New Biotech Launches as Part of a Father’s Quest to Get Treatment for Son

Rare Daily Staff

A father’s effort to find a treatment for his infant son has led to the launch of Astrosalva Therapeutics, a biotechnology company developing medicines for Alexander disease and cerebral adrenoleukodystrophy, two rare genetic diseases that damage the brain’s white matter.

In announcing its launch, the San Mateo, California-based company named Unravel Biosciences as its founding development partner. Unravel co-founder and CEO Richard Novak has also joined Astrosalva’s board of directors.

Unravel uses computational biology and patient-derived gene-activity data to identify potential treatments. The company describes its approach as creating molecular models of individual patients to predict how they might respond to therapies. Its pipeline spans neurodegenerative, neurodevelopmental, and metabolic disorders.

Astrosalva’s programs are in preclinical development. The company has no active clinical trial or expanded-access program through which patients can receive an investigational treatment.

The company is initially focused on Alexander disease and expanded its work to cerebral adrenoleukodystrophy, or cALD, after learning more about the challenges faced by families affected by rare neurodegenerative diseases.

Both conditions affect the brain’s white matter. In cALD, the fatty covering that insulates nerve fibers, called myelin, breaks down, interfering with the transmission of nerve signals. The childhood cerebral form typically affects boys and can cause learning and behavioral changes, followed by problems with movement, vision, speech comprehension, and swallowing. Progression can be rapid, leading to profound disability within a few years if untreated.

The underlying genetic condition, X-linked adrenoleukodystrophy, affects approximately one in 15,000 people worldwide, according to MedlinePlus. That figure includes several forms of the condition, not just cALD.

Thomas Wagner, whose son Max was diagnosed with Alexander disease at seven months old, co-founded the company with Paul Schmidt, a biopharma veteran who serves as its CEO.

“I read hundreds of research papers and wrote to the scientists who had written them, and many wrote back,” said Wagner.

Those exchanges helped turn his search for answers into a drug-development venture.

“Those conversations showed me how much promise there is in the research,” Wagner said. “Astrosalva exists to turn it into medicines, for Max and for every family facing these diseases.”