Conference Coverage

9 days ago

ASGCT

Trends in RNA Therapeutic Research

9 days ago

ASGCT

Robert Alexander Wesselhoeft, PhD, on Broader Trends in the RNA Therapeutic Research Landscape

10 days ago

ASGCT

The Therapeutic Potential of Circular RNA

12 days ago

ASGCT

Robert Alexander Wesselhoeft, PhD, on the Therapeutic Potential of Circular RNA

14 days ago

ASGCT

Preclinical Evidence for PH1 Gene Therapy

15 days ago

ASGCT

John Murphy, PhD; and Tia DiTommaso, PhD, on Preclinical Evidence for PH1 Gene Therapy

19 days ago

ASGCT

CD19 CAR EngTregs: The Next Step in Autoimmune Disease Cell Therapy?

20 days ago

ASGCT

Annaiz Grimm, BS, on Using EngTregs to Treat Autoimmune Disease

29 days ago

ASGCT

AAV Capsids That Evade Neutralizing Antibodies

about 1 month ago

ASGCT

Damien Maura, PhD, on AAV Capsids That Evade Neutralizing Antibodies

Latest News

Sep 17, 2025 | News

Around the Helix: Cell and Gene Therapy Company Updates – September 17, 2025

Sanofi’s Neovascular Age-Related Macular Degeneration Gene Therapy SAR402663 Garners FDA Fast Track Designation

Sep 17, 2025

Eric Crombez, MD, the chief medical officer at Ultragenyx.

Ultragenyx's Glycogen Storage Disease Gene Therapy DTX401 Continues to Enable Decreases in Reductions in Daily Cornstarch Intake at 96 Weeks

Sep 16, 2025

ImmunoLogic, Episode 6: "The Future of Personalized Cancer Vaccines”

Sep 15, 2025

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

Sep 15, 2025

Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at GCTI

Robert Alexander Wesselhoeft, PhD, on Broader Trends in the RNA Therapeutic Research Landscape

Robert Alexander Wesselhoeft, PhD, on the Therapeutic Potential of Circular RNA

Tia DiTommaso, PhD, the senior director of preclinical pharmacology Arbor Biotechnologies, and John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies

John Murphy, PhD; and Tia DiTommaso, PhD, on Preclinical Evidence for PH1 Gene Therapy

Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute.

Annaiz Grimm, BS, on Using EngTregs to Treat Autoimmune Disease

Prerna Mewawalla, MD, medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network, as well as an associate professor at the Drexel University College of Medicine.

Prerna Mewawalla, MD, on Unmet Needs Following Early-Line CAR T for R/R Multiple Myeloma

Surbhi Sidana, MD, an assistant professor of medicine, bone marrow transplantation, and cellular therapy at Stanford

Surbhi Sidana, MD, on Cilta-Cel's Efficacy in Second-Line Relapsed/Refractory Multiple Myeloma

Damien Maura, PhD, a senior scientist at Voyager Therapeutics

Damien Maura, PhD, on AAV Capsids That Evade Neutralizing Antibodies

Rajeev Sivasankaran, PhD, vice president of neuroscience at Voyager Therapeutics

Rajeev Sivasankaran, PhD, on Voyager’s Single-Dose AAV Strategy for Silencing Tau for Alzheimer Disease

Sven Moller-Tank, PhD, a senior director at Regeneron

Sven Moller-Tank, PhD, on Regeneron’s Precision Approach to AAV Targeting

Alexey Danilov, MD, PhD, the Marianne and Gerhard Pinkus Professor of Early Clinical Therapeutics, medical director of the Early Phase Therapeutics Program for the Systems Clinical Trials Office, codirector of the Toni Stephenson Lymphoma Center, and a professor in the Division of Lymphoma in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope in Duarte, California

Alexey Danilov, MD, PhD, on CAR T and Bispecific Antibody Sequencing for DLBCL

Shorts

View All Shorts

1:02

Innovative Treatment Considerations for CAR-T Therapies

Stephen J. Schuster, MD, discussed the use of fractionated CAR-T in patients with progressive disease.

20 days ago

Stephen J. Schuster, MD, Abramson Cancer Center

0:47

The Importance of Nonmyeloablative Chemotherapy to Cell Therapy

Michael T. Lotze, MD, discussed one of the major advances enabling the practicality of cell therapy in oncology.

20 days ago

Michael T. Lotze, MD

0:25

Which Therapy Predicts a Better Response to TIL in Melanoma?

Michael T. Lotze, MD, quizzed Joseph Fraietta, PhD, on therapies that have predictive potential on outcomes for subsequent tumor infiltrating lymphocyte (TIL) therapy.

20 days ago

Michael T. Lotze, MD

0:58

Marcela Maus, MD, PhD on Autologous vs. Allogenic Therapies

Janna Minehart, MD, asks Marcela Maus, MD, PhD, for her most controversial opinion on CAR-T therapies.

20 days ago

Marcela V. Maus, MD, PhD(+1 more)

1:02

Unexpected Results in CAR T Cell Therapy Study in Glioblastoma

Marcela Maus, MD, PhD, discusses some of the exciting results of CAR-T therapy in a glioblastoma trial.

20 days ago

Marcela V. Maus, MD, PhD

1:30

The History and Evolution of CAR T Cell Therapy

Marcela Maus, MD, PhD, shares her knowledge on the history and evolution of CAR-T cell therapy.

20 days ago

Marcela V. Maus, MD, PhD

Podcasts

September 15, 2025

ImmunoLogic, Episode 6: "The Future of Personalized Cancer Vaccines”

Mind Moments, a podcast by NeurologyLive

September 7, 2025

Standardizing Gene Therapy Care in Duchenne: An Overview of Consensus Guidelines

July 30, 2025

ImmunoLogic, Episode 5: "Tumor Infiltrators and Triumphs: The Evolution of TIL Therapy" With Michael T. Lotze, MD

June 18, 2025

Cell Therapy in Parkinson Disease: Bemdaneprocel and What Lies Ahead

May 29, 2025

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

Videos

Nathan Yozwiak, PhD, the head of research at the GCTI

Nathan Yozwiak, PhD, on Fostering Collaboration in Cell and Gene Therapy Research

Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira, and Kilian Guse, PhD, the vice president of genetic medicine platforms at Pacira

Derek Jackson, BS, MA, and Kilian Guse, PhD, on High-Capacity Adenovirus Vectors for Gene Therapy

Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira

Derek Jackson, BS, MA, on Evaluating Gene Therapy PCRX-201 for Osteoarthritis of the Knee

Jeffrey Chamberlain, PhD, on the Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy

Tami John, MD, on β-Thalassemia Gene Therapy in the Context of the Broader World

Tami John, MD, on Considerations for β-Thalassemia Gene Therapy in the Real World

Tami John, MD, on Barriers to Access for Gene Therapy in β-Thalassemia

Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital

Matthew Ku, MBBS, PhD, FRACP, RACP, FRCPA/RCPA, on CAR T-Cell Therapy JNJ-90014496's Safety Profile in R/R LBCL

Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine

Saurabh Dahiya, MD, FACP, on CAR-T KITE-363's Safety Profile in R/R B-Cell Lymphoma

Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic

Shahzad Raza, MD, on Future Plans for CAR T-Cell Therapy NXC-201 in R/R Light Chain Amyloidosis

Continuing Medical Education

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

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!

All News

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

ByNoah Stansfield

September 14th 2025

In observance of Duchenne Action Month and World Duchenne Day, held annually in September, we took a look back at recent news in gene and cell therapy for DMD.

CGTLive®’s Weekly Rewind – September 12, 2025

ByNoah Stansfield

September 12th 2025

Review top news and interview highlights from the week ending September 12, 2025.

First Patient Treated in Capsida Biotherapeutics’ Trial for Encephalopathy Gene Therapy CAP-002 Passes Away

ByNoah Stansfield

September 11th 2025

Capsida has made the move to pause the trial voluntarily.

Some Patients Treated in Anixa's Ovarian Cancer CER T Trial Show Longer-Than-Expected Survival

ByNoah Stansfield

September 11th 2025

The company specifically highlighted that one patient who was treated in the trial’s first cohort is still alive at 28 months.

Patients Treated With REGENXBIO's MPS II Gene Therapy RGX-121 Sustain 82% Median Reduction in CSF HS D2S6 Levels Through 1 Year

ByNoah Stansfield

September 10th 2025

According to REGENXBIO, HS D2S6 is “a key biomarker of MPS II brain disease that is reasonably likely to predict clinical benefit.”

Around the Helix: Cell and Gene Therapy Company Updates – September 10, 2025

ByNoah Stansfield

September 10th 2025

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

Michael Flanagan, PhD, the chief scientific officer at Avidity

Patients With DMD Treated With Avidity Biosciences’ Antibody-Oligonucleotide Conjugate Del-Zota Show Functional Improvements

ByNoah Stansfield

September 10th 2025

The findings come from patients with DMD44 treated in the phase 1/2 EXPLORE44 clinical trial and the phase 2 EXPLORE44OLE clinical trial.

Linda Marbán, PhD, the CEO of Capricor Therapeutics

In Response to FDA’s Release of Full CRL for Deramiocel, Capricor Publishes Full Response to the Agency

ByNoah Stansfield

September 9th 2025

Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website.

Trends in RNA Therapeutic Research

ByRobert Alexander Wesselhoeft, PhD,Noah Stansfield

September 8th 2025

Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, expressed optimism for RNA therapy's future.

Robert Alexander Wesselhoeft, PhD, on Broader Trends in the RNA Therapeutic Research Landscape

ByRobert Alexander Wesselhoeft, PhD,Noah Stansfield

September 8th 2025

The director of RNA therapeutics at MGB’s Gene and Cell Therapy Institute expressed optimism for RNA’s potential role in CAR T-cell therapy, gene editing, and more.

Standardizing Gene Therapy Care in Duchenne: An Overview of Consensus Guidelines

ByBarry Byrne, MD, PhD,Marco Meglio

September 7th 2025

Mind Moments, a podcast from our sister site Neurology Live, held an exclusive interview with Barry Byrne, MD, PhD.

The Therapeutic Potential of Circular RNA

ByRobert Alexander Wesselhoeft, PhD,Noah Stansfield

September 7th 2025

Alex Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, discussed the Institute's work in exploring the new modality.