The role that genetic mutations play in causing disease is understood, and a large number of drug discovery efforts have focused on the molecules that address this abnormality. But the human genome also contains genes that suppress mutations, which Scenic Biotech believes are both valuable and druggable.This startup discovers molecules capable of hitting these new gene targets $31 million raised to advance their development in cancer and rare diseases.
Scenic’s research focuses on a type of gene called a genetic modifier. These genes affect how the disease appears and develops, either exacerbating its severity or suppressing it. According to the company, the presence of genetic modifiers helps explain why some people with genetic mutations associated with serious diseases end up with mild symptoms or no symptoms at all. Scenic is designed to address the ability to suppress disease using genetic modifiers.
Genetic modifiers are scattered among the estimated 20,000 genes that make up the human genome. Scenic’s first task is to find them. The company’s Cell-Seq platform identifies genetic modifiers of multiple diseases. The technology uses computational techniques to analyze genes and generate “disease maps” that can be used to identify potential targets for new drugs.
Scenic in Amsterdam is Founded in 2017, spun out from the Netherlands Cancer Institute and the University of Oxford. The company’s lead drug candidate is based on research at the Cancer Institute and Leiden University Medical Center.This immuno-oncology drug Targeting QPCTL, an enzyme in cancer cells that regulates the function of a protein called CD47. The protein sends a signal to macrophages, the immune cells that devour pathogens, that reads “don’t eat me.”
Scenic’s primary cancer program blocks QPCTL to prevent cancer cells from expressing CD47. Therefore, macrophages should be able to identify and track cancer cells. The prospect of CD47 as a drug target has led to several big deals. Gilead Sciences to acquire 47 for $4.9 billion 2020; Pfizer to acquire Trillium Therapeutics for $2.3 billion last year. Other companies developing CD47-targeted drugs include I-Mab and Arch Oncology. All of these efforts have focused on antibodies that block the protein. Scenic stands out with a small molecule approach that may have advantages over antibodies targeting CD47. Small molecules can be formulated into oral drugs. The company also said its small molecules may be able to treat solid tumors, which have been harder targets for biological drugs.
Scenic’s gene modifier research has also resulted in three programs targeting rare metabolic diseases. The first is Niemann-Pick disease type C, a disease that impedes the ability of cells to move cholesterol and other fatty substances. The accumulation of these substances in various tissues can be fatal. The second rare disease target is Barth syndrome, a mitochondrial disease that causes heart problems. These diseases still lack any FDA-approved therapy. In the past year, FDA rejects Orphayzme’s Neimann-Pick disease type C drug and issued Letter of Refusal to Submit Its Barth Syndrome Treatment Candidate to Stealth BioTherapeutics.
Scenic’s third rare disease target is unspecified severe inherited metabolic syndrome. Scenic said the new funding will help advance preclinical research across all three rare disease programs to support investigational new drug applications.
In addition to the internal drug pipeline, Scenic has also Partnership with Roche’s Genentech subsidiary. The partnership began in 2020 with the goal of identifying drug targets in multiple therapeutic areas.Last May, partner expand Their genetic modification alliance, but did not disclose financial or drug target details. In its funding announcement, Scenic said the collaboration spans six scientific fields.
The Series A round, announced Thursday, was co-led by Eir Ventures, BioMedPartners and Vesalius Biocapital. Early investors Inkef Capital, BioGeneration Ventures and Oxford Science Enterprises, as well as the founders and management of Scenic, also participated. In the funding announcement, Stephan Christgau, general partner at Eir Ventures, said that Scenic has the potential to become one of the most exciting biotech companies in Europe.
“It is a pioneer in a new field of groundbreaking and promising gene modifiers,” he said. “We are impressed with the power of its Cell-Seq platform and how it powers a portfolio of internal and collaborative projects across multiple therapeutic areas. This financing will allow Scenic to continue developing its platform and move its lead programs into the human clinic trials, while bringing its rare disease program to critical value infection points.”
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