Richard Lewis, PhD is the Principal Regulatory Device & Biologics Expert at Hyman, Phelps & McNamara, P.C.

More info about the SCGE Meet the Expert series can be found at scge.mcw.edu/meet-the-exp...

#RegulatoryGuidance #RareDisease #Preclinical #Biotech #BiomedicalResearch

James Valentine, JD, MHS is a Director at Hyman, Phelps & McNamara, P.C. where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters relating to new drug and biologic development and approval.

With enhanced targeting versatility, Flex-Cas12a unlocks access to previously inaccessible genomic loci, providing new opportunities for both therapeutic and agricultural genome engineering. (3/3)

#Research #CRISPR #GeneEditing

To mitigate this constraint, SCGE researchers used a bacterial-based directed evolution assay combined with rational engineering to identify variants of Lachnospiraceae bacterium Cas12a with expanded PAM recognition. (2/3)

Our hope is that these documents can be useful to investigators that are developing gene editing therapies for rare diseases.

#Regulatory #GeneTherapy #RareDisease #BiotechResearch #BiomedicalResearch

The goal of the SCGE program is to accelerate the translation of genome editing therapies into the clinic. One of the ways we are working to achieve this goal is by publicly sharing successful strategies for starting clinical trials.

This discussion builds on their work in which they created a bespoke corrective gene-editing therapy for an infant's severe urea cycle disorder, and highlights the importance of regulatory innovation.

#GeneTherapy #Platform #PersonalizedMedicine #Regulatory

The AAV.k13 and AAV.k20 variants are promising vectors for therapeutic gene-transfer applications in kidney diseases and transplantation. (3/3)

Here SCGE researchers show that new variants of adeno-associated viruses (AAVs) displaying robust and widespread transduction in the kidneys of mice, pigs and non-human-primates can be obtained by evolving capsid libraries via cross-species cycling in different kidney models. (2/3)

Meet the Expert is a webinar series held by the Somatic Cell Genome Editing (SCGE) consortium to connect with experts involved in different aspects of gene therapy development and regulatory approval. Learn more at scge.mcw.edu/meet-the-exp...