FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

The draft guidance recommends that no more than 15 questions are included in the briefing package. CBER will not commit to reviewing packages greater than 250 pages. Where vector presence is detected, transgene mRNA and/or protein expression levels should also be measured.

Therapies

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Already having been in clinical studies at Roche, all four candidates have strong clinical and preclinical safety packages.

RNA

Drug Target Review

SEPTEMBER 10, 2024

However, developing an accelerated IND application requires that good laboratory practice (GLP) toxicological data must be generated, collected, interpreted and integrated in the IND-enabling data package. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.

Drug Development

Drug Target Review

MAY 22, 2024

Secondly, circRNAs can be engineered for more efficient protein expression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. What improvements in protein expression and durability have been demonstrated by the circVec 2.1

Therapies