RNA editing

For an investment of up to $1.9 billion, Eli Lilly will be able to use Ascidian Therapeutics’ platform that removes mutated exons from mRNA molecules, avoiding the expression of disease-causing proteins.

Nobel laureate Sir Michael Houghton and colleagues at Applied Pharmaceutical Innovation (API) outline how rigorous early testing, smart IP and regulatory planning, and scalable CMC choices can help founders reach first-in-human faster.

In this episode of Denatured, Jennifer C. Smith-Parker speaks to Erik Digman Wiklund, CEO of Circio and Jacob Becraft, Co-founder and CEO of Strand Therapeutics. They discuss how post-COVID, emerging platforms like circular and logic circuit RNA are expanding the field’s therapeutic horizons.

After a cacophony of troubles hit the RNA editing biotech last fall, CEO Ram Aiyar is in San Francisco to develop partnerships, pitch the potential of its new AATD program and find more money to keep the dream alive.

Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.

Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company’s stock is down 80% on all the news.

Wave’s RNA editor resulted in protein levels that were “exceedingly close” to what investors were expecting, but nevertheless fell short of that bar, according to analysts at Truist Securities.

Arnatar is developing ART4, an antisense oligonucleotide that ups the expression of its target protein to treat a rare disease called Alagille syndrome.

There is no certainty that the buyout will come to pass, according to The Financial Times, which first reported the rumors.

ADARx Pharmaceuticals CEO Zhen Li found her way to biopharma through Merck, where she was inspired by the application of powerful science to human medicine.