USF Research News

PTC Therapeutics Acquires USF Incubator Member Agilis Biotherapeutics, Inc.

August 2, 2018

Agilis' gene therapy platform expands and diversifies PTC Therapeutics' current pipeline

TAMPA, Fla. – Agilis Biotherapeutics, Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system (CNS), announced plans to be acquired by global biopharmaceutical company PTC Therapeutics. With this acquisition, PTC Therapeutics will obtain ownership of Agilis Biotherapeutics' four gene therapy programs.

Agilis' lead gene therapy candidate, GT-AADC, has compelling clinical data in treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. AADC Deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene. In its severe forms, AADC Deficiency causes the inability to develop any motor control (global muscular hypotonia/dystonia) resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care.

"I am proud of the accomplishments achieved by Agilis culminating with this value-creating transaction," said Mark Pykett, DVM, Ph.D., President and Chief Executive Officer of Agilis Biotherapeutics, Inc. "PTC provides a global infrastructure and proven capabilities, which we believe will enable our goal of delivering innovative therapies to patients suffering from rare CNS disorders. We look forward to the advancement of the Agilis programs and to providing value to patients."

Agilis Biotherapeutics joined the University of South Florida's Tampa Bay Technology Incubator (TBTI) in 2015, where the company focused on advancing gene therapy for AADC deficiency, Friedreich's ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions.

"We appreciate the support of the TBTI and have had a very positive experience in the Incubator program and with USF," said Pykett. "The TBTI resources and people, along with funding opportunities like the Florida High Tech Corridor's Matching Grant program, can help companies progress to the next level."

USF's TBTI supports technology research as a catalyst for economic development and facilitates the growth and development of USF spinout companies and community startups through their early stage commercialization with facilities, access to costly scientific equipment, state of the art shared wet lab space, and—most importantly—programming.

"We are so excited for Agilis Biotherapeutics and their accomplishments," said Valerie Landrio McDevitt, USF associate vice president. "The goal of TBTI is to increase the capacity for companies to be successful by providing entrepreneurial support programs, services, funding opportunities and physical infrastructure."

About Agilis Biotherapeutics, Inc.
Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis' gene therapies are engineered to impart sustainable clinical benefits by inducing persistent expression of a therapeutic gene through precise targeting and restoration of lost gene function to achieve long-term efficacy. Agilis' rare disease programs are focused on gene therapy for AADC deficiency, Friedreich ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions.

About the University of South Florida
University of South Florida, established in 1956 and located in Tampa, is a high-impact global research university dedicated to student success. The USF System includes three separately accredited institutions: USF, USF St. Petersburg and USF Sarasota-Manatee. Serving more than 50,000 students, the USF System has an annual budget of $1.8 billion and an annual economic impact of $4.4 billion. USF ranks in the top 30 nationally for research expenditures among public universities, according to the National Science Foundation. In 2018, the Florida Board of Governors designated USF as a Preeminent State Research University, placing USF in the most elite category among the state’s 12 public universities. USF is a member of the American Athletic Conference.