Lysosomal Disorders

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Florian Eichler, MD, on Promising Efficacy of AXO-AAV-GM2 in GM2 Gangliosidosis

Florian Eichler, MD, on How Novel Gene Therapy Delivery Improved Targeting in GM2 Gangliosidosis

Improvements in Developmental Milestones Observed With GM1 Gangliosidosis Gene Therapy

RP-A501 for Danon Disease: Gene Therapy Maintains Clinical Stability

"Low and Slow" vs "One and Done"? Unlocking the Potential of Gene Therapy

Improving Speed and Fidelity of CRISPR/Cas9: Kenneth Johnson, PhD, and David Taylor, PhD

Combining RNA and CAR T-Cell Therapy for Myasthenia Gravis

Gene Therapy for Rare Diseases: Latest Updates

Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD

Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD

CME Content

$New Horizons in Multiple Myeloma Treatment – Selection and Sequencing From Newly Diagnosed to Relapsed/Refractory Disease$

Video

New Horizons in Multiple Myeloma Treatment – Selection and Sequencing From Newly Diagnosed to Relapsed/Refractory Disease

Amrita Krishnan, MD; Suzanne Lentzsch, MD, PhD; Sagar Lonial, MD, FACP

Multimedia

Exploring the Dynamic Shifts in the MDS Treatment Paradigm: Unveiling Advances in Disease Modifying Therapies, Spotlight on Cutting-Edge Clinical Trials, and the Latest Approvals

Amer Zeidan, MBBS, MHS; Michael R. Savona, MD

Video

Medical Crossfire®: Uncovering The Clinical Utility of Combination Therapies and MRD Assessment in Newly Diagnosed Multiple Myeloma

Ajai Chari, MD; Adriana Rossi, MD; Anupama Kumar, MD

Multimedia

BURST Expert Illustrations and Commentaries™: Exploring the Mechanistic Rationale for CSF-1R– Directed Treatment in Chronic GVHD

Corey Cutler, MD, MPH, FRCP(C); Carrie L. Kitko, MD

Multimedia

Community Practice Connections™: 8th Annual School of Nursing Oncology™

Beth Sandy, MSN, CRNP, FAPO; Laura Wood, RN, MSN, OCN; Michael Lai, ARNP; Tiffany Richards, PhD, ANP-BC, AOCNP®; Lauren Verity Moore, DNP, MSN, AGACNP-BC; Megan Wanca, APRN, DNP, FNP-C

Case-based Simulation

SimulatEd™: Patient-Centered Care to Improve Outcomes in Chronic Lymphocytic Leukemia

Catherine C. Coombs, MD

Video

(COPE) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

Video

(CME) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

Multimedia

Community Practice Connections™: 6th Annual Precision Medicine Symposium – An Illustrated Tumor Board

Andre H. Goy, MD; Monique Hartley-Brown, MD, MMSc (Guest Co-Chair); Chiara Battelli, MD, PhD; Marios Giannakis, MD, PhD; Shail Maingi, MD; Aparna Parikh, MD

Video

Addressing Pivotal Updates in AML – Expert Perspectives on Individualizing Management for Special Populations

Eytan M. Stein, MD; Marina Kremyanskaya, MD, PhD; Hannah Levavi, MD

Video

Innovations in MDS Care: Navigating New Treatment Paradigms, Breakthrough Therapies, and Emerging Clinical Evidence

Rami Komrokji, MD; Maen Hussein, MD

In-Person + Virtual Event

Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition

December 5, 2025

Video

Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice

Craig Emmitt Cole, MD; Sikander Ailawadhi, MD; Irene Ghobrial, MD; Monique Hartley-Brown, MD, MMSc

Multimedia

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Elias Jabbour, MD

Video

How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Cristina Gasparetto, MD; Katja Weisel, MD

Video

Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET

Rami S. Komrokji, MD; Ghaith Abu-Zeinah, MD; Gabriela Hobbs, MD; Barbara Mora, MD

Video

From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice

Krina Patel, MD, MSc; Alfred L. Garfall, MD; Ajay K. Nooka, MD, MPH, FACP

Video

Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?

Corey Cutler, MD, MPH, FRCPC; Amin M. Alousi, MD; Yi-Bin Chen, MD; Carrie Lynn Kitko, MD

Video

Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead

Alexey V. Danilov, MD, PhD; Leidy L. Isenalumhe, MD, MS; Lori A. Leslie, MD; Mazyar Shadman, MD, MPH; Jason R. Westin, MD, MS, FACP

Video

Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios

Elias Jabbour, MD; Hagop M. Kantarjian, MD; Aaron Logan, MD, PhD, MPhil; Rob Pieters, MD, PhD; Claire Roddie, MD, PhD

Case-based Simulation

SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients

Amrita Krishnan, MD

In-Person Event

30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

February 26-28, 2026

Video

Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes

Jennifer R. Brown, MD, PhD; Alexey V. Danilov, MD, PhD; Matthew S. Davids, MD, MMSc

Multimedia

Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer

Hope S. Rugo, MD, FASCO; Neil M. Iyengar, MD; Komal Jhaveri, MD, FACP

Video

29th Annual International Congress on Hematologic Malignancies: Focus on Leukemias, Lymphomas, and Myeloma

André H. Goy, MD; Jorge E. Cortes, MD; Sagar Lonial, MD, FACP

Video

Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights

Gail J. Roboz, MD; Hagop Kantarjian, MD; Catherine Lai, MD, MPH; Amer Zeidan, MBBS, MHS

Video

Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

Video

Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches

Rami Komrokji, MD; Gabriela Hobbs, MD; John Mascarenhas, MD; Naveen Pemmaraju, MD

$Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma$

Video

Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

In-Person Event

10th Annual Live Medical Crossfire®: Hematologic Malignancies

July 11, 2026

Video

Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma

Amrita Krishnan, MD; Meletios Athanasios (Thanos) C. Dimopoulos, MD; Shaji Kumar, MD; Katja Weisel, MD

Multimedia

Imaging Matters: Decoding PSMA PET for Better Decision-Making

Jeremie Calais, MD, PhD; Tanya B. Dorff, MD; Scott T. Tagawa, MD, MS, FACP, FASCO

In-Person + Virtual Event

Go To PER in Chicago

On-Demand Sessions Available Now!

More News

Carolyn Riley Chapman, PhD, MS, Lead Investigator, Division of Global Health Equity (DGHE), Multi-Regional Clinical Trials (MRCT) Center, Department of Medicine, Brigham and Women’s Hospital, and Harvard Medical School

Ethical Framework Needed for Gene Therapy Clinical Trials

ByVictoria Johnson

May 29th 2024

An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.

Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials

ByZheng-Yi Chen, DPhil

May 24th 2024

The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.

CGTLive®’s Weekly Rewind – May 24, 2024

ByCGTLive Staff

May 24th 2024

Review top news and interview highlights from the week ending May 24, 2024.

Maria Escolar, MD, the chief medical officer of Forge Biologics

Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease

ByMaria Escolar, MD

May 22nd 2024

The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.

Around the Helix: Cell and Gene Therapy Company Updates – May 22, 2024

ByNoah Stansfield

May 22nd 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies

John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1

ByJohn Murphy, PhD

May 21st 2024

The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.

Maria Escolar, MD, on Continued Positive Results With Gene Therapy and UCBT in Krabbe Disease

ByMaria Escolar, MD

May 20th 2024

The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.

Harish Venkatesh, BS candidate, Department of Regulatory and Quality Sciences, University of Southern California, Los Angeles, California

Cell and Gene Therapy Trials Face Disproportionate Amount of Clinical Holds

ByVictoria Johnson

May 17th 2024

Oncological trials of biologics also face particularly higher rates of clinical holds.

CGTLive®’s Weekly Rewind – May 17, 2024

ByCGTLive Staff

May 17th 2024

Review top news and interview highlights from the week ending May 17, 2024.

Considerations for Gene Therapy Logistics and Implementation

ByVictoria Johnson

May 16th 2024

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.