A novel, small molecule therapeutics platform for addressing historically undruggable targets.

Reprogramming growth factors to treat rare disease.

Pioneering oligonucleotide based therapeutics to leverage RNA binding protein biology in CNS and other serious disease.

Revolutionizing in vivo gene delivery to usher in a new era of genetic medicines.

Developing novel antibody drug conjugates (ADCs) against novel targets in multiple solid tumors.

Pioneering a powerful new approach to improving the effectiveness of antibody-drug conjugates (ADCs) and other antibody-based therapeutics.

On a mission to revolutionize treatment options for patients with severe liver disease.