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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Paulina Velasquez, MD, on Finding New Targets for Cell Therapy
The assistant member at St. Jude Children’s Research Hospital discussed the ASH 2022 basic and translational science session she moderated.
Crigler-Najjar Syndrome Gene Therapy Trial Enters Pivotal Portion
Some patients previously treated with GNT-003 were able to maintain the stoppage of phototherapy for at least 1 year.
Second Generation MUC1-CAR T Therapy Cleared for Solid Tumor Trial
huMNC2-CAR22 is designed to avoid off-tissue expression and reduce T-cell exhaustion.
Val-rox Meets End Points in Hemophilia A at 3 Years
The new analysis data was requested by the FDA ahead of the therapy’s March 31, 2023 PDUFA date.
Canavan Disease Gene Therapy Continues to Show Promise
No serious adverse events related to the gene therapy have been reported.
Celularity’s MSC Platform Shows Clinical Benefit for Crohn Disease in Legacy Analyses
Phase 1/2 studies evaluated PDA001 in 50 participants with CD.
Leber Congenital Amaurosis-2 Gene Therapy Trial Doses First Patient
The company expects to provide initial data from the trial in the second half of this year.
Developing Nonviral Gene Therapy for Hemophilia A
Brent Warner, president, gene therapy, Poseida Therapeutics, discussed data on the preclinical P-FVIII-101 presented at ASH 2022.
First Patient Dosed in Treg Cell Therapy Trial for Myelofibrosis
CKO804 is being evaluated as an add-on therapy to ruxolitinib in patients whose disease had a suboptimal response to ruxolitinib alone.
MPS-IH HSC Gene Therapy Gets IND Clearance
Promising results were previously reported from a proof-of-concept study involving 8 patients.
Sickle Cell Disease Gene-Editing Therapy Trial Voluntarily Paused Following Serious Adverse Event
The first patient dosed in the study experienced a case of prolonged pancytopenia that has required ongoing transfusion and growth factor support.
CGTLive’s Weekly Rewind – January 6, 2023
Review top news and interview highlights from the week ending January 6, 2023.
Intellia to Advance Development of ATTR Amyloidosis, Angioedema, AATD Gene Therapies
The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.
