Clinical trial participant receives first dose of UMass Chan-developed treatment for alopecia areata by Alys Pharmaceuticals

A siRNA-based therapy for alopecia areata developed by Anastasia Khvorova, PhD; John Harris, MD, PhD’05; Julia Alterman, PhD; and Qi Tang, PhD, at UMass Chan Medical School and licensed by Alys Pharmaceuticals Inc, has been administered to the first participant in a Phase IIa clinical trial.

Alys Pharmaceuticals, a Boston-based biotechnology company formed from the aggregation of Aldena Therapeutics, Graegis Pharmaceuticals, Granular Therapeutics, Klirna Biotech, NIRA Biosciences and Vimela Therapeutics, will trial ALY-101, the first clinical candidate from its pipeline of siRNA molecules developed for dermatological applications.

“This is a fantastic milestone for our teams,” said Dr. Khvorova, the Remondi Family Chair in Biomedical Research and professor of RNA therapeutics at UMass Chan. “These siRNA molecules have been designed for precise target specificity, stability, enhanced skin retention and long-lasting clinical efficacy while minimizing systemic exposure, which is critical for safety across a broad range of clinical indications.” 

The Phase IIa trial will assess if ALY-101 is safe and well tolerated following injections to the scalp in subjects with alopecia areata. Participants will randomly receive treatment versus placebo in four-week cycles during the eight-week trial. The study is expected to be completed in 2026.  

Alopecia areata is a chronic autoimmune skin disease with unmet medical need that often leads to significant psychological distress and a reduced quality of life. In patients with the disease, the immune system attacks healthy hair follicles, which leads to hair loss from some areas of the body, including the scalp. It is the second most common form of hair loss and affects nearly 7 million people in the United States, 20 percent of whom are children. It is not known what triggers the autoimmune reaction, though there is believed to be a genetic component to the disease, as 20 percent of patients have a family history of the condition. While medications and other treatments can help patients with alopecia areata manage symptoms, there is no cure for the disease.  

“We are thrilled to be partnering with Alys Pharmaceuticals to bring siRNA-based therapies to patients suffering from alopecia areata and other dermatological diseases,” said Dr. Tang, assistant professor of dermatology. “Alys Pharmaceuticals’ focus on dermatological innovation provides a clinical path forward for stable siRNA chemistries developed at UMass Chan.” 

The siRNA drug in the trial is a result of a collaboration between the Harris and Khvorova labs, and was led by Dr. Tang, who was a postdoctoral associate in both labs at the time. Dr. Alterman, assistant professor of RNA therapeutics, led a team in collaboration with Alys Pharmaceuticals, which has advanced the drug into clinic.

“This research is a prime example of the collaborative nature at UMass Chan,” said Dr. Harris, the Lambi and Sarah Adams Chair in Genetic Research, chair and professor of dermatology and co-founder and chief innovation officer of Alys Pharmaceuticals. “Working between labs and with industry leaders, we are developing new treatments that we can bring to patients suffering with disease.” 

ALY-101 is a small interfering RNA that shuts down or represses JAK1, a human tyrosine kinase protein essential for signaling cytokines that activate the immune system. The initial application of the lipid-conjugated JAK1 siRNA molecule is being delivered through intradermal injection. Topical applications of this siRNA-based platform are expected to reach clinical stage in 2026 for other diseases such as atopic dermatitis and vitiligo.  

“We are very pleased that our collaboration with the world-class scientists at UMass Chan Medical School has led to these first steps for bringing a siRNA-based alopecia areata treatment to patients,” said Thibaud Portal, PhD, CEO of Alys Pharmaceuticals. “This first clinical trial highlights the success of our collaborative work; it is a major milestone for Alys Pharmaceuticals as we continue building our diverse pipeline of innovative dermatological products.” 

Huseyin Mehmet, PhD, executive director of new ventures in BRIDGE Innovation and Business Development at UMass Chan, which facilitated the license with Alys Pharmaceuticals, added, “Our partnership with Alys Pharmaceuticals has been extremely fruitful. We are excited to see technology developed at UMass Chan Medical School, a leader in RNA-based therapeutics, advancing for the benefit of patients.”