Roquefort Therapeutics (ROQ) , a biotech company focused on next generation medicines for hard to treat cancers, has successfully developed new siRNA sequences and new patent filing for its family of novel anti-cancer siRNA therapeutics. siRNA therapeutics are an innovative new class of medicines based on RNA interference (RNAi).
The portfolio of pre-clinical siRNA anti-cancer medicines was acquired in September 2022, following the company's acquisition of Oncogeni. Since the portfolio acquisition, four additional siRNA sequences have been developed. These new siRNA sequences attack the targets STAT-6 (Signal Transducer and Activator of Transcription) and its SH2 (Src-homology-2) domain.
Roquefort's siRNA targets STAT-6 and SH2 received a significant endorsement from Sanofi, by validating the effectiveness of these siRNA targets through a licensing deal with Recludix. The deal involved a $125 million upfront payment and had a total value of up to $1.2 billion for a pre-clinical stage development program that targets the SH2 domain of STAT6.
Additionally, Roquefort Therapeutic's siRNA programs, which are also in pre-clinical development, focus on targeting STAT-6 and the SH2 domain. These programs have shown promising in vivo anti-cancer activity in validated colon cancer models.
Ajan Reginald, Chief Executive Officer of Roquefort Therapeutics, said: "We are encouraged by the recent commercial validations of both siRNA as a therapeutic modality and STAT-6 and SH2 as highly attractive medicinal targets. The recent multi-billion dollar licensing deals indicate a high level of interest in this niche in which we were early innovators and were able to acquire strong patent positions before the market valued these asset classes. Our siRNA programs target the very attractive SH2 domain and have demonstrated highly specific efficacy in validated STAT-6 models. The patents that we have recently filed consolidate our leadership position in targeting STAT-6 and SH2 with siRNA.”
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This is an exciting development from Roquefort, which is well-aligned with the optimism that surrounds siRNA as a breakthrough therapeutic tool within the industry. siRNA works by 'knocking down' the genes which are directly or indirectly responsible for the abnormal proliferation of cancerous cells.
The recent validation of targets such as STAT-6 and the emergence of innovative modalities like siRNA are commanding substantial deal values, as they offer huge potential to pave the way for groundbreaking medications, potentially generating revenues in the multi-billion dollar range.
Roquefort’s portfolio is positioned to create significant shareholder value in this market, with its portfolio spanning newly validated targets (STAT-6 and Midkine) as well as novel modalities (siRNA, mRNA and cell therapy).
Its novel modalities portfolio has recently been reaping rewards. In June, Roquefort concluded successful in vitro studies for its anti-cancer mRNA therapeutic, focusing on breast and liver cancer, with studies demonstrating a significant reduction in cancer growth and migration, holding the potential to be a groundbreaking medical treatment once it progresses through clinical trials.
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