Details:
JAG201 is being evaluated in preclinical studies for the treatment of SHANK-3 mutated autism spectrum disorder & Phelan-McDermid syndrome. It aims to deliver functional SHANK3 via the AAV9 vector to treat the root cause of the disease.
Lead Product(s): JAG201
Therapeutic Area: Genetic Disease Product Name: JAG201
Highest Development Status: IND Enabling Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 31, 2024
Details:
Proceeds will be used to advance the Company’s pipeline, including its lead Anti Sense Oligonucleotide product, SPL84-23. SPL84-23 to treat the 3849+10kb C->T CFTR mutation, that has showed ability to completely restore CFTR channel function in patient-derived cell cultures.
Lead Product(s): SPL84-23
Therapeutic Area: Genetic Disease Product Name: SPL84-23
Highest Development Status: IND Enabling Product Type: Large molecule
Partner/Sponsor/Collaborator: Biotel Limited
Deal Size: $28.5 million Upfront Cash: Undisclosed
Deal Type: Series B Financing May 13, 2021