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Few drugs exist that treat amyotrophic lateral sclerosis, a progressive disease that kills the nerve cells that allow patients to initiate and control muscle movement.

QurAlis, a Cambridge, MA-based startup, has an ambitious plan to develop a number of precision therapies for the disease based on forms of the condition identified by genetic mutation or a biomarker that CEO Kasper Roet (pictured) hopes to could one day, in combination, help most ALS patients.

Now the company has raised $42 million from investors in the US, Europe, and Japanmoney that will fund a move from the LabCentral incubator in Kendall Square to its own office, more than double the companys headcount by years end, and get at least one of its programs into human testing sometime next year.

The company is leveraging stem cell research from company co-founders Kevin Eggan and Clifford Woolf, Harvard University professors whoby harvesting normal skin cells from ALS patients and turning them into cells such as the motor neurons that damages as the disease progresseshave created models with the same DNA and gene mutations as those patients in an effort to identify new therapeutics for known ALS genes.

Mutations in more than 25 human genes have been implicated in ALS, the company says, and its strategy is to systematically investigate treatments targeting specific disease-causing mechanisms in patient subgroups. Some of those genes are also believed to cause frontotemporal dementia, a common cause of dementia that QurAlis also plans to treat.

One program QurAlis is advancing is intended for patients whose neurons are damaged and killed by the overactivation of certain receptors for glutamate, a key neurotransmitter, in a process known as excitotoxicity.

The company is also working on a treatment intended to return the autophagy process, through which cells recycle unwanted or damage components, to normal functioning. To do so, QurAlis is looking to target the enzyme TBK1, which plays a key role.

Roet, in an interview, said the company views its strategy as analogous to that pursued by Bostons Vertex Pharmaceuticals (NASDAQ: VRTX), which has developed multiple drugs for forms of cystic fibrosis (CF) caused by certain mutations, and late last year received approval for a combination of those drugs for about 90 percent of all CF patients.

We have identified ALS as a disease that we think we understand now, at least for specific subgroups of patients, he said. We understand what is driving the disease and we are able to develop very specific therapies for those patients.

Eggan, Woolf, Roet, and Jonathan Fleming launched QurAlis just over two years ago with seed funding from investors including MP Healthcare Venture Management, the investment arm of Mitsubishi Tanabe Pharma; the investment arm of Amgen (NASDAQ: AMGN); and Alexandria Venture Investments. Mitsubishi Tanabe markets edaravone (Radicava), one of four FDA-approved treatments for ALS. The FDAs 2017 nod for the drug made it the only ALS therapy OKd in the past 20 years.

The Cambridge, MA-based company said the new capital, a Series A financing round, brings the total it has raised to $50.5 million. The investment was led by LS Polaris Innovation Fund, Mission BioCapital, Dutch firm Inkef Capital, and the Dementia Discovery Fund. New investors including Droia Ventures, which operates from Luxembourg and Belgium, Mitsui Global Investment, and Dolby Family Ventures also participated, as did earlier investors including Amgen, MP Healthcare, and Sanford Biosciences.

As part of the deal, LS Polariss Amy Schulman, Inkef Capitals Roel Bulthuis, Dementia Discovery Funds Jonathan Behr, and Droia Ventures Luc Dochez join Mission BioCapitals Johannes Fruehauf on the QurAlis board.

Earlier this year some of the same investors, including Amgen and Dolby Family Ventures, backed a Series A financing for EnClear Therapies, a spinout of QurAlis. That company raised $10 million to advance the development of a dialysis-like medical device designed to filter out harmful proteins in the cerebral spinal fluid of patients with neurodegenerative diseases.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing - Xconomy