Helping early-stage teams know what their programs need next

Gene Petrella, Founder

Provided solutions across RNA research, development, and manufacturing lifecycle. Brokered partnerships and collaborations.

Led enabling technologies and IND-enabling in vitro pharmacology, including development of LNP–mRNA delivery approaches for engineered protein therapeutics and biomarker-driven precision medicine in autoimmune/inflammatory disease, resulting in one pending U.S. patent.

Leveraged scientific depth and network relationships to secure new clients and expand discovery biology capabilities.

Led platform initiatives spanning RNA editing and in vivo biology operations, including scaling internal capabilities and CRO execution models.

Founded TargetRNA, a start-up focused on enabling rapid discovery of small-molecule therapeutics targeting RNA. Led fundraising, team building, platform development, scientific strategy, and company operations.

Led RNA-targeting drug discovery efforts in neuromuscular diseases, spearheading a myotonic dystrophy program approved at the highest research level and catalyzing the institute-wide global RNA Initiative. 

Developed and deployed high-throughput biochemical, cellular, and imaging platforms, executed a 1.5M-compound high-throughput screening (HTS), and discovered novel small-molecule chemotypes that reversed disease phenotypes in patient-derived cellular models. 

Piloted muscle atrophy and facioscapulohumeral muscular dystrophy (FSHD) programs later adopted by the Musculoskeletal Disease Area, with compounds advancing toward clinical development.

Helped establish the Drug Discovery Incubator and co-founded the Academic HTS Partnership Program, building early discovery biology capabilities and leading a synthetic lethality screen in p53-mutant cancers that launched an institute-wide oncology program. 

Co-founded and led a proteomics-based target identification platform supporting multiple disease areas through mechanism-of-action and target discovery studies.

These contributions were recognized with the NIBR Team Award (2010) and NIBR Star Award (2013).

Advanced HTS-ready biochemical assays and developed protein-stability approaches that enabled determination of the first 3D structure of ZAP kinase.

Helped develop and industrialize ThermoFluor® (differential scanning fluorimetry/DSF) into a core discovery platform and led biophysics/HTS operations supporting internal programs and major partnerships. These contributions included five issued U.S. patents covering ThermoFluor®/thermal shift assay methods.

Gene Petrella, independent drug development advisor