Devyn Smith – VJRegenMed

The past, present & future of gene delivery

Simon Ng — Thu, 04 Nov 2021 18:04:43 +0000

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, shares his highlights from the “The past, present & future of gene delivery” session at Meeting on the Mesa 2021. Multiple approaches in delivering gene therapies have been successfully developed, including nanoparticles and adeno-associated virus (AAV) vectors, which will enable delivery of therapeutic products into new areas of the body. Dr Smith additionally focuses on the contents of these delivery systems, and how these treatments aim to permanently cure patients. This interview took place at Meeting on the Mesa 2021.

Expanding the repertoire of CRISPR-based genomic editors

Simon Ng — Thu, 04 Nov 2021 18:04:42 +0000

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, provides a brief overview of the tailored nuclease therapies developed at Arbor Biotechnologies. Multiple nucleases have been discovered that are superior to the original Cas9 endonuclease and a portfolio of nucleases, which have been modified to improve efficacy, has been developed. Certain nucleases can be adapted for specific diseases such as Huntington’s disease, a trinucleotide repeat disorder, by having the optimal protospacer adjacent motif (PAM). This interview took place at Meeting on the Mesa 2021.

Tailored nucleases for primary hyperoxaluria

Simon Ng — Thu, 04 Nov 2021 18:04:40 +0000

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, discusses utilizing tailored nuclease-based CRISPR therapies to treat primary hyperoxaluria, a genetic condition that leads to the accumulation of oxalate in the liver and subsequent end-stage renal disease in children. Current treatments have a mixed efficacy and new genetic therapies aim to treat primary hyperoxaluria by multiplex gene knockout, where the nuclease targets two known protein targets. This interview took place at Meeting on the Mesa 2021.

The future landscape of gene therapies

Simon Ng — Thu, 04 Nov 2021 18:04:39 +0000

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, outlines his opinions on how the gene therapy sector can advance. Whilst there are a small number of gene therapies that have been approved, future development needs to focus on increased durability of the treatments. Dr Smith additionally focuses on streamlining the manufacturing of gene therapies to enable commercialization, as well as novel delivery systems including synthetic vectors and nanoparticles to target more cell types. This interview took place at Meeting on the Mesa 2021.