The Potential of Gene Therapy in Inherited Retinal Disease
Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, discussed OCU400, the company’s gene-agnostic gene therapy for IRDs.
Cell Therapies Hope to Be Next Frontier for Alzheimer Disease
As cell therapy investigations mature in the field of AD, some programs show promising signs of efficacy.
Daniela van Eickels, MD, PhD, MPH, on Addressing Unmet Needs in Autoimmune Disease With Cell Therapy
The vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization discussed the Autoimmunity Cell Therapy Network.
Immix and Nexcella Make Progress in US Trial for Light Chain Amyloidosis CAR-T NXC-201
NEXICART-2 has moved on to its expansion cohort after completing dosing of patients in its initial cohort.
CGTLive®’s Weekly Rewind – October 4, 2024
Review top news and interview highlights from the week ending October 4, 2024.
Data Roundup: September 2024 Features Updates in Ophthalmology Gene Therapy, CAR-T for Lymphoma, and More
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
First Patient Dosed Achieves Complete Response in Estrella Immunopharma’s Trial for CD19-Targeted T-cell Therapy EB103 in B-Cell Lymphomas
Estrella noted that the patient had been diagnosed with follicular lymphoma grade 3A with high-risk 3B symptoms.
CRISPR-Mediated In Vivo Epigenomic Activation
Daniel Hart, PhD, the senior director and head of technology development at Epic Bio, discussed potential applications of the new technology.
Around the Helix: Cell and Gene Therapy Company Updates – October 2, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Myrtelle’s Canavan Disease Gene Therapy rAAV-Olig001-ASPA Decreases N-Acetylaspartate Levels in Phase 1/2 Study
CSF analyses from 7 patients treated in the study showed reductions of more than 80% in NAA levels from baseline.
Anixa Biosciences Seeks Protocol Change to Enable Repeat Dosing in Trial for Ovarian Cancer CER-T
Anixa noted that it had previously obtained clearance of a single-patient investigational new drug application to redose a patient in the trial.
Cilta-cel Boosts Overall Survival in New Early Line Multiple Myeloma Data
The data, from the CARTITUDE-4 clinical trial, compared outcomes to patients treated with standard of care options.
FDA Activity Recap: September 2024 Features Acute Lymphoblastic Leukemia Trial Clearance, Multiple RMAT Designations, 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.
Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial
The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.
Atamyo Therapeutics Hits Milestones for Limb-Girdle Muscular Dystrophy Programs
The company pointed out that today, September 30, is LGMD Awareness Day.
News in Cell and Gene Therapy for Rare Cancer Day 2024
In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news related to gene and cell therapies in development for these indications.
World Heart Day 2024: Looking Back at Cardiology's Year of Progress in Cell and Gene Therapy
In observance of World Heart Day, held annually on September 29, we took a look back at the past year's news in cell/gene therapy for cardiovascular disease.
Potential Upcoming Treatments for Duchenne Muscular Dystrophy
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed several still-investigational drugs and therapies for DMD.
CGTLive®’s Weekly Rewind – September 27, 2024
Review top news and interview highlights from the week ending September 27, 2024.
BMS and 2seventy bio Axe Trial for Ide-Cel in Newly Diagnosed Multiple Myeloma
Enrollment difficulties were cited as the reason for discontinuing recruitment activities for KarMMa-9.
Canada Approves Vertex and CRISPR Therapeutics’ Gene Therapy Casgevy for Sickle Cell Disease and TDT
The approvals are indicated for patients 12 years and older.
Genprex Makes Moves to Bring its Gene Therapy Product Reqorsa to Mesothelioma
Notably, today, September 26, is annually observed as Mesothelioma Awareness Day by the patient and clinician communities.
Arun Upadhyay, PhD, on the Potential of Gene Therapy in Inherited Retinal Disease
The chief scientific officer and head of research, development, and Medical at Ocugen, discussed OCU400, the company’s gene-agnostic gene therapy for IRDs.
Around the Helix: Cell and Gene Therapy Company Updates – September 25, 2024
Navigating the New Landscape of Care for DMD
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.
Biomarker Data from Oncology Trial for Adicet Bio’s Gamma Delta T-cell Therapy ADI-001 Indicates Potential for Use in Autoimmune Disease
In lymph node biopsies from patients treated in the study, a mean exposure of 236,701 CAR T-cells per million across all dose levels was observed.
Beacon Therapeutics’ XLRP Gene Therapy AGTC-501 Continues to Show Favorable Benefit-Risk Profile at 36 Months
There were no SUSARS and no endophthalmitis observed among 29 patients treated in the study.
Kiromic BioPharma Gets Greenlight to Move to Dose Expansion in Trial for NSCLC T-cell Therapy Deltacel
Kiromic anticipates that Deltacel-01's expansion portion, which is expected to include around 9 new patients, will begin screening patients before the end of September.
Balancing Risks and Rewards With Muscular Dystrophy Gene Therapy
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.
AAVantgarde Bio Doses First Patient in Trial for Usher Syndrome Gene Therapy AAVB-081
AAVB-081 is intended to address retinitis pigmentosa that results from Usher syndrome type 1B.