bluebird bio's Lovo-Cel Has Durable Effect in Sickle Cell Disease
Additional data on 2 patients who developed persistent anemia suggests a genetic cause may be behind the serious adverse event.
Enhanced Cell Therapy Gets Fast Track Designation for HPV16+ Solid Tumors
Two of 4 evaluable patients showed stable disease in data presented at ESMO-IO 2022.
Maria Escolar, MD, on Combining HSCT With Gene Therapy in Krabbe Disease
The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.
Maria Escolar, MD, on Addressing Unmet Needs in Krabbe Disease With Gene Therapy
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
Amod Sarnaik, MD, on New Frontiers in Cell Therapy for Advanced Melanoma
Sarnaik discussed several potential areas of innovation on the horizon.
Terese Hammond, MD, on Investigating iNKT Cells in COVID-Associated ARDS
The director of the CCU/ICU at Saint John’s Health Center discussed unmet respiratory treatment needs in the beginning of the COVID-19 pandemic.
Amod Sarnaik, MD, on Lifileucel’s Place in the Treatment Lanscape for Advanced Melanoma
Sarnaik discussed the investigational TIL therapy’s potential as an additional option for patients.
Combination Adenovirus Therapy Shows Survival Gains in Newly Diagnosed High-Grade Glioma
CAN-2409 transduces tumor cells with the thymidine kinase gene, sensitizes these cells to valacyclovir, and stimulates patients’ immune response.
CT-0508 Demonstrates Cytotoxicity in Solid Tumors
Updated data from a first-in-human trial were presented at the SITC 2022 meeting.
Omid Hamid, MD, on Improving Duration of Response in Refractory Melanoma With Lifileucel
The chief of research and immunotherapy at Cedars-Sinai The Angeles Clinic and Research Institute discussed updated data from the phase 2 C-144-01 study of the lifileucel TIL therapy.
HLA LOH-Targeting Tmod Cell Therapies Demonstrate Selective Killing of Tumor Cells in Mice
A2B530 and A2B694 target CEA and MSLN cells that have HLA loss of heterozygosity.
CRISPR-CAR T Therapy Yields First Allogeneic Complete Response in R/R Solid Tumors
Data from the COBALT-RCC study in renal cell carcinoma were presented at SITC 2022.
Susan Foy, PhD, on Evaluating Personalized, Neoantigen-Specific T Cell Therapy in Solid Tumors
The CRIPSR-edited neoantigen-specific T cell therapy demonstrated safety and feasibility in the first-in-human PACT-0101 study presented at SITC 2022.
Gene Expression Profiles Characterize CAR T-Cells in Solid and Hematological Tumors
Further analysis will be performed to elucidate which characteristics correlate with optimal cell therapy behavior.
Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.
Paul Melmeyer on Regulatory Advocacy for Patients With Neuromuscular Diseases
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.
Benjamin L. Schlechter, MD, on TAC01-HER2 in HER2-Positive Solid Tumors
Schlechter discussed the potential advantages of TAC01-HER2 over current standards of care.
Benjamin L. Schlechter, MD, on Addressing Unmet Needs in HER2 Positive Solid Tumors
Schlechter discussed the design of the TACTIC-2 clinical trial for patients with HER2-positive solid tumors.
EBV-targeted T-cell Immunotherapy Associated With Improvements in Progressive Multiple Sclerosis
MRI revealed that participants who achieved sustained or confirmed disability improvement showed significantly less ventricular enlargement at 12 months.
Vinaya Murthy, MS, MPH, on Genetic Counseling Services for Rare Disorders
As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.
Chris Bond, PhD, on Allogeneic and Induced Pluripotent Stem Cell Approaches to Cell Therapy
The senior vice president of Research & Early Development at Notch Therapeutics discussed the company’s approach to overcoming the drawbacks of autologous cell therapies.
Improving Access to Rare Disease Care
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
Bridging the Gap: Education, Awareness Key to Translating Medical Innovation to Community Health Care in Rare Diseases
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
Michel Michaelides, MD, FACP, on Gene Therapy's Potential in Retinitis Pigmentosa
The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.
Gene Therapy Continues to Show Promise in Canavan Disease
Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.
Devon J. Shedlock, PhD, on Shifting from Autologous to Allogeneic Therapies
The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.
Deyaa Adib, MD, on Optimizing Manufacturing of Solid Tumor Cell Therapies
The chief medical officer of Triumvira Immunologics discussed the company’s future plans.
Patients With Fabry Disease Withdrawn From ERT After Isaralgagene Civaparvovec Treatment
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
Janssen’s Gene Therapies Show Promise in Retinitis Pigmentosa, Geographic Atrophy
Both botaretigene sparoparvovec and JNJ-81201887 were well-tolerated in treated patients, according to data from the 2022 AAO meeting.
