Duchenne Muscular Dystrophy – VJRegenMed

Challenges in manufacturing viral vectors for the clinic

Simon Ng — Mon, 28 Feb 2022 17:56:23 +0000

Nathalie Clement, PhD, Unicorn Consultations, Gainesville, FL, describes challenges in the development and production of clinically effective viral vectors. Manufacturing larger doses on a bigger scale has been a barrier for companies, and difficulties in purification has led to issues in potency and safety. Finding a standard method for purifying viral vectors is especially difficult when there are numerous varieties of viral vectors including adeno-associated viruses and lentiviruses. Whilst the demand for viral vector gene therapies have increased in certain disease areas such as Duchenne muscular dystrophy, safety concerns possibly relating to impurities in the product must be addressed. This interview took place at Advanced Therapies Week 2022.

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.

Cardiosphere-derived cells in myocardial regeneration

admin_vjr_user — Fri, 13 Aug 2021 12:05:07 +0000

Linda Marban, PhD, Capricor Therapeutics, Beverly Hills, CA, introduces CAP-1002, an allogeneic cardiosphere-derived cell (CDC) therapy that promotes the regeneration of cardiac muscle by stimulating the release of exosomes, thus inducing endogenous repair mechanisms, a reduction in inflammation and ultimately, muscle tissue generation. Clinical trial data indicates that CAP-1002 therapy can result in significant improvements in cardiac and skeletal muscle in patients with Duchenne muscular dystrophy (DMD). CDC-derived extracellular vesicles (EVs) are also being investigated as a therapeutic given their tropism to cardiac and skeletal muscle and ease of storage and delivery. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Improving patient access to advanced therapies: are we ready for them?

admin_vjr_user — Mon, 05 Jul 2021 13:47:09 +0000

Durhane Wong-Rieger, PhD, Rare Disease International, Toronto, ON, calls for the field of cell and gene therapies and healthcare systems to work towards improving patient access to therapies. The costs associated with novel therapies and the lack of established payment systems, as well as a lack of adequate infrastructure for the delivery of therapies, remain key obstacles for many patients, which will only be exacerbated as therapies for more common indications such as cardiovascular disease become approved in the future. This interview took place during the Cell & Gene Meeting on the Mediterranean 2021 virtual conference.

NextGen & GenX AAV vectors for DMD gene therapy

admin_vjr_user — Fri, 21 May 2021 17:21:47 +0000

Arun Srivastava, PhD, University of Florida, Gainesville, FL, explains that due to the high volume of muscle is the body, clinical trials investigating first-generation adeno-associated virus (AAV)-based gene therapies for muscular dystrophy require the use of high vector doses, resulting in the need for immune-suppression. In an effort to address this, next-generation (NextGen) AAVrh74 vectors and single-stranded generation X (GenX) AAV vectors have been developed. The encapsulation of the GenX genome containing the micro-dystrophin gene into NextGen AAVrh74 vector capsid, referred to as an optimized (Opt) AAVrh74 vector, is also being investigated and offers the potential to reduce vector doses while maintaining therapeutic efficacy for muscular dystrophy. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.