CGTLive’s Weekly Rewind – January 20, 2023
Review top news and interview highlights from the week ending January 20, 2023.
Late-Onset Pompe Disease Gene Therapy Cleared to Continue Trial
The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.
Diane Simeone, MD, on Addressing Unmet Needs in Colorectal Cancer, NSCLC, and Pancreatic Cancer
Simeone discussed the design of BASECAMP-1, an observational study for patients previously treated for solid tumors who are at a high risk of relapse.
Editas Sells off iNK Preclinical Cell Therapy Program, Gene-Editing Technologies to Shoreline
The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.
Gene Therapy Improves Visual Acuity in MT-ND4 LHON Compared to Natural History
Investigators analyzed data from 3 phase 3 studies and a long-term follow-up study.
Etranacogene Dezaparvovec “Transformative” for Patients With Hemophilia B
Steven Pipe, MD, CS Mott Children’s Hospital, discussed the latest follow-up data from the HOPE-B study of the approved therapy, Hemgenix.
Alan Trounson, PhD, MSc, on New Frontiers in Allogeneic Cell Therapy
The chief executive officer of Cartherics discussed the company’s development of NK cell, T-cell, and macrophage cell therapies for various solid tumor indications.
Around the Helix: Cell and Gene Therapy Company Updates – January 18, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CAR-T for NMOSD Demonstrates Manageable Safety in Phase 1 Trial
It was previously announced that CT103A received FDA clearance of its investigational new drug application for the treatment of relapsed/refractory (r/r) multiple myeloma.
Neovascular AMD Gene Therapy Cleared for Trials
A phase 1 clinical trial of EXG102-031 is expected to initiate in the first quarter of 2023.
Akshay Sharma, MBBS, on Investigating CRISPR/Cas9 Edited Cells for Sickle Cell Disease
The assistant member of the bone marrow transplant department at St. Jude Children’s Research Hospital discussed advantages of OTQ923 in treating SCD.
Krabbe Disease Gene Therapy Receives EMA PRIME Designation
Forge Biologics’ FBX-101 previously demonstrated promising safety and efficacy in early data from the phase 1/2 RESKUE clinical trial.
CAR-T in Fibrin Glue-based Carrier Shows Potential as Adjuvant to Solid Tumor Surgery
Of the 20 mice treated with the experimental approach, 19 achieved clearance of residual tumor cells.
Arshad Khanani, MD, on Restoring Aflibercept Expression in Wet AMD With Gene Therapy
The director of clinical research at Sierra Eye Associates discussed data from the phase 1/2 PRISM study.
Small Molecule to Be Evaluated for Treatment of CAR-T-related Cytokine Release Syndrome
Poolbeg Pharma’s POLB 001 previously showed promise in a lipopolysaccharide human challenge trial.
Terese Hammond, MD, on Promising Data With iNKT Cells in COVID-associated ARDS
The director of the CCU/ICU at Saint John’s Health Center discussed updated data from a phase 1/2 trial of agenT-797.
Preclinical Research, Genetic Testing, Help Pave Gene Therapy’s Road to Clinic in Parkinson Disease
Seelos Therapeutics’ SLS-004 showed the ability to downregulate α-synuclein, and CENTOGENE’s ROPAD study will be extended.
Armin Ghobadi, MD, on Assessing CAR-T Cell Combination Therapies
The associate professor of medicine at Siteman Cancer Center and WUSTL discussed further research to be done with CAR T-cell therapies.
Virus-Specific T-Cell Therapy Makes Headway in Virus Prevention After Allo-HCT
Several phase 3 trials for AlloVir’s posoleucel are expected to complete enrollment in 2023.
Advocating for Patients With Chronic Lymphocytic Leukemia
Brian Koffman, MDCM, DCFP, FCFP, DABFP, MSEd, Co-Founder, Executive Vice President, and Chief Medical Officer, CLL Society, discussed the role the society plays in the field.
CGTLive’s Weekly Rewind – January 13, 2023
Review top news and interview highlights from the week ending January 13, 2023.
Ivan Horak, MD, on Early Clinical Efficacy of Allogeneic CD30.CAR EBVST Cell Therapy in Lymphomas
The chief medical and scientific officer of Tessa Therapeutics discussed data from the BESTA and CHARIOT studies presented at ASH 2022.
Hypertrophic Cardiomyopathy Gene Therapy Receives IND Clearance
Tenaya Therapeutics expects the dosing of the first patient in the planned phase 1b trial in Q3 2023.
Advanced Solid Tumor CAR-T Therapy Trial Doses First Patient
The trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of B4t2-001.
Giedre Krenciute, PhD, on Revitalizing T-cells in CAR T-cell Therapy
The assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.
Cell Therapy Development Advances in Hemorrhagic Trauma, Acute Ischemic Stroke
Athersys has made progress in enrollment with its MATRICS-1 and MASTERS-2 clinical trials.
B-VEC PDUFA Date Extended to May 2023
The FDA made the decision in response to manufacturing process information that it considered a major amendment.
Around the Helix: Cell and Gene Therapy Company Updates – January 11, 2023
Rebecca Epperly, MD, on Emerging Clinical Research With Cell Therapy in Leukemia, Lymphoma
The clinician scientist at St. Jude Children’s Research Hospital discussed the session on cell therapies in acute leukemias and Hodgkin lymphoma she moderated at ASH 2022.
Engineered TIL Therapy Trial Reopens Enrollment After Voluntary Pause Related to Manufacturing Analysis
Instil Bio, ITIL-306’s developer, expects to present initial data from the phase 1 trial’s dose escalation cohorts this year.
