FEATURE STORY
● The National Gene Vector Biorepository. Eleven years of providing resources to the gene therapy community
GENE THERAPY
● Recommendations for the development of cell-based anti-viral vector neutralizing antibody assays
DISRUPTIVE TECHNOLOGIES
● A small molecule-controlled Cas9 repressible system
● CRISPR/Cas9: targeted genome editing for the treatment of hereditary hearing loss
CLINICAL TRIALS - DATA
● AGTC reports positive six-month data from its ongoing Phase I/II clinical trial in X-Linked Retinitis Pigmentosa
INDUSTRIAL LANDSCAPE & AGREEMENTS
● Empirico scores $660M deal with Ionis and lands series A-2 financing
● MiNA Therapeutics announces research collaboration with AstraZeneca in metabolic diseases
● Avacta and Daewoong Pharmaceutical form immunotherapy focused joint-venture
● EQRx launches to create innovative medicines at dramatically lower prices for people, healthcare systems and society
MISCELLANEOUS
● ICER posts draft scoping document for the assessment of treatments for beta-thalassemia