News

Obe-cel Shows Complete Remissions up to 3 Years in R/R B-ALL

December 13th 2023

Marcela Maus, MD, PhD, Associate Professor of Medicine at Harvard Medical School discussed innovative new research in the field and in her own lab.

Obe-cel Yields Long Term Complete Remissions in R/R B-ALL

Jay Spiegel, MD, FRCPC, on 5-Year Real-World Outcomes From Axi-cel in R/R LBCL

December 13th 2023

A pooled analysis of the ALLCAR19 and FELIX trials including patients with R/R B-ALL, B-CLL, and B-NHL were presented at ASH 2023.

Jay Spiegel, MD, FRCPC

Steven W. Pipe, MD, on Data Continuing to Support Etranacogene Dezaparvovec Over SOC in Hemophilia B

The hematologist and cell therapist from the University of Miami Sylvester Comprehensive Cancer Center spoke about long-term outcome data he presented at ASH’s 2023 conference.

Steven W. Pipe, MD

Novel BCMA-Directed CAR Anito-Cel Is Highly Effective for Myeloma

The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed 3-year follow-up data from the HOPE B trial of the approved gene therapy, branded as Hemgenix.

Scott McClellan, MD, on Overcoming Limitations of AlloHSCT With Novel Cell Therapies

BCMA-directed CAR T-cell therapy anitocabtagene autoleucel demonstrated a 76% complete remission rate and 89% negative minimal residual disease in patients with relapsed/refractory multiple myeloma.

Scott McClellan, MD

Etranacogene Dezaparvovec Shows Durable Activity and Reduced Consumption of Factor IX in Hemophilia B After 3 Years

The chief medical officer of Orca Bio discussed data the company presented on its Orca-T and Orca-Q programs at ASH 2023.

Piers Blombery, MBBS, PhD, on Investigating CAR+ T-Cell Lymphoma After Cilta-Cel

The CSL Behring/uniQure gene therapy produced significant reductions in annualized bleeding rates and the number of total bleeds, with a consistent safety profile and no new adverse events.

Piers Blombery, MBBS, PhD

Etranacogene Dezaparvovec Demonstrates Safety and Efficacy in Patients With a History of Chronic HCV/HBV

The associate professor at Peter MacCallum Cancer Centre discussed case study research from the center and possible contributions to the case.

Liso-cel Shows Continued Durability in Long-Term Follow-Up Data of r/r CLL/SLL

No significant differences were observed between patients with and without HCBV in the reductions seen in ABR for all bleeds during the 7 to 36 months posttreatment timeframe.

Franck Morschhauser, MD, PhD, on Encouraging Data on Second Line Liso-Cel for Follicular Lymphoma

Among patients with chronic lymphocytic leukemia and small lymphocytic lymphoma, the Bristol Myers Squibb CAR T cell product showed consistent efficacy in patients with a lack of success with BTK inhibitor and venetoclax treatment.

Franck Morschhauser, MD, PhD

Akshay Sharma, MBBS, on Ongoing Challenges of Using Patient Reported Outcomes in Sickle Cell Disease

The professor of hematology at University of Lille and chair of LYSA shared updated data from the TRANSCEND-FL trial.

Akshay Sharma, MBBS

Colleen E. Annesley, MD, on Working to Mitigate Inferior Cell Persistence With Manufactured T-APCs

The bone marrow transplant physician at St. Jude Children’s Research Hospital also discussed the recent approval of exa-cel.

Colleen E. Annesley, MD

CRISPR/Cas9 Edited Allograft Eases AEs of Maintenance Therapy for AML

The medical Director and interim co-Chief Medical Officer at Seattle Children's Therapeutics discussed complete data from the PLAT-03 feasibility trial assessing SCRI-CAR19 and CD19t T-APCs in B-ALL.

John F. DiPersio, MD, PhD, Division of Oncology, Washington University School of Medicine

CRISPR/Cas9 Cell Therapy Increases Hemoglobin in Transfusion-Dependent β-Thalassemia Across HBB Genotypes

December 10th 2023

A CRISPR/Cas9-edited allogeneic stem cell transplantation effectively reduced hematopoietic toxicity associated with maintenance gemtuzumab ozogamicin for high-risk acute myeloid leukemia (AML).

CAR T Cells Highly Effective for Autoimmune Disorders

December 10th 2023

Data from 3 early phase clinical trials assessing BRL-101 in 10 patients were presented at the ASH 2023 meeting.

Fabian Müller, MD, of Bavarian Cancer Research Institute and Friedrich-Alexander University of Erlangen-Nuremberg

Akshay Sharma, MBBS, on the Importance of Patient Reported Outcomes for Exa-cel

December 10th 2023

CD19-directed CAR-T-cell therapy resulted in sustained remission for over a year and no return of autoimmunity for patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis.

Akshay Sharma, MBBS

Lovo-Cel Efficacy Maintained at 60 Months for Preventing VOEs in SCD

The bone marrow transplant physician at St. Jude Children’s Research Hospital discussed an analysis of the sickle cell disease gene therapy he’s presenting on at ASH’s 2023 meeting.

Julie Kanter, MD, Division of Hematology and Oncology, University of Alabama

Beti-cel Displays Long-Term Success in Adult and Pediatric Transfusion-Dependent β-Thalassemia

Long-term data on lovo-cel gene therapy for sickle cell disease (SCD) reveals sustained efficacy at 60 months.

Hemophilia B Gene Therapy Etranacogene Dezaparvovec Shown to Be Safe and Effective in Subset of Clinical Trial Patients With Comorbid HIV

Bluebird bio’s gene therapy, marketed as Zynteglo, showed successful rates of transfusion independence up to 9 years with a reasonable safety profile in data presented at ASH 2023.

FDA Experts Weigh In on Exa-cel and Lovo-cel Approvals for Sickle Cell and Corresponding Black Box Safety Warnings

There were no treatment-related serious adverse events reported.

CGTLive’s Weekly Rewind – December 8, 2023

The FDA’s Nicole Verdun, MD; and Peter Marks, MD, PhD, offered insight on the recent approvals of exa-cel and lovo-cel, answering questions about the safety of the gene therapies and the continued collection of data on their use.

CGTLive’s Weekly Rewind – December 1, 2023

CGTLive Staff

FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease

Review top news and interview highlights from the week ending December 8, 2023.

FDA Approves Exa-cel, Vertex and CRISPR Therapeutics’ Gene Therapy, for Sickle Cell Disease

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185).

Sukumar Nagendran, MD, on Addressing Unmet Needs in Rett Syndrome With Gene Therapy

The FDA’s decision was based on data from the CLIMB-121 and CLIMB-131 clinical trials, making it the first CRISPR-based gene therapy to be approved in the US.

Sukumar Nagendran, MD

Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Trial Doses First Patient

The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.

M. Peter Marinkovich, MD, on Developing B-VEC, First Approved Derm Gene Therapy

December 7th 2023

Benetic anticipates an announcement of interim results sometime around the midpoint of 2024.

M. Peter Marinkovich, MD

December 7th 2023

The associate professor of dermatology at Stanford University discussed his experiences investigating and helping to develop Vyjuvek, approved for treating DEB.

MSC Therapy Improves Pain, Stiffness, Function in Osteoarthritis Compared to Placebo