New Genetic Target May Hold Promise in Treating Blindness
The SARM1 gene is a key driver in the damage that ultimately leads to impaired vision.
Around the Helix: Cell and Gene Therapy Company Updates – March 2, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.
A Positive Outlook for Rare Disease Research: Michael Singer, MD, PhD
The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.
Latest Results in ATTR Amyloidosis CRISPR Trial Bolsters Potential of In Vivo Gene Editing
The therapy was well-tolerated in 15 patients and no clinically relevant liver findings were observed.
Cilta-Cel Approved for Relapsed/Refractory Multiple Myeloma
The CAR T-cell therapy from Janssen and Legend Biotech is the second to receive approval with an indication for treatment of multiple myeloma.
Improving Rare Disease Awareness: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.
Advancing Treatments for Rare Diseases Requires a Team Approach
Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.
Developing Therapies for Rare Diseases: Kinnari Patel, PharmD
The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.
Celyad Pauses CAR T Cell Therapy Trial of CYAD-101 Following Patient Deaths
The phase 1b KEYNOTE-B79 trial is investigating the allogeneic cell therapy in participants with metastatic colorectal cancer.
Improving Rare Disease Awareness: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.
Gene Therapy Updates in Neuromuscular Diseases
Crystal Proud, MD, outlines advances in technology and research in neuromuscular diseases.
Ongoing Gene Therapy Trials in Stargardt Disease : Prof. Carel Hoyng
The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
CGTLive’s Weekly Rewind – February 25, 2022
Review top news and interview highlights from the week ending February 25, 2022.
Advanced Ovarian Cancer Immunotherapy Trial Continues
An interim safety review of 81 patients informed the DSMB's recommendation for the trial to continue.
Launching PDGENEration to Advance Genetic Testing in Parkinson Disease: James Beck, PhD
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.
Exploring Cell Therapy for the Treatment of Drug-Resistant Epilepsy
Cory Nicholas, PhD, cofounder and chief executive officer, Neurona Therapeutics, discussed the company’s lead program in mesial temporal lobe epilepsy.
R/R Multiple Myeloma CAR T-Cell Therapy Receives Orphan Drug Designation
CT103A is co-developed by IASO Biotherapeutics and Innovent Biologics.
EtranaDez’s Efficacy in Hemophilia B: Wolfgang Miesbach, MD, PhD
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed new efficacy findings from the HOPE-B study.
Around the Helix: Cell and Gene Therapy Company Updates - February 23, 2022
Managing Safety in Fitusiran Treatments of Hemophilia: Guy Young, MD
The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.
Patient-Informed Study to Evaluate Hunter Syndrome Gene Therapy
The trial design was informed by both patient and physician perspectives.
Unmet Needs in Hemophilia: Steven W. Pipe, MD
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.
Enrollment Pauses in Mesothelioma CAR T-Cell Therapy Trial
The pause follows a fatal serious adverse event in the first patient treated in the highest dose cohort.
CAR T-Cell Therapy Demonstrates Feasibility in Resistant Prostate Cancer
The trial, sponsored by Mustang Bio, will continue to enroll patients and doses will be escalated to 300 million cells.
Second Phenylketonuria Gene Therapy Study Put on Clinical Hold
The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.
Liso-Cel Gets Priority Review for Second-Line R/R Large B-Cell Lymphoma
The new application is based on data from the phase 3 TRANSFORM trial.
Gene Therapy for Diabetic Retinopathy Yields Continued Improvements
The therapy has been well-tolerated in the 15 patients treated so far.
Assessing Safety of Fibroblast Cell Therapy in Multiple Sclerosis: Hamid Khoja, PhD
The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.
Stem Cell Model Developed to Study Albinism-Related Eye Conditions
Researchers will use the model to study how lack of pigmentation affects RPE physiology and function.
