Frederick “Eric” Arnold, PhD, on Investigating Alternative Polyadenylation in ALS Models
The postdoctoral scholar at University of California – Irvine discussed his research looking at TDP-43 and alternative polyadenylation in neuronal cells.
Around the Helix: Cell and Gene Therapy Company Updates – March 6, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Alan Beggs, PhD, on Digging Dipper Into Unexpected Toxicities With Neuromuscular Gene Therapy
The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed investigations into liver toxicity and deaths after AT132 gene therapy.
Cardiac Function Preserved in Long-Term Follow-Up of Patients Treated With Duchenne Muscular Dystrophy Gene Therapy SGT-001
The data comes from 7 patients treated in the phase 1/2 IGNITE-DMD study (NCT03368742).
PJ Brooks, PhD, on Working to Bring Gene Therapy Development to More Neuromuscular Disorders
The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.
Patients With Spinal Muscular Atrophy Who Required Tracheostomies Show Motor Function Improvements in Real World Setting After Treatment With Zolgensma
The real-world data, which includes patients who received Zolgensma before and after tracheostomy, comes from the noninterventional RESTORE registry (NCT04174157).
Carlos Moraes, PhD, on Organelle, RNA Function and Trafficking in Neuromuscular Disease
The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed the role of organelle transport in neuromuscular diseases such as CMT.
Zolgensma Demonstrates Clinical Benefit and Safety in Heavier Patients With Spinal Muscular Atrophy
The phase 3b SMART trial included 24 patients in total who each weighed from 8.5 kg to 21 kg at the time of treatment.
RGX-202 Gene Therapy Reduces CK, Yields Detectable Microdystrophin in Patients With DMD
RGX-202 delivers a slightly larger form of microdystrophin than other gene therapies, including the CT domain of dystrophin.
Regeneron Subsidiary Decibel Therapeutics Looks to Assess Gene Therapy for Otoferlin Gene-Mediated Hearing Loss in Phase 1/2 CHORD Clinical Trial
In observance of World Hearing Day, held annually on March 3, CGTLive® took a closer look at the clinical evaluation of hearing loss gene therapy DB-OTO.
George Tachas, PhD, on Targeting Inflammation in DMD With Antisense Oligonucleotides
The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.
Bruce Cree, MD, PhD, MAS, on the Importance of Understanding Disease Pathobiology for Novel Treatment Approaches
The clinical research director of the UCSF Multiple Sclerosis Center discussed the difference between developing targeted therapies for well-understood diseases like SCD and less well-understood diseases like MS.
FDA Activity Recap: February 2024 Features Major Approval, Priority Review for a BLA Efficacy Supplement, and PDUFA Date Change
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
David Porter, MD, on Enabling the Expansion of CAR T to Autoimmune Diseases
The director of Cell Therapy and Transplant at Penn Medicine discussed how experience with CAR T in oncology has enabled the use of the technology in autoimmune diseases.
Data Roundup: February 2024 Features Updates in Blood Cancers, Lysosomal Storage Disorders at WorldSymposium, Tandem Meetings
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Judy Lieberman, MD, PhD, on Developing siRNA Therapies That Target Specific Cell Types
The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed past, present, and potential future applications of siRNA in the treatment of various diseases.
CGTLive®’s Weekly Rewind – March 1, 2024
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.
Alexandra Gomez-Arteaga, MD, on The Benefit of Orca-T Over PTCy HCT
The Assistant Professor of Medicine at Weill Cornell Medical College shared the background rationale of her retrospective analysis presented at Tandem 2024.
First Patient With Esophageal Cancer Receives TAC101-CLDN18.2 Cell Therapy
The patient is doing well so far and may receive a booster dose of the TAC cell therapy.
MSC Therapy Well-Tolerated in Lumbar Disc Disease, Blinded Data Show Trends of Pain Improvement
BioRestorative has submitted an IND amendment to compare BRTX-100 to sham injection instead of saline injection.
Paul Harmatz, MD, on Harnessing Accelerated Approval for LSDs, Rare Disease Treatment Advancements
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges in investigating therapies for rare diseases.
Latest News in Cell and Gene Therapy for Rare Disease Day 2024
In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.
Mark Walters, MD, on Lessons Learned With Investigating Lovo-Cel for Sickle Cell
The professor in residence of pediatrics at University of California San Francisco discussed his experiences investigating Lyfgenia in clinical trials.
Around the Helix: Cell and Gene Therapy Company Updates – February 28, 2024
UX111 Reduces Heparin Sulfate, Correlating With Benefit in Cognitive Function in Pediatric MPSIIIA
Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.
Bruce Cree, MD, PhD, MAS, on Selecting the Right Neurologic Autoimmune Disease Patient Populations for CAR-T
The clinical research director of the UCSF Multiple Sclerosis Center discussed situations where CAR-T clinical trials may or may not be a good option for patients with various neurologic autoimmune diseases.
Cilta-cel Achieves High Rates of MRD Negativity in Early-Line Multiple Myeloma
The long-term data comes from 2 cohorts in the phase 2 CARTITUDE-2 clinical trial.
Paul Harmatz, MD, on Reducing D2S6 in Neuronopathic Hunter Syndrome With RGX-121 Gene Therapy
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed the dose response seen with RGX-121.
mHA-Specific Donor Tregs Show Potential in Preventing GvHD in Donor HCT
The expanded donor Treg clones maintained lineage fidelity and were persistent through 1 year post-HCT.
Emilie Aschenbrenner, PharmD, BCOP, on CAR-T Versus Bispecific Antibodies for the Treatment of LBCL
The hematology coordinator for pharmacy at Froedtert and the Medical College of Wisconsin discussed the advantages of each of the 2 modalities in the third and second line settings.
