A blood-brain barrier shuttle company delivering RNA therapeutics to the CNS, led by ex-Alnylam/Aliada leadership.
Leading CRISPR discovery company co-founded by Feng Zhang and David Walt developing novel genome editors. In vivo programs in ALS and PH1 (patients dosed). $2B partnership with Chiesi.
UC Berkeley spinoff developing next-generation cell therapies using engineered biomaterials. Lead programme in Parkinson's Disease.
Developing polymer nanoparticles for non-viral delivery of RNA therapeutics to extrahepatic tissues. $644M partnership with Genentech.
Engineered growth factors for tissue-selective regeneration, initially focussed on the eye and the muscle.
Epigenome editors based on CasMINI, an ultra-compact CRISPR system. Currently in a Phase 1/2 trial for FSHD, a muscular dystrophy with no approved drug.
University College London spinout developing AAV gene therapy vectors to deliver engineered potassium channels directly to the seizure focus in focal refractory epilepsy.
Developing the first systemic protein therapy for the rare genetic condition Dystrophic Epidermolysis Bullosa (DEB).
Antibody-directed mRNA-LNP platform for non-viral delivery and in vivo gene editing, initially for sickle cell disease.
University of Maryland spinout developing tolerogenic microparticles for local induction of antigen-specific immune tolerance.
Proteinea employs proprietary AI models and computational tools to engineer next-gen antibodies with improved efficacy, safety and convenience.
Developers of a cell differentiation platform based on mimicking embryogenic conditions, initially for blood disorders. Based on the renowned work of Prof. Jacob Hanna.
Developing a small molecule therapy for hereditary hemorrhagic telangiectasia, the second largest genetic bleeding disorder.
Develops blood-brain barrier shuttles of RNA therapeutics using its multiplexed in vivo screening platform, initially for Dravet syndrome.
UC Berkeley spinout using directed evolution to engineer oncolytic viruses that unmask and destroy cancer cells in immunologically cold tumors.
