Tags : In Vivo


Sarepta Signs a License Agreement with Stride Bio to Develop

Shots: The companies collaborated to develop in vivo AAV-based therapies for up to eight central nervous system and neuromuscular targets. StrideBio will be responsible for research, development and manufacturing of the first four CNS targets which include MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick) Stride Bio to receive $48M upfront […]Read More


Bluebird Bio Signs a Three-Year Research Collaboration with Novo Nordisk

Shots: Bluebird Bio to utilize its mRNA-based megaTAL technology providing a specific and efficient way to silence, edit or insert genetic components combining Novo Nordisk’s deep expertise in hemophilia research and therapeutics  The focus of the collaboration is to identify gene therapy candidates for people with severe genetic diseases with initial interest of correcting FVIII-clotting […]Read More


ProBioGen and CRISPR Therapeutics Signs a Multi-Year Research Collaboration for

Shots: The companies collaborated to develop and commercialize novel in vivo delivery of CRISPR/Cas9 using ProBioGen’s technology. CRISPR Therapeutics also holds an option to license the developed product The focus of the agreement is to expand CRISPR Therapeutics’ in vivo delivery technology in fields of hemoglobinopathies, oncology, regenerative medicine and rare diseases CRISPR Therapeutics’ CRISPR/Cas9 […]Read More