LYNNFIELD, Mass.--(BUSINESS WIRE)--Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that it is holding a scientific symposium “Rare Inherited Disorders of Pediatric Neurotransmitter Synthesis— Insights Into Diagnosis and Clinical Experience” at the 21st Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) May 19, 2018 in Chicago, IL, to support its disease awareness and education campaign for aromatic L-amino acid decarboxylase (AADC) deficiency. The symposium speakers are global leaders in pediatric neurological disorders, diagnostics, and patient advocacy. The event is chaired by Paul Hwu, MD, PhD, Professor of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan and features Toni Pearson, MD, Associate Professor of Neurology, Washington University, St Louis, MO, Keith Hyland, PhD, Executive Vice President, Medical Neurogenetics, Atlanta, GA, and Lisa Flint, Founder & Managing Director of the AADC Research Trust, Surrey, UK, as speakers. The symposium faculty will present important information about the genetic cause of AADC deficiency, the profound consequences of the disease, the differential diagnosis and laboratory tests available to accurately identify it, and the real-life experience of a parent caring for a child with AADC and the challenges they face.
“This symposium is an important event to raise the awareness and understanding of the devastating nature of AADC deficiency, its poor prognosis, its profound impact on patients and families, the likely underdiagnosis of the disease today, the diagnostic tools available to identify patients, and the importance of rapidly diagnosing the patients so they can receive appropriate care,” said Dr. Markus Peters, Agilis Chief Commercial Officer.
Agilis is developing innovative gene therapies for patients with CNS diseases for which few, if any, treatment options exist. Agilis’ lead program (AGIL-AADC) is an AAV-based gene therapy being investigated for the treatment AADC deficiency. AADC deficiency is a rare disease with a devastating clinical course arising from mutation in the DDC gene encoding the AADC enzyme responsible for the synthesis of dopamine and other neurotransmitters in the brain. Symptoms of AADC deficiency include the absence of motor milestone development, seizure-like events (termed oculogyric crisis) and hypotonia often described as “floppiness”. Because of the nature of symptoms, the disease can be confused with other, better known disorders such as cerebral palsy, making diagnosis difficult and often leading to a protracted diagnostic odyssey in correctly identifying the disease, and misdiagnosis.
ASGCT is one of the leading membership organization for scientists, physicians, professionals, and patient advocates involved in gene and cell therapy. The Society’s mission is to advance knowledge, awareness, and education leading to discovery and clinical application of gene and cell therapies to alleviate disease. ASGCT’s vision is to serve as a catalyst to transform medicine by advancing gene and cell therapies to benefit patients and society.
About AADC Deficiency
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic condition resulting in lack of functioning AADC enzyme responsible for the final step in the synthesis of key neurotransmitters dopamine (a precursor of norepinephrine and epinephrine) and serotonin (a precursor of melatonin). AADC deficiency results in developmental failure, global muscular hypotonia, severe, seizure-like episodes known as oculogyric crises, autonomic abnormalities, and the need for life-long care. Given this neurologically devastating illness, patients with severe AADC deficiency have a high risk for death during childhood. Treatment options for patients with AADC deficiency are limited and there are currently no approved therapies.
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, for which the company is preparing regulatory filings in the US and EU, Friedreich ataxia and Angelman syndrome, rare genetic diseases that include neurological deficits and result in physically debilitating conditions.
We invite you to visit our website at www.agilisbio.com.
Safe Harbor Statement
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.