Navigation Links
Enzyvant to Develop Novel Biologic Therapy for Complete DiGeorge Syndrome
Date:1/6/2017

BASEL, Switzerland, Jan. 6, 2017 /PRNewswire/ -- Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced entering into an exclusive worldwide licensing agreement with Duke University to develop RVT-802, a tissue-based therapy for the treatment of complete DiGeorge Syndrome (cDGS), a rare and fatal congenital immunodeficiency disease. In the absence of treatment, children born with cDGS typically succumb to infections within the first two years of life due to their severe immunodeficiency.

"Patients with complete DiGeorge Syndrome lack effective treatment options, but this novel program offers the hope of a life-saving solution," said Chief Executive Officer Dr. Alvin Shih. "It is our responsibility to ensure that the children with this disease who need this treatment are able to receive it."

Under the terms of the agreement, data from Duke's experience will be used to support submission of a Biologics License Application (BLA) with the Food and Drug Administration (FDA) for approval. RVT-802 expands Enzyvant's pipeline of potentially transformative biologic therapies for rare genetic diseases with high unmet need.

Dr. M. Louise Markert, Professor of Pediatrics at Duke University, has led research on the treatment of immunodeficiency in patients with cDGS. The findings of Dr. Markert and her research team have been published in the New England Journal of Medicine as well as numerous other peer-reviewed scientific journals and clinical publications. "This is an encouraging development for the patients and parents confronting this disease," said Dr. Markert. "I look forward to working with Enzyvant to make this life-saving therapy more available to babies who so desperately need it."

About Complete DiGeorge Syndrome

Complete DiGeorge Syndrome (cDGS) represents approximately one percent of all patients with DiGeorge Syndrome, a congenital immunodeficiency disease. The three main characteristics of cDGS are congenital heart disease, hypoparathyroidism (leading to low calcium levels), and athymia (lack of thymus tissue). Athymia leads to severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGS is uniformly fatal if untreated, with death typically occurring in the first two years of life due to susceptibility to infection.

About RVT-802

RVT-802 is a biologic therapy that was initially developed by Dr. Markert and her colleagues using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency resulting from cDGS. Preliminary clinical results suggest a survival rate of over 70% for treated patients. RVT-802 has been granted orphan drug designation by the FDA and Enzyvant anticipates a potential BLA filing in 2018.

About Enzyvant

Enzyvant is a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases. Enzyvant is currently preparing a clinical trial of RVT-801, an investigational enzyme replacement therapy for the treatment of acid ceramidase deficiency (ACD), a rare lysosomal storage disease which manifests as Farber disease. Enzyvant is also planning to advance the development of RVT-802, an investigational biologic therapy for complete DiGeorge Syndrome. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.

Related Links
http://www.enzyvant.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/enzyvant-to-develop-novel-biologic-therapy-for-complete-digeorge-syndrome-300386922.html


'/>"/>
SOURCE Enzyvant
Copyright©2017 PR Newswire.
All rights reserved


Related medicine technology :

1. Guardant Health to create a new 500-plus-gene liquid biopsy panel with several leading pharmaceutical companies to accelerate clinical trials and drug development
2. Menlo Therapeutics Recruits Experienced Management Team and Board Leadership to Accelerate Development of Serlopitant for Treatment of Pruritus
3. Eisai Acquires All Global Development And Marketing Rights For Chronic Weight Management Treatment Lorcaserin HCI
4. Global Transdermal Prescription Gels Market: Essential Formulation Factors, Technologies and Market Development - Research and Markets
5. ImmuNext Enters into a Strategic Alliance to Develop Novel Therapies for Autoimmune Diseases
6. Global Diagnostic Imaging Market Size, Share, Development, Growth and Demand Forecast to 2022- Industry Insights by Type, by End User
7. Global Ultrasound Device Market Size, Share, Development, Growth and Demand Forecast to 2022- Industry Insights by Type, by Portability, by Display, by Technology, by Application
8. Global Neurostimulation Device Market Size, Share, Development, Growth and Demand Forecast to 2022- Industry Insights by Type, and by Application
9. Global Enteral Nutrition Market Size, Share, Development, Growth and Demand Forecast to 2022 - Industry Insights by Type
10. Daiichi Sankyo and DarwinHealth Announce Strategic Partnership to Deploy a Quantitative Systems Biology Discovery Platform to Prioritize Daiichi Sankyo Cancer Enterprise Compounds for Development
11. Sun Pharma & Moebius Medical Sign Exclusive Global Licensing Deal to Develop Novel Liposomal Non-opioid Pain Product for Osteoarthritis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/1/2020)... ... September 01, 2020 , ... Neil Oberfeld of global ... to the Board of Trustees and committee chair of the Governance Committee for ... Denver-based nonprofit organization that fights for the education, health, and financial stability of ...
(Date:9/1/2020)... ... September 01, 2020 , ... Breast and ovarian cancers are ... (CDC), About 3% of breast cancers (about 7,500 women per year) and 10% of ... and BRCA2 genes. Both genes are detectable in the Women’s Excellence Genetic Testing ...
(Date:8/31/2020)... TEL AVIV, Israel (PRWEB) , ... August 31, ... ... hub to specialize in wellness-related technologies, today announced that they have invested $400,000 ... start-up competition that ran in collaboration with the Global Wellness Institute (GWI). ...
Breaking Medicine Technology:
(Date:9/1/2020)... ... September 01, 2020 , ... ... in Glendale. While the office will be the first to bear her ... tucks, buttock augmentation (Brazilian butt lift), as well as rhinoplasty, facial rejuvenation, and ...
(Date:8/31/2020)... ... August 31, 2020 , ... ... (AHN), are joining biomedical engineers from Carnegie Mellon University (CMU) to develop a ... patients who have undergone open heart surgery. , The AGH/CMU team was recently ...
(Date:8/31/2020)... ... August 31, 2020 , ... Genesis Chiropractic Software and ... the launch of its integrated, HIPAA compliant, telehealth solution, available to US-based clients ... under 90 seconds. , According to the U.S. Department of Health & ...
(Date:8/31/2020)... ... , ... In an effort to give back to the community in which ... hotel room booked with a special new rate code to benefit children and families ... immediately. Donations will be applied to any room type, any time, now through ...
(Date:8/28/2020)... ... August 28, 2020 , ... WHAT , Hespiro™ is a ... respiratory conditions, including COVID-19, while reducing the need for scarce mechanical ventilators. It ... gas and scrubs it free of carbon dioxide in a completely closed “rebreather” ...
Breaking Medicine News(10 mins):