Meeting on the Mesa 2021 – VJRegenMed

Overcoming immunogenicity of gene therapies

Simon Ng — Mon, 06 Dec 2021 09:29:02 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, discusses the impact of pre-existing immunogenicity against adeno-associated virus (AAV)-based gene therapies. Patients who have an immune response to recombinant AAV vectors will require immunosuppression to benefit from gene therapies. Various strategies can be employed to overcome this issue by utilizing combination therapies, especially in the case of FBX-101, an AAV-based gene therapy for patients with Krabbe disease. This interview took place at Meeting on the Mesa 2021.

RESKUE: AAV-based gene therapy for Krabbe disease

Simon Ng — Mon, 06 Dec 2021 09:29:01 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, describes the trial design of the Phase I/II RESKUE trial (NCT04693598), assessing FBX-101, an adeno-associated virus (AAV)-based gene therapy for patients with Krabbe disease receiving a bone marrow transplant. The preliminary results of the dose escalation trial will aim to be released by 2022. This interview took place at Meeting on the Mesa 2021.

Increasing manufacturing capabilities for gene therapies

Simon Ng — Mon, 06 Dec 2021 09:29:00 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, addresses major hurdles facing gene therapy manufacturers. Commercialization of therapies remains a challenge, due to the accelerated development of novel gene therapies for various conditions including Duchenne muscular dystrophy (DMD) and hemophilia and a lack of suitable bioreactors. Scaling up manufacturing processes and increasing quality control will be necessary to meet increased demand from developers. This interview took place at Meeting on the Mesa 2021.

Treating Krabbe disease with AAV-based gene therapies

Simon Ng — Mon, 06 Dec 2021 09:28:59 +0000

Timothy Miller, PhD, Forge Biologics, Columbus, OH, provides an overview of the current treatment landscape for Krabbe disease and the potential for gene therapy as a curative treatment. Krabbe disease is a lysosomal storage disorder that affects the nervous system, and the current standard of care is a bone marrow transplant. Dr Miller additionally describes an adeno-associated virus (AAV)-based gene therapy that when administered in conjunction with the bone marrow transplant, lacks the immunogenicity-related issues associated with AAV vectors. This interview took place at Meeting on the Mesa 2021.

Remaining barriers in commercializing advanced therapies

Simon Ng — Mon, 29 Nov 2021 11:36:57 +0000

Pascal Touchon, President & CEO, Atara Biotherapeutics, San Francisco, CA, addresses issues relating to the commercialization of cell and gene therapies. Major challenges remain in terms of logistics and delivery. Off-the-shelf allogeneic therapies can be given to patients quicker than autologous therapies, as in the case of tabelecleucel, a novel CAR-T cell therapy which can be delivered to patients in three days. He additionally highlights reimbursement issues for these one-off treatments. This interview took place at Meeting on the Mesa 2021.

ATA3219, ATA3271 & ATA188: T-Cell immunotherapy products in development

Simon Ng — Mon, 29 Nov 2021 11:36:56 +0000

Pascal Touchon, President & CEO, Atara Biotherapeutics, San Francisco, CA, describes the pipeline of Atara Biotherapeutics including the ATA3219, an anti-CD19 allogeneic T-cell immunotherapy targeting B-cell malignancies, as well as ATA3271, a chimeric antigen receptor (CAR) T-cell therapy targeting mesothelin for solid tumors. He additionally discusses the latest results from the Phase I/II EMBOLD study (NCT03283826) of ATA188, a T-cell immunotherapy that targets Epstein-Barr virus (EBV) in patients with progressive multiple sclerosis. This interview took place at Meeting on the Mesa 2021.

Next steps for procine-derived bioengineered organs

Simon Ng — Thu, 25 Nov 2021 18:05:25 +0000

Jeff Ross, PhD, Miromatrix, Eden Prairie, MN, describes the process of taking bioengineered organs derived from porcine organs to clinical trials. The porcine organ scaffold produced as a result of perfusion decellularization has been successfully commercialized and is currently used in transplants Published studies have additionally shown the process of perfusion recellularization to be successful in creating viable bioengineered organs. This interview took place at Meeting on the Mesa 2021.

Can manufacturing bioengineered organs be scaled up?

Simon Ng — Thu, 25 Nov 2021 18:05:23 +0000

Jeff Ross, PhD, Miromatrix, Eden Prairie, MN, discusses the manufacturability of bioengineered organs developed from porcine organs. Dr Ross highlights how the decellularized porcine cells leaves behind an extracellular matrix and that the scaffold has already been commercialized. The current techniques to mass produce bioengineered organs can subsequently be scaled up by using bioreactors. This interview took place at Meeting on the Mesa 2021.

Remaining challenges in developing cell therapies for cancer

Simon Ng — Thu, 25 Nov 2021 18:05:21 +0000

Pascal Touchon, President & CEO, Atara Biotherapeutics, San Francisco, CA, discusses the current progress in successfully developing cell therapies for liquid and solid tumors. In the field of hematological malignancies, cell therapies need to improve their response rate and durability of remission in patients, whereas in solid tumors, developing treatments that are as effective as ones used for liquid tumors remain a challenge. Improving in vivo persistence of cell therapies is one strategy to develop treatments that are more efficacious against solid tumors, as in the case of ATA2271, an chimeric antigen receptor (CAR) T-cell therapy targeting mesothelin. This interview took place at Meeting on the Mesa 2021.

The current landscape of allogeneic CAR T-cell therapies

Simon Ng — Thu, 25 Nov 2021 18:05:19 +0000

Pascal Touchon, President & CEO, Atara Biotherapeutics, San Francisco, CA, provides an overview of allogeneic chimeric antigen receptor (CAR) T-cell therapies in development. He describes ATA3219, a novel anti-CD19 CAR-T cell therapy developed for B-cell malignancies, as well as ATA2271, a mesothelin-targeted CAR-T immunotherapy for solid tumors. Tabelecleucel is an additional CAR-T cell therapy that treats Epstein-Barr virus (EBV)-associated post-transplant lymphoproliferative disease (PTLD) following hematopoietic cell or solid organ transplantation. This interview took place at Meeting on the Mesa 2021.