Lysosomal Disorders

Latest News

Intellia CRISPR Gene Editing Therapy
November 9th 2025
Patient Treated in Trial for Intellia’s Transthyretin Amyloidosis Gene Editing Therapy Nex-Z Dies

According to Intellia, the FDA placed clinical holds on both MAGNITUDE and MAGNITUDE-2 on October 29, 2025.

CGTLive®’s Weekly Rewind
November 7th 2025

CGTLive®’s Weekly Rewind – November 7, 2025

November 5th 2025

Around the Helix: Cell and Gene Therapy Company Updates – November 5, 2025

November 3rd 2025

Data Roundup: October 2025 Features Updates from ESMO, ESGCT, and More

CGTLive®’s Weekly Rewind
October 31st 2025

CGTLive®’s Weekly Rewind – October 31, 2025

Latest Videos

Podcasts

Special Report Episode 10
Quarter Century Update: The Impact of Artificial Intelligence
Special Report Episode 9
Quarter Century Update: Thoughts on Reducing Costs of Goods
Special Report Episode 7
Quarter Century Update: Future Predictions for the Field
Special Report Episode 7
Quarter Century Update: What Does the Field Need Most Right Now?
Special Report Episode 6
Quarter Century Update: What Lessons Should Guide the Next Generation of Developers and Regulators?
Special Report Episode 5
Quarter Century Update: The Continued Unknowns of Cell and Gene Therapy
Special Report Episode 4
Quarter Century Update: How can Partnerships Between Academia, Biotech, and Pharma be Better Structured?
Special Report Episode 2
Quarter Century Update: Platform Approaches and Industry Investment
Special Report Episode 2
Quarter Century Update: Where are the Biggest Bottlenecks Today?
Special Report Episode 1
Quarter Century Update: What Were the Most Significant Inflection Points or Breakthroughs in the Field?

CME Content

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

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

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

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

Rami Komrokji, MD; Maen Hussein, MD

View more
Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition
In-Person + Virtual Event

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

December 5, 2025

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

View more
Medical Crossfire® Navigating the Chronic Myeloid Leukemia Landscape: Treatment Strategies, Mechanisms, and Patient-Centered Care
Virtual Event

Medical Crossfire® Navigating the Chronic Myeloid Leukemia Landscape: Treatment Strategies, Mechanisms, and Patient-Centered Care

December 15, 2025

View more
Evolving Strategies and Emerging Agents in PTCL: Subtypes, Mutations, and Modern Therapies
Virtual Event

Evolving Strategies and Emerging Agents in PTCL: Subtypes, Mutations, and Modern Therapies

December 19, 2025

View more
Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment
Multimedia

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Elias Jabbour, MD

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

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

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

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

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

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

View more
Navigating the Next Wave in BTK Inhibition Strategies: Maximizing Clinical Benefit in CLL and MCL, What’s Next in B-Cell Malignancies
Video

Navigating the Next Wave in BTK Inhibition Strategies: Maximizing Clinical Benefit in CLL and MCL, What’s Next in B-Cell Malignancies

Catherine C. Coombs, MD, MS; Paolo Ghia, MD, PhD; Tycel J. Phillips, MD; Jeffrey V. Matous, MD; Grzegorz S. Nowakowski, MD, FASCO

View more
SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients
Case-based Simulation

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

Amrita Krishnan, MD

View more
30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma
In-Person Event

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

February 26-28, 2026

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

View more
Upstate New York Hematology & Hematologic Oncology Conference: A Review of the 67th ASH Annual Meeting
In-Person + Virtual Event

Upstate New York Hematology & Hematologic Oncology Conference: A Review of the 67th ASH Annual Meeting

April 25-26, 2026

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

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

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

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

View more
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

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

View more
10th Annual Live Medical Crossfire®: Hematologic Malignancies
In-Person Event

10th Annual Live Medical Crossfire®: Hematologic Malignancies

July 11, 2026

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

View more
Imaging Matters: Decoding PSMA PET for Better Decision-Making
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

View more

More News
Contact Info

259 Prospect Plains Rd, Bldg H
Cranbury, NJ 08512

609-716-7777

HCP Live
Brand Logo

© 2025 MJH Life Sciences®

All rights reserved.

Home
About Us
News
Contact Us