Sangamo Therapeutics Inc (NASDAQ:SGMO) entered an international licensing collaboration accord with Novartis AG (NYSE:NVS) to commercialize/ develop gene regulation therapies (GRT).
GRT targets ASD
Sangamo will use GRTs to address ASD (Autism Spectrum Disorder) and other neurodevelopmental disorders. The company will use its proprietary GRT and ZFP-TFs (zinc finger protein transcription factors) to upregulate the key genes responsible for neurodevelopmental disorders.
CEO of Sangamo, Sandy Macrae, said its team is capable of engineering zinc finger proteins to treat any genomic target. The company is developing a broad line of partnered and wholly owned programs to provide its genomic medicines to the patients. Sandy further said its zinc finger platform can address several neurological disease gene targets for the central nervous system.
Addresses challenging neurodevelopmental conditions
Sangamo’s collaboration accord allows the company to use the deep experience of Novartis in neuroscience drug development. The company can address several challenging neurodevelopmental conditions by joining forces with Novartis and expanding its pipeline.
The main target of Sangamo is to develop genomic medicines for patients suffering from neurodevelopmental disorders like autism. President of NIBR (Novartis Institutes for BioMedical Research), Jay Bradner, said the teaming agreement with Sangamo is part of its commitment to developing innovative neurodevelopmental treatments.
The onus is on developing GRTs that act at the genomic level and cure the most challenging neurodevelopmental disorders. ZFP-TF GRT of Sangamo is delivered with AAVs (adeno-associated viruses) for accomplishing desired therapeutic effects by activating specific gene expression at the DNA level.
Under the three-year collaboration accord, Novartis holds exclusive rights to Sangamo’s ZFP-TFs to address three undisclosed genes responsible for intellectual disability and ASD. It also has an option to license proprietary AAVs of Sangamo.
Novartis will provide funding support of $75 million to Sangamo for R&D and production activities. It will assume additional responsibilities for IND and research, regulatory interactions, clinical development, global communication, and production. Sangamo could earn up to $750 million for other commercial and development milestones.
Sangamo posts an increase of 23.29% YoY to $21.6 million in revenues in Q2 2020. Its net loss surged to $35.9 million in Q2 2020.
Sangamo also provided updated results of Phase 1/ 2 Alta clinical study that investigates SB-525 in patients suffering from severe hemophilia A. The company is conducting the study in alliance with Pfizer and obtained sustained Factor VIII activity levels with no bleeding.