Christopher Evans – VJRegenMed

Chemically modified mRNA encoding BMP-2 for bone healing

admin_vjr_user — Thu, 12 Aug 2021 18:50:20 +0000

Christopher Evans, PhD, Mayo Clinic, Rochester, MN, provides an overview of research into healing bone defects using gene therapy. Bone grafts such as Infuse, which contains recombinant human bone morphogenetic protein-2 (rhBMP-2), can have severe side effects and is expensive due to challenges with delivery, meaning a high dose is required. Gene therapy approaches offer the potential to overcome these challenges, such as the use of adenovirus vectors to improve delivery of BMP-2. The transfection of chemically modified mRNA encoding BMP-2 protein is also being investigated and has proven to be efficacious in healing bone defects in preclinical models. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

The gene therapy landscape for osteoarthritis

admin_vjr_user — Thu, 12 Aug 2021 10:07:38 +0000

Christopher Evans, PhD, Mayo Clinic, Rochester, MN, summarizes the current research landscape for gene therapy in osteoarthritis (OA) and the rationale behind the use of gene therapy to deliver genes encoding anti-arthritic products to the joint. Dr Evans highlights two ongoing Phase I studies involving vector-based gene delivery in OA and a Phase III study (NCT03203330) evaluating the ex vivo delivery of allogeneic chondrocytes transduced with transforming growth factor-beta 1 (TGF-β1) for the treatment of knee OA. Challenges in developing gene therapies for OA include cost, as well as reluctancy in the field around utilizing a gene therapy for a non-genetic condition. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

IL-1Ra gene therapy for knee OA: interim Phase I results

admin_vjr_user — Thu, 03 Jun 2021 16:50:52 +0000

Christopher Evans, PhD, Mayo Clinic, Rochester, MN, describes interim results of an ongoing Phase I dose escalation study (NCT02790723) evaluating the safety and tolerability of an adeno-associated virus (AAV) vector encoding the human interleukin-1 receptor antagonist (IL-1Ra) in patients with knee osteoarthritis (OA). Nine patients with mid-stage OA that had not responded to conventional therapy received an intra-articular injection of the gene therapy vector into one knee and were followed up for one year. Results have demonstrated the safety and tolerability of the therapy, with only one adverse reaction of swelling following injection, which have prompted plans for a Phase II trial to evaluate therapeutic effect. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.