepsy

American Epilepsy Society (AES)

The life science research professional at Stanford University discussed preclinical research she presented at the American Heart Association’s Scientific Sessions 2023.

Eileen Tzng, BA, on Exploring Exosome and RNA-based Treatment Methods for Heart Failure

American Heart Association (AHA)

American Neurological Association (ANA)

Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed factors holding back more widespread adoption of newborn screening for indications treatable with gene therapy.

Gene Therapy and the Global Initiative for Newborn Screening

Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed the Institute’s efforts to bring about clinical translation of preclinical work.

Fostering Collaboration in Cell and Gene Therapy Research

American Society for Gene & Cell Therapy (ASGCT)

The internal medicine resident at SUNY Upstate Medical University Hospital discussed the implications of findings from a deidentified database of EMR data.

Deevyashali Parekh, MBBS, on Evaluating the Prevalence of Second Primary Malignancies After CAR T-Cell Therapy

American Society of Clinical Oncology (ASCO)

Mark Hamilton, MD, PhD, a hematology-oncology and BMT cell therapy fellow at Stanford University, discussed implications of his institution’s findings on treatment-related secondary malignancies.

Analyzing PostCAR Myeloid Neoplasms

American Society of Hematology (ASH)

Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.

Orchard Therapeutics’ Gene Therapy OTL-203 Stabilizes or Improves Sensory Symptoms in Patients With MPS-IH

Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT)

Sang Yoon Moon, PhD, postdoctoral Research Associate, Lions Eye Institute, discussed research with CRISPRa in human fibroblast cultures. 

Investigating CRB1 Mutations in Retinitis Pigmentosa 12  

Association for Research in Vision and Ophthalmology (ARVO)

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the foundation’s mission. 

Susan Ruediger on Pursuing Gene Therapies for CMT 

BIO International Convention

The chief development officer of Anixa Biosciences discussed the company’s trial of a CAR-T in ovarian cancer that is looking at the effect of regional administration and lymphodepletion on efficacy.

Pamela Garzone, PhD, on Exploring Unknowns in CAR-T for Solid Tumors

CAR-TCR Summit

The 3 FDA-approved CAR T-cell therapies showed comparable survival outcomes in DLBCL over a 3-year period.

Survival Outcomes Found to Be Comparable for Axi-Cel, Liso-Cel, and Tisa-Cel in Real-World DLBCL Data

European Hematology Association (EHA)

Updated 52-week follow-up data were presented at the EBMT meeting. 

T-cell Therapy Prevents Multiple Virus Infections after Allo-HSCT 

European Society for Blood and Marrow Transplantation (EBMT)

CARVac RNA vaccine may amplify the persistence of the BNT211 CAR T-cell therapy. 

CLDN6-Targeted CAR Shows Some Signs of Activity in Solid Tumors 

European Society for Medical Oncology Congress (ESMO)

Seven of 10 evaluable patients achieved minimal residual disease negativity after cilta-cel treatment.

Cilta-Cel Yields High MRD-Negativity Rates in Multiple Myeloma After Prior Anti-BCMA Treatment

International Myeloma Workshop (IMW)

Data from a phase 1/2 clinical trial of NRTX-1001 were presented at the ISSCR 2023 Annual Meeting. 

Inhibitory Interneuron Cell Therapy Reduces Seizures in First 2 Patients With MTLE 

International Society for Stem Cell Research (ISSCR)

A second cohort has been dosed with 7.7e11 vg/kg to hopefully confirm the dose for phase 3 trials. 

Freeline Moves Closer to Identifying Hemophilia B Gene Therapy Dose 

International Society on Thrombosis and Haemostasis (ISTH)

Linda Marbán, PhD, the CEO of Capricor Therapeutics, discussed future plans for the product after the promising data presented at MDA’s 2025 conference.

The Future of Capricor’s Cardiomyopathy Cell Therapy Deramiocel

Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval. 

Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks 

NORD Breakthrough Summit

The chief medical officer of Triumvira Immunologics discussed promising early results from the company’s phase 1/2 clinical trial. 

Deyaa Adib, MD, on Targeting HER2 in Solid Tumors With the TAC Platform 

Onco Cell Therapy Summit (OCTS)

Interim data from a phase 1 study from Intellia Therapeutics and Regeneron Pharmaceuticals is the first to support in vivo CRISPR genome editing in humans. 

CRISPR Gene Editing for Transthyretin Amyloidosis Safe, Effective

Peripheral Nerve Society (PNS)

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

Improving Rare Disease Awareness: Bruce Dezube, MD 

Rare Disease Day (RDD)

The median progression-free survival was 11 months and the SCG101 was well-tolerated. 

TCR-T Therapy Yields Some Responses in HBV-Related Hepatocellular Carcinoma 

Society for Immunotherapy of Cancer (SITC)

Alfred L. Garfall, MD, MS, discussed data from the phase 2 BMT CTN 1902 trial.


Ide-Cel and Lenalidomide Maintenance in MM Following Suboptimal ASCT Response

Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR 

The president of MAGIC Clinic and CEO of Discovery DNA discussed the implications of gene therapy data presented at the 2025 WORLDSymposium.

Aneal Khan, MD, MSc, FCCMG, FRCPC, on Evaluating Gene Therapy for Fabry Disease

WORLDSymposium

The cofounder and chief executive officer of NKILT Therapeutics discussed unique advantages of the CIR platform.