MLSC Portfolio Spotlight
Valerion Therapeutics, based in Concord, MA, is a biotechnology company focused on developing targeted therapies for orphan genetic diseases. Valerion has developed a first in class and multifunctional antibody-delivery platform capable of efficiently targeting biotherapeutics to key tissues including neurons and skeletal muscle fibers. More importantly, the company’s proprietary platform delivers functional cargo to intracellular locations not previously accessible.
In 2010, Valerion, formerly 4s3 Bioscience, received $600,000 in funding through the MLSC’s Accelerator Loan Program. Just two years later, the company raised $20M and was able to pay back the loan, with interest, for a total of $685,134. “For us, the MLSC accelerator loan was a critical bridge that supported our growth from a small grant-funded startup to a preclinical company worthy of outside investment,” said Dustin Armstrong, Ph.D., a Valerion founder and Chief Scientific Officer. At the time of its award, Valerion had just 2 employees and the company now operates with over 25 full and part-time staff.
Valerion’s lead product, VAL-1221, an antibody-directed enzyme replacement therapy for Pompe disease, recently received regulatory approval to initiate a Phase 1/2 clinical study at Duke University. A second clinical site in the United Kingdom will be added later this month and topline results will be available by December of this year.
“VAL-1221 is uniquely designed to enhance muscle delivery and provide additional replacement enzyme to intracellular locations not currently served by existing Pompe disease treatments. More broadly, this first clinical study is an important step to further demonstrate the gateway potential of this platform to address a larger series of glycogen storage disorders and genetic deficiencies with no current therapies,” said Dr. Armstrong.
Valerion currently has a robust pipeline and is developing other preclinical candidates for indications such as Lafora disease and Myotubular Myopathy.