Gene Therapy – VJRegenMed

CARBON trial: investigating CTX110 allogeneic CRISPR-Cas9–engineered CAR-Ts in R/R LBCL

Anya Kerkache — Thu, 26 Jan 2023 12:11:30 +0000

Constantine Tam, MBBS (Hons), MD, FRACP, FRCPA, Peter MacCallum Cancer Centre, St. Vincent’s Hospital, University of Melbourne, Melbourne, Australia, reports results from the CARBON Phase I dose-escalation study (NCT04035434), testing the safety and efficacy of a novel allogeneic CRISPR-Cas9-engineered CAR-T product, CTX110, in heavily pre-treated patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). The study reported that the CAR-Ts showed clinical activity with the ability to expand in the body, and a low risk of graft-versus-host disease (GvHD). This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

The safety and efficacy of UCART123v1.2 in R/R AML: results from AMELI-01

Anya Kerkache — Thu, 26 Jan 2023 12:11:29 +0000

David Sallman, MD, Moffitt Cancer Center, Tampa, FL, discusses the results of the AMELI-01 Phase I trial of UCART123v1.2 in adult patients with relapsed refractory (R/R) CD123+ acute myeloid leukemia (AML; NCT04106076). UCART123v1.2 is an anti-CD123 allogeneic CAR-T product, that has been genetically modified to minimize the occurrence of graft-versus-host disease (GvHD). Overall, results suggest UCART123v1.2 is well tolerated, though severe cytokine release syndrome (CRS) did occur, and that three-drug lymphodepletion optimizes CAR-T expansion and patient responses. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

Results from a Phase I trial of rapidly manufactured Prgn-3006 Ultracar-T in R/R AML

Anya Kerkache — Thu, 26 Jan 2023 12:11:28 +0000

In this video, David Sallman, MD, Moffitt Cancer Center, Tampa, FL, outlines the completed Phase I results for Prgn-3006 Ultracar-T in patients with relapsed/refractory (R/R) CD33+ acute myeloid leukemia (AML). Prgn-3006 Ultracar-T is an autologous CAR-T cell product with decentralized manufacturing that significantly reduces the time between apheresis and CAR-T infusion compared to standard autologous CAR-T therapies. Overall, results suggest that Prgn-3006 Ultracar-T is well tolerated, with only a few grade 3 cytokine release syndrome (CRS) events and no dose-limiting toxicities. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

Treating otoferlin deficiency with gene therapy

Simon Ng — Thu, 23 Jun 2022 09:28:24 +0000

Nawal Ouzren, Sensorion, Montpellier, France, gives an overview of the rationale behind developing a gene therapy for patients with hearing loss who have an otoferlin deficiency. Otoferlin is a protein expressed in inner hair cells, which is sensitive to a certain frequency. Initial in vivo studies have successfully restored expression of otoferlin in animal models, who have regained functional hearing as a result of the gene therapy. This interview was conducted during Meeting on the Mediterranean 2022.

Novel gene therapy solutions for Usher syndrome type I

Simon Ng — Thu, 23 Jun 2022 09:28:23 +0000

Usher syndrome type I is a rare, congenital disease characterized by issues in the vestibular system, resulting in deafness, balance issues and blindness. Nawal Ouzren, Sensorion, Montpellier, France, discusses research efforts in treating Usher syndrome type I with gene therapy. Whilst vestibular function has been successfully restored in animal models, further research is required to restore hearing. This interview was conducted during Meeting on the Mediterranean 2022.

Developing gene therapies for GJB2 gene related hearing loss

Simon Ng — Thu, 23 Jun 2022 09:28:22 +0000

Nawal Ouzren, Sensorion, Montpellier, France, describes ongoing research into developing gene therapies for patients with hearing loss due to mutations in the GJB2 gene. This form of hearing loss is the most common form of childhood deafness, and some forms of adult hearing loss can be attributed to certain mutations of the GJB2 gene. Current efforts include elucidating a suitable therapeutic candidate in animal models. This interview was conducted during Meeting on the Mediterranean 2022.

Implementing automation technology in the CGT space

Simon Ng — Thu, 28 Apr 2022 08:38:40 +0000

The advent of automation has brought improvements in the manufacturing of advanced therapies in terms of reducing the number of staff needed, cost, and safety. Saskia Rösch, PhD, Miltenyi Biotec, Bergisch Gladbach, Germany, discusses potential barriers developers might face in automating their manufacturing processes. Companies will need to make sure the product can be translated from a research to clinical setting, as well as if the reagents can be scaled-up if necessary. This interview took place at the International Conference on Lymphocyte Engineering (ICLE) 2022.

Applications of stem cell gene therapy beyond genetic diseases

admin_vjr_user — Fri, 22 Apr 2022 12:21:18 +0000

Luigi Naldini, MD, PhD, San Raffaele Telethon Institute for Gene Therapy, Milano, Italy, comments on the potential application of stem cell gene therapy in oncology. In recent years, growing evidence supporting the efficacy and tolerability of this technology in genetic diseases has suggested it as a promising strategy to target tumors by engineering stem cells to target the tumor or to deliver a therapeutic agent. This interview took place at the Transplantation & Cellular Therapy (TCT) Meetings of ASTCT and CIBMTR® 2022 in Salt Lake City, Utah.

Innovations in treating rare diseases with gene therapies

Simon Ng — Wed, 20 Apr 2022 16:45:46 +0000

John Maslowski, CCO, Forge Biologics, Columbus, OH, comments on the current treatment landscape for rare diseases, especially in the field of gene therapies. With a large number of rare diseases lacking any form of treatment, finding curative solutions for these rare diseases remain an unmet need. Finding a suitable clinical model is essential to develop treatments, especially when the natural history group in a clinical trial is preferable to placebo groups, as in the case of studies targeting Krabbe disease. Introducing closed automated systems in the manufacturing process will additionally enable the production of gene therapies in an economical manner. This interview took place at Advanced Therapies Week 2022.

Overcoming manufacturing issues in gene therapies for rare diseases

Simon Ng — Wed, 20 Apr 2022 16:45:46 +0000

John Maslowski, CCO, Forge Biologics, Columbus, OH, discusses strategies to optimize the manufacturing of gene therapies for rare diseases. In addition to regulatory barriers, maintaining the consistency of the product is vital, especially when manufacturing occurs in large-scale bioreactors. To ensure the cost of production remains low and the patients’ needs are successfully met, he emphasizes the need to use automated analytical tools in a closed system. Assessing product quality via high throughput systems can expedite production times on a larger scale. This interview took place at Advanced Therapies Week 2022.