Manali Kamdar, MD, on Sequencing of Treatment in Third-Line R/R LBCL
The clinical director of lymphoma services at the University of Colorado discussed the choice between treating patients with liso-cel or bispecific T-cell engagers.
First Patient With Multiple Myeloma Dosed in EsoBiotec's Trial for In Vivo CAR-T ESO-T01
According to EsoBiotec, this is the first time a patient has been treated with an in vivo BCMA-directed CAR-T therapy in a clinical trial.
Tenaya’s Hypertrophic Cardiomyopathy Gene Therapy TN-201 Well-Tolerated in Early Clinical Data
The data comes from 3 patients treated at the trial’s low dose.
Around the Helix: Cell and Gene Therapy Company Updates – January 8, 2025
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
RESTEM’s ULSC Therapy Restem-L Fast Tracked by FDA for Idiopathic Inflammatory Myopathy
Restem-L previously received orphan drug designation from the FDA for PM/DM in December 2024.
Ultragenyx Puts BLA for MPSIII Gene Therapy UX111 in Front of FDA
Ultragenyx is seeking accelerated approval for the BLA based on the use of CSF heparan sulfate data from the phase 1/2/3 Transpher A clinical trial.
Arbor Biotechnologies’ CRISPR-Based Gene Editing Therapy ABO-101 Cleared for US Trial in Primary Hyperoxaluria Type 1
Arbor noted it has designed redePHine, a phase 1/2 clinical trial (NCT identifier pending) that will evaluate ABO-101 in adults and children with PH1.
Data Roundup: December 2024 Features Updates for Cilta-Cel in Multiple Myeloma, mRNA CAR-T in Myasthenia Gravis, and More
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
FDA Activity Recap: December 2024 Features New Approval, a Clinical Hold, and More
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews
In 2024, CGTLive spoke with experts at various medical conferences to seek their insight on the data they presented and more.
CGTLive®’s Weekly Rewind – January 3, 2025
Review top news and interview highlights from the weeks ending December 27, 2024, and January 3, 2025.
Leveling Up: Cell Therapy Research Expands Horizons in Autoimmune Disease
Cell therapy, particularly CAR-T, is expanding into autoimmune diseases like lupus and multiple sclerosis. While promising, safety, efficacy, and broad applicability remain uncertain.
CGTLive's 2024 Pillars of Progress: Top News in Endocrinology/Dermatology
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular endocrinology and dermatology stories in 2024.
Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA
The company requested priority review for the BLA.
CGTLive's 2024 Pillars of Progress: Top News in Lysosomal Disorders
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular lysosomal disorders stories in 2024.
CGTLive's 2024 Pillars of Progress: Most-Read Conference Coverage Stories
Look back on the 2024 stories that our readers were on top of during the medical meetings covered by the CGTLive team.
CGTLive's 2024 Pillars of Progress: Top News in Hematology
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular hematology stories in 2024.
CGTLive's 2024 Pillars of Progress: Top News in Cardiology
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular cardiology stories in 2024.
CGTLive's 2024 Pillars of Progress: Top News in Oncology
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular oncology stories in 2024.
CGTLive's 2024 Pillars of Progress: Top News in Neurology
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular neurology stories in 2024.
CGTLive's 2024 Pillars of Progress: Top News in Ophthalmology
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular ophthalmology stories in 2024.
CGTLive's 2024 Pillars of Progress: Most-Read Features
Take a look what stood out as pillars of progress and success from all of CGTLive's most popular feature stories in 2024.
CGTLive's 2024 Pillars of Progress: Most-Read FDA News in Cell and Gene Therapy
Take a look at the stories that stood out as pillars of progress and success from all of CGTLive's coverage of FDA actions in 2024.
CGTLive's 2024 Pillars of Progress: Most-Watched Expert Interviews
In 2024, CGTLIve spoke with experts outside the context of the conference cycle to seek their insight on company announcements, pipeline therapies, and disease awareness, among other topics.
The Curative Potential of Liso-Cel in Third-Line R/R LBCL
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.
Aurion Biotech’s Cell Therapy AURN001 Demonstrates Efficacy and Safety in Phase 1/2 ABA-1, CLARA Clinical Trial
The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.
Confirming the Safety of Pfizer's Hemophilia B Gene Therapy Beqvez
Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.
CGTLive®’s Weekly Rewind – December 20, 2024
Review top news and interview highlights from the week ending December 20, 2024.
Evaluating siRNA Therapy Fitusiran in Combination With Antithrombin Modulation for Hemophilia
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, discussed findings from the open-label extension of the ATLAS studies at ASH 2024.
The Power and Potential of Natural Killer Cell Therapies: A New Awakening
Natural killer cells could redefine medicine and offer new hope for cancer, autoimmune diseases, and neurological disorders through advanced CAR-NK therapies.
