Meeting on the Med 2022 – VJRegenMed

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.

CYP-001 in adults with steroid-resistant GvHD

Simon Ng — Tue, 03 May 2022 16:17:07 +0000

Mesenchymal stem cells (MSCs) derived from induced pluripotent stem cells (iPSCs) have shown promising efficacy as a cell therapy. Ross Macdonald, PhD, Cynata Therapeutics, Melbourne, Australia, discusses results from a Phase I trial (NCT02923375) assessing CYP-001, a MSC-based cell therapy in patients with steroid resistant graft-versus-host disease (GvHD). For patients who progress from corticosteroids, treatment options remain poor and mortality remains high. CYP-001 resulted in a complete response in over half of the cohort and after a two year follow-up, a survival rate of over 50% was reported. This interview was conducted during Meeting on the Mediterranean 2022.

SCUlpTOR: CYP-004 in osteoarthritis

Simon Ng — Tue, 03 May 2022 16:17:06 +0000

Ross Macdonald, PhD, Cynata Therapeutics, Melbourne, Australia, discusses the Phase III SCUlpTOR trial (ACTRN12620000870954) assessing CYP-004, a novel mesenchymal stem cell (MSCs)-based cell therapy for patients with osteoarthritis. Whilst previous studies investigating autologous stem cell therapies have shown promise, there is an unmet need for curative therapies. The randomized double blind trial will assess the investigational therapy in 440 patients with degenerative osteoarthritis. The interview was conducted during Meeting on the Mediterranean 2022.

Plasmacytoid dendritic cell-based cancer vaccines

Simon Ng — Tue, 03 May 2022 16:17:05 +0000

Eric Halioua, PDC*line Pharma, Liege, Belgium, provides an overview of PDC*line, a novel cell line derived from plasmacytoid dendritic cells (PDCs) in the bones of patients with leukemia. The cell line is more potent than allogeneic or autologous myeloid cells and are not rejected in humans. He additionally highlights the ease of manufacturing cancer vaccines from PDC*line due to the consistency of the cell line. This interview was conducted during Meeting on the Mediterranean 2022.

Developing a therapeutic cancer vaccine for patients with NSCLC

Simon Ng — Tue, 03 May 2022 16:17:04 +0000

Eric Halioua, PDC*line Pharma, Liege, Belgium, comments the development of PDC*lung, a novel therapeutic plasmacytoid dendritic cell (PDC)-derived vaccine for patients with non-small cell lung cancer (NSCLC). He describes the mechanism of action of the vaccine, where irradiated PDCs are loaded with peptides encoding various lung tumor antigens. A Phase I/II trial (NCT03970746) will assess the vaccine in 64 patients in either a high or low dose. This interview was conducted during Meeting on the Mediterranean 2022.

Assessing PDC*mel, dendritic cell-based vaccine in melanoma

Simon Ng — Tue, 03 May 2022 16:17:04 +0000

Eric Halioua, PDC*line Pharma, Liege, Belgium, discusses the development of PDC*mel, a plasmacytoid dendritic cell (PDC)-derived vaccine for patients with melanoma. The investigational therapy consists of PDC*line loaded with four melanoma peptides and have the ability to be stored in liquid nitrogen. He also describes a Phase I trial (NCT01863108), which tested the vaccine in three different doses. This interview was conducted during Meeting on the Mediterranean 2022.

Addressing challenges in using MSCs in cell therapies

Simon Ng — Thu, 21 Apr 2022 15:04:28 +0000

Mesenchymal stem cells (MSCs) have demonstrated potential in cell therapies, as they have unique immunomodulatory properties. Ross Macdonald, PhD, Cynata Therapeutics, Melbourne, Australia, comments on the hurdles in commercializing MSC-based cell therapies. Unlike autologous chimeric antigen receptor (CAR) T-cell therapies, MSCs are derived from allogeneic sources, which in itself presents logistical issues in terms of obtaining enough donors. Cell expansion for commercial usage additionally is an issue, as MSCs become less potent as they expand, and Dr Macdonald presents solutions offered by Cynata Therapeutics that attempt to overcome the aforementioned barriers. This interview was conducted during Meeting on the Mediterranean 2022.

Generating iSPC-derived MSCs with the Cymerus™ Platform

Simon Ng — Thu, 21 Apr 2022 15:04:27 +0000

Ross Macdonald, PhD, Cynata Therapeutics, Melbourne, Australia, discusses the process of manufacturing mesenchymal stem cells (MSCs) derived from induced pluripotent stem cells (iPSCs) with the Cymerus Platform. MSCs have shown encouraging efficacy as cell therapies and using iPSCs enables the MSCs to remain potent, as they do not require expansion. The iSPCs also do not require multiple donors as they can replicate indefinitely, resulting in MSCs of a consistent quality. This interview was conducted during Meeting on the Mediterranean 2022.