Sangamo’s (SGMO) has abundant positive news since it offered proof of concept and feasibility of its zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation approach and other approaches.
The most recent announcement was made early today by Sangamo and Pfizer (PFE) about a new collaboration. The cooperation aimed at developing a potential therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene. The firms will be using Sangamo’s zinc finger protein transcription factors (ZFP-TFs) in treating these neurodegenerative diseases. ALS and FTLD are neurodegenerative disorders caused by mutations in the C9ORF72 gene. This mutation ultimately leads to the deterioration of motor neurons in the case of ALS, or neurons in the frontal and temporal lobes.
Currently, there are no cures to halt or reverse the progression of ALS or FTLD. The C9ORF72 mutation is linked to approximately one-third of cases of familial ALS.
Sangamo’s ZFP-TF technology involves introducing an engineered zinc finger protein (ZFP) designed to identify and bind to a precise sequence of DNA, suppressing the mutated gene’s expression.
Sangamo and Pfizer will investigate allele-specific ZFP-TFs with the potential to differentiate the mutant C9ORF72 allele from the wild-type allele and to specifically downregulate the expression of the mutant form of the gene.
Terms of the New Collaboration
– Sangamo will receive a $12 million upfront payment from Pfizer.
– Sangamo will be responsible for the development of ZFP-TF candidates.
– Sangamo is eligible to receive potential development and commercial milestone payments of up to $150 million, as well as tiered royalties on net sales.
– Pfizer will be operationally and financially responsible for the research, development, manufacturing and commercialization for the C9ORF72 ZFP-TF program and any resulting products.
This collaboration was not the first this year between Sangamo and Pfizer. In May 2017, Sangamo and Pfizer entered into an exclusive, global collaboration and license agreement for the development and commercialization of potential gene therapy products for Hemophilia A, including SB-525, which entered the clinic in August 2017.
In addition to Pfizer’s agreement, Sangamo’s technologies attracted more firms, including Shire Pharmaceuticals (SHPG), for the treatment of Huntington’s disease, Bioverativ Inc.. – a spin-off of Biogen for the treatment of hemoglobinopathies, based on Sangamo’s ZFN-mediated genome editing cell therapy platform, specifically targeting rare blood diseases beta-thalassemia and sickle cell diseases.
Sangamo has also established strategic partnerships with Sigma-Aldrich (acquired by (Merck) and Dow AgroSciences for non-therapeutic uses of Sangamo’s technologies.
Sangamo made a huge comeback after its value has deteriorated and its stock tumbled at the hands of impatient investors, By using its strong scientific fundamentals, Sangamo was able to demonstrate the values of its technologies, bringing back life into its zinc finger approaches and attracting collaborators.
Sangamo’s stock, which Prohost had placed in its Aggressive Portfolio, was trading at $3.10 on January 3, 2017. The stock is trading today, January 3, 2018, at around $18.
We Expect SGMO to outperform.
The Prohost Letter Year in Review Issue #416 Part 2 will be posted on January 10, 2018. A lot of achievements will be highlighted which will help guiding us towards making accurate evaluations.
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