Partnership to ensure material manufacturing for RVL001 trials

Investigational therapy will be tested in proof-of-concept trials in US, Colombia

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Unravel Biosciences has teamed up with Quality Chemical Laboratories (QCL) to manufacture materials for clinical trials of RVL001, an investigational therapy for Rett syndrome.

RVL001 (vorinostat) will be tested in proof-of-concept trials in the U.S. and Colombia. Last year, the U.S. Food and Drug Administration (FDA) provided positive feedback to Unravel about such a study, along with guidance about starting a trial in Rett syndrome, which affects brain function and results in cognitive, emotional, sensory, and motor disturbances, along with problems with the heart, breathing, and digestion. The disease occurs in one in 10,000 female births.

The QCL partnership is intended to guarantee that the production of key Unravel-owned technology follows good manufacturing practice (GMP) standards to make sure clinical material is of  high quality.

“We are pleased to announce yet another key milestone in our RVL001 clinical development program,” Richard Novak, PhD, Unravel’s co-founder and CEO, said in a company press release. “The initiation of manufacturing clinical trial material is a key remaining step to initiating clinical testing in our global Rett syndrome RVL001 trials.”

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What does Unravel do?

Unravel integrates artificial intelligence systems with screening in living organisms and a clinical validation platform for newly identified therapeutic targets.

Scientists at Unravel first analyze patients’ unique RNA signature to build a computational network using systems biology and the company’s model of human health. Unravel’s platform BioNAV combines target and drug discovery, preclinical screening, and patient subgroup classifications to find therapies for complex diseases.

According to Unravel, its approach involves de-risking therapeutic mechanisms by using the off-target effects of existing drugs.

“QCL is pleased to leverage its strong synthetic and analytical chemistry expertise to help Unravel move the RVL001 program into the clinic,” said Yousry Sayed, PhD, president and CEO of QCL. “Our capabilities will allow Unravel to efficiently initiate their clinical trial programs in the U.S. and overseas with a rapid scale-up as needed of clinical and commercial supply as the program advances.”

Unravel is also working on RVL002 a potential small molecule therapy that targets the metabolism of mitochondria, the structures in the cell that are responsible for producing energy, and also discovered RVL027, a molecule that may treat movement disorder that causes the muscles to contract involuntarily, called dystonia, using a new mechanism.