Vico Therapeutics Raises $31M to Fund Advances in Central Nervous System Treatments

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by David Melamed, PhD |

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Vico Therapeutics has raised $31 million in Series A financing to continue pursuing the development of RNA modulating technologies that could potentially act as treatments for central nervous system disorders.

It is the company’s first significant round of investment funding.

At the forefront of Vico’s plans is further developing its Antisense OligoNucleotides (AON) platform, a potential treatment for Huntington’s disease and spinocerebellar ataxia (SCA), both neurodegenerative disorders. The biotech company intends to advance its AON platform from the late preclinical stage to the first in-human tests by the end of 2021.

“This Series A funding will allow us to advance our lead program into the clinic and continue to build our capabilities as a leader in the development of novel therapies for serious CNS disorders,” Luc Dochez, founder and chairman of Vico, said in a press release.

“We see tremendous potential to advance the field and apply the breadth of our antisense oligonucleotide (AON) expertise to address severe neurological disorders,” he added.

Huntington’s disease is caused by a mutation in the HTT gene that codes for the protein huntingtin, which is believed to play an important role in nerve cells (neurons) in the brain.

The HTT gene has a naturally occurring region containing from 10 to 35 repeats of the DNA nucleotide sequence CAG.  However, people with Huntington’s have anywhere from 36 to 120 repeats of the CAG sequence, which can cause abnormalities in the final protein structure.

In protein coding, every three nucleotides in the sequence of a gene correspond to a unique amino acid in the composition of a protein. The sequence CAG codes for the amino acid glutamine, which is abbreviated as the letter Q.

Huntington’s is considered a polyQ disease — as are type 1 and type 3 SCA — since its cause is attributed to excess glutamine in a protein structure, due to the repeated CAG sequences.

AONs are a single stranded genetic material, designed in a lab with the ability to bind to RNA and influence the subsequent protein production. Of note, RNA is the intermediate product between DNA and protein.

In preclinical studies at Vico, its AON platform was found to reduce the levels of mutant polyQ protein in comparison with healthy protein, prompting the interest in developing the research.

The company also is in the early discovery phase for an RNA editing platform, which is being developed as a treatment for Rett syndrome, another rare neurodevelopmental disorder.

“We are looking forward to accelerating the development of our platform technologies around AON technology and RNA-modulation/editing to bring best-in-class therapies to patients,” Dochez said. “The Vico team built this company around a unique synergy between state-of-the-art molecular biology, AON chemistry laboratories and industry experience in successfully developing drugs based on AON technology.”

The funding was led by Life Science Partners (LSP) and Kurma Partners, with contributions by Pontifax, Droia Genetic Disease, Polaris Partners, Pureos Bioventures, and Idinvest Partners.

According to LSP, Vico has a number of strengths that contributed to the fundraising, including experienced researchers and top-of-the-line facilities. The infrastructure to promote treatments into clinical trials, and the wide ranging application for polyQ disease modifying technology also were cited by LSP.

“Vico is building a leading team and has a cutting-edge infrastructure to engineer best-in-class therapies for severe neurological conditions and accelerate their advancement into human clinical trials,” said Martijn Kleijwegt, a leading partner at LSP and a board member at Vico. “A major strength of Vico’s approach is the broad applicability to different polyQ diseases and the selectivity for mutant proteins.”