Lysosomal Disorders

Latest News

Latest Videos

David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases

Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth

Sowmya Viswanathan, PhD, on Translating Cell Therapies to the Clinic at ISCT 2024

Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility

Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary

Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology

Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy

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

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

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

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

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

CME Content

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!

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

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

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

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®: 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

More News

Around the Helix: Cell and Gene Therapy Company Updates – June 18, 2025

ByNoah Stansfield

June 18th 2025

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

CGTLive®’s Weekly Rewind – June 13, 2025

ByNoah Stansfield

June 13th 2025

Review top news and interview highlights from the week ending June 13, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – June 11, 2025

ByNoah Stansfield

June 11th 2025

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

CGTLive®’s Weekly Rewind – June 6, 2025

ByNoah Stansfield

June 6th 2025

Review top news and interview highlights from the week ending June 6, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – June 4, 2025

ByNoah Stansfield

June 4th 2025

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

CGTLive®’s Weekly Rewind – May 30, 2025

ByNoah Stansfield

May 30th 2025

Review top news and interview highlights from the week ending May 30, 2025.

ImmunoLogic, Episode 4: "Inflammatory Storms and Gentle Balances: Navigating Toxicities in Immunotherapies" With Caroline Diorio, MD, FRCPC, FAAP

ByJoseph A. Fraietta, PhD,Janna Minehart, MD,Caroline Diorio, MD, FRCPC, FAAP,Noah Stansfield,Matt Hoffman

May 29th 2025

In Episode 4 of ImmunoLogic, Caroline Diorio, MD, FRCPC, FAAP, discussed her research on adverse events associated with cell therapy.

Around the Helix: Cell and Gene Therapy Company Updates – May 28, 2025

ByNoah Stansfield

May 28th 2025

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

CGTLive®’s Weekly Rewind – May 23, 2025

ByNoah Stansfield

May 23rd 2025

Review top news and interview highlights from the week ending May 23, 2025.

Kiran Musunuru, MD, PhD, a physician-scientist and Barry J. Gertz Professor for translational research in the Perelman School of Medicine at the University of Pennsylvania and CHOP

The Implications of the First Successful Personalized Gene Editing Therapy

ByKiran Musunuru, MD, PhD, MPH, ML, MRA,Rebecca Ahrens-Nicklas, MD, PhD,Noah Stansfield

May 22nd 2025

Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed long-term future expectations for personalized gene editing therapy.

Around the Helix: Cell and Gene Therapy Company Updates – May 21, 2025

ByNoah Stansfield

May 21st 2025

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

Rebecca Ahrens-Nicklas, MD, PhD, a physician-scientist and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP

The Next Steps for Personalized Gene Editing

ByRebecca Ahrens-Nicklas, MD, PhD,Kiran Musunuru, MD, PhD, MPH, ML, MRA,Noah Stansfield

May 21st 2025

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, physician-scientists at CHOP, discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

Treating CPS1 Deficiency With Personalized Gene Editing

ByKiran Musunuru, MD, PhD, MPH, ML, MRA,Rebecca Ahrens-Nicklas, MD, PhD,Noah Stansfield

May 20th 2025

Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Implications of the First Successful Personalized Gene Editing Therapy

ByKiran Musunuru, MD, PhD, MPH, ML, MRA,Rebecca Ahrens-Nicklas, MD, PhD,Noah Stansfield

May 17th 2025

The physician-scientists at CHOP discussed long-term future expectations for personalized gene editing therapy.

CGTLive®’s Weekly Rewind – May 16, 2025

ByNoah Stansfield

May 16th 2025

Review top news and interview highlights from the week ending May 16, 2025.

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Next Steps for Personalized Gene Editing

ByKiran Musunuru, MD, PhD, MPH, ML, MRA,Rebecca Ahrens-Nicklas, MD, PhD,Noah Stansfield

May 16th 2025

The physician-scientists at CHOP discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

REGENXBIO’s BLA for MPS II Gene Therapy RGX-121 Accepted for Priority Review by the FDA

ByNoah Stansfield

May 15th 2025

The PDUFA action date for the BLA has been set as November 9, 2025.

Rebecca Ahrens-Nicklas, MD, PhD; and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on Treating CPS1 Deficiency With Personalized Gene Editing

ByKiran Musunuru, MD, PhD, MPH, ML, MRA,Rebecca Ahrens-Nicklas, MD, PhD,Noah Stansfield

May 15th 2025

The physician-scientists at CHOP discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

Around the Helix: Cell and Gene Therapy Company Updates – May 14, 2025

ByNoah Stansfield

May 14th 2025

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

Aneal Khan MD, MSc, FCCMG, FRCPC, president of MAGIC Clinic and CEO of Discovery DNA

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

ByAneal Khan MD, MSc, FCCMG, FRCPC,Noah Stansfield

May 12th 2025

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

CGTLive®’s Weekly Rewind – May 9, 2025

ByNoah Stansfield

May 9th 2025

Review top news and interview highlights from the week ending May 9, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – May 7, 2025

ByNoah Stansfield

May 7th 2025

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

Clinical Stage Biotech Bloomsbury Genetic Therapies Shuttering its Doors

ByNoah Stansfield

May 6th 2025

The company’s CEO announced via LinkedIn that the company is shutting down its operations, citing a “difficult funding environment.”

CGTLive®’s Weekly Rewind – May 2, 2025

ByNoah Stansfield

May 2nd 2025

Review top news and interview highlights from the week ending May 2, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – April 30, 2025

ByNoah Stansfield

April 30th 2025

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

Lysosomal Disorders

Latest News

Latest Videos

David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases

Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth

Sowmya Viswanathan, PhD, on Translating Cell Therapies to the Clinic at ISCT 2024

Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility

Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary

Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology

Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy

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

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

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

CME Content

Imaging Matters: Decoding PSMA PET for Better Decision-Making

Go To PER in Chicago

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

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

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

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

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

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

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

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

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

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

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

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

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

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

10th Annual Live Medical Crossfire®: Hematologic Malignancies

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

More News

Around the Helix: Cell and Gene Therapy Company Updates – June 18, 2025

CGTLive®’s Weekly Rewind – June 13, 2025

Around the Helix: Cell and Gene Therapy Company Updates – June 11, 2025

CGTLive®’s Weekly Rewind – June 6, 2025

Around the Helix: Cell and Gene Therapy Company Updates – June 4, 2025

CGTLive®’s Weekly Rewind – May 30, 2025

ImmunoLogic, Episode 4: "Inflammatory Storms and Gentle Balances: Navigating Toxicities in Immunotherapies" With Caroline Diorio, MD, FRCPC, FAAP

Around the Helix: Cell and Gene Therapy Company Updates – May 28, 2025

CGTLive®’s Weekly Rewind – May 23, 2025

The Implications of the First Successful Personalized Gene Editing Therapy

Around the Helix: Cell and Gene Therapy Company Updates – May 21, 2025

The Next Steps for Personalized Gene Editing

Treating CPS1 Deficiency With Personalized Gene Editing

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Implications of the First Successful Personalized Gene Editing Therapy

CGTLive®’s Weekly Rewind – May 16, 2025

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Next Steps for Personalized Gene Editing

REGENXBIO’s BLA for MPS II Gene Therapy RGX-121 Accepted for Priority Review by the FDA

Rebecca Ahrens-Nicklas, MD, PhD; and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on Treating CPS1 Deficiency With Personalized Gene Editing

Around the Helix: Cell and Gene Therapy Company Updates – May 14, 2025

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

CGTLive®’s Weekly Rewind – May 9, 2025

Around the Helix: Cell and Gene Therapy Company Updates – May 7, 2025

Clinical Stage Biotech Bloomsbury Genetic Therapies Shuttering its Doors

CGTLive®’s Weekly Rewind – May 2, 2025

Around the Helix: Cell and Gene Therapy Company Updates – April 30, 2025

Trending on CGTlive®

Top News in Lymphoma Cell Therapy for World Lymphoma Awareness Day 2025

Duchenne Action Month 2025: Looking Back at News and Expert Insights