Envisagenics, a startup founded by scientists from Cold Spring Harbor Laboratory, has raised a $25 million series B round to develop drugs for cancer and neurodegenerative disease, the company told Endpoints News.
The New York company is based on the science of RNA splicing, a process that cells use to stitch small pieces of genetic code into an mRNA molecule. It’s a quirk of evolution that enables cells to mix and match pieces of code to create different versions of a protein from the same gene.
Scientists have already tapped the process to make medicines, including Biogen’s drug Spinraza, which treats spinal muscular atrophy by hijacking RNA splicing to create a backup version of a vital nerve protein.
Maria Luisa Pineda and Martin Akerman, two scientists who worked on the academic science that led to Spinraza, co-founded Envisagenics in 2014 to apply the concept to other diseases. “We’re trying to unlock that world of opportunity,” Pineda, the startup’s CEO, told Endpoints.
Bristol Myers Squibb chipped into the new funding, along with existing investors Third Kind Venture Capital, Empire State Development and Red Cell Partners, which participated in the startup’s $19 million series A round in 2019.
Envisagenics says it has built up a database of 14 million RNA splicing events from public and private sources. Some reflect normal biology, while others are implicated in diseases. Pineda said that artificial intelligence is key to quickly sifting through these vast possibilities to identify unusual splicing events that could become the basis of a drug program.
“It takes us one year to go from analysis all the way to a lead compound,” Pineda said.
Unusual RNA splicing can create new tidbits of proteins, called epitopes, displayed on the surface of cancer cells that are not found in normal cells. Those new epitopes can give drugmakers a much-needed foothold for honing potent antibody-drug conjugates, CAR-T cell therapies or radioligands onto a unique aspect of cancer cells.
It’s the discovery of those novel epitopes and antibody-like drugs that bind them, that’s the core output of Envisagenics. The startup has cancer-focused partnerships with Biogen, Bristol Myers Squibb, and Johnson & Johnson. Pineda wouldn’t disclose the financial or scientific details of those collaborations.
Pineda said the startup has cataloged 370 disorders where RNA splicing is implicated, with cancer as one of the biggest areas. She thinks aberrant RNA splicing could help explain why some tumors don’t have lots of DNA mutations. “If DNA errors are not in it, then what is it? It is RNA splicing events,” she said.
The startup’s new funding will help it grow its nearly 50-person team and push its first program into clinical trials by 2025, Pineda said. It currently has three of its own preclinical programs focused on immunotherapies for lung cancer, colorectal cancer, and blood cancers including acute myeloid leukemia and multiple myeloma.
Envisagenics also has two preclinical programs in ALS, a neurodegenerative disease. Like Spinraza, these programs use genetic drugs called antisense oligonucleotides to alter splicing. But unlike most experimental drugs for the neurodegenerative condition, which have focused on rare genetic subsets of the disease, Pineda said the programs are geared towards the majority of ALS patients with the sporadic form of the disease.