LONDON, UK and NEW YORK, NY, USA I October 31, 2018 I MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced an exclusive licensing agreement with the National Institute of Dental and Craniofacial Research (NIDCR), a division of the National Institutes of Health (NIH), an agency of the United States Department of Health & Human Services. Under the agreement, MeiraGTx will receive worldwide rights to adeno-associated virus vector mediated gene delivery of aquaporin-1, designated AAV-AQP1, for Sjögren’s syndrome patients with associated xerostomia (dry mouth) or xerophthalmia (dry eyes). The license agreement includes standard and customary US Government terms and provisions. MeiraGTx and the NIDCR are currently partnered in two cooperative research and development agreements (CRADAs), one supporting preclinical work investigating AAV-AQP1 as a treatment for inadequate salivary gland function associated with Sjögren’s syndrome, and the other covering an ongoing Phase 1 dose escalation clinical study of AAV-AQP1 in patients with grade 2 or 3 radiation-induced xerostomia.
Sjögren’s syndrome is a systemic autoimmune disorder that impairs one’s ability to secrete fluids such as saliva and tears. Symptoms include dry mouth, which can cause difficulty speaking, tasting food or swallowing, and dry eyes, which can be associated with itching, burning, blurry vision or intolerance to fluorescent lighting. Other serious symptoms can occur in Sjögren’s syndrome, such as fatigue and chronic pain or damage to major organs.
“With as many as 4 million Americans living with Sjögren’s syndrome, it is one of the most common autoimmune diseases, yet there are few therapeutic options. Sjogren’s syndrome can be debilitating for patients and significantly impact their quality of life,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “New treatment options for this disorder are urgently needed and we are very excited to continue working closely with the NIH on AAV-AQP1.”
About Sjögren’s Syndrome
Sjögren’s syndrome is an autoimmune disorder which primarily attacks the glands that produce tears and saliva, impairing ability to secrete these fluids. The most common symptoms include dry eyes, dry mouth, fatigue, chronic pain and in severe cases result in damage to major organs. Sjögren’s syndrome is often difficult to diagnose as characteristic features of this disorder can also be caused by many other conditions. It’s estimated that Sjögren’s syndrome affects more than 4 million Americans, and while the disorder can appear in both sexes and at all ages, Sjögren’s syndrome is 10 times more prevalent in women than men. There is currently no cure for Sjögren’s syndrome.
About MeiraGTx
MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.
For more information, please visit www.meiragtx.com.
SOURCE: MeiraGTx
Post Views: 43
LONDON, UK and NEW YORK, NY, USA I October 31, 2018 I MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced an exclusive licensing agreement with the National Institute of Dental and Craniofacial Research (NIDCR), a division of the National Institutes of Health (NIH), an agency of the United States Department of Health & Human Services. Under the agreement, MeiraGTx will receive worldwide rights to adeno-associated virus vector mediated gene delivery of aquaporin-1, designated AAV-AQP1, for Sjögren’s syndrome patients with associated xerostomia (dry mouth) or xerophthalmia (dry eyes). The license agreement includes standard and customary US Government terms and provisions. MeiraGTx and the NIDCR are currently partnered in two cooperative research and development agreements (CRADAs), one supporting preclinical work investigating AAV-AQP1 as a treatment for inadequate salivary gland function associated with Sjögren’s syndrome, and the other covering an ongoing Phase 1 dose escalation clinical study of AAV-AQP1 in patients with grade 2 or 3 radiation-induced xerostomia.
Sjögren’s syndrome is a systemic autoimmune disorder that impairs one’s ability to secrete fluids such as saliva and tears. Symptoms include dry mouth, which can cause difficulty speaking, tasting food or swallowing, and dry eyes, which can be associated with itching, burning, blurry vision or intolerance to fluorescent lighting. Other serious symptoms can occur in Sjögren’s syndrome, such as fatigue and chronic pain or damage to major organs.
“With as many as 4 million Americans living with Sjögren’s syndrome, it is one of the most common autoimmune diseases, yet there are few therapeutic options. Sjogren’s syndrome can be debilitating for patients and significantly impact their quality of life,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “New treatment options for this disorder are urgently needed and we are very excited to continue working closely with the NIH on AAV-AQP1.”
About Sjögren’s Syndrome
Sjögren’s syndrome is an autoimmune disorder which primarily attacks the glands that produce tears and saliva, impairing ability to secrete these fluids. The most common symptoms include dry eyes, dry mouth, fatigue, chronic pain and in severe cases result in damage to major organs. Sjögren’s syndrome is often difficult to diagnose as characteristic features of this disorder can also be caused by many other conditions. It’s estimated that Sjögren’s syndrome affects more than 4 million Americans, and while the disorder can appear in both sexes and at all ages, Sjögren’s syndrome is 10 times more prevalent in women than men. There is currently no cure for Sjögren’s syndrome.
About MeiraGTx
MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.
For more information, please visit www.meiragtx.com.
SOURCE: MeiraGTx
Post Views: 43
