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Rare Diseases: Applied Genetic Technologies Corporation And Gene Therapy

December 29, 2014

Applied Genetics Technologies Corporation (AGTC), a Florida-based clinical stage biotechnology company, is developing Adeno-Associated Virus (AAV)-based gene therapies for rare inherited eye diseases. AGTC’s preclinical product candidates are for the following rare eye diseases:

•   X-Linked Retinoschisis (XLRS)

•   Achromatopsia (ACHM)

•   X-Linked Retinitis Pigmentosa (XLRP).

XLRS is an inherited form of retinal degeneration affecting young males, resulting in poor vision at a young age and worsening in teenage years. Achromatopsia is an inherited condition that results in visual acuity loss, light sensitivity, and loss of color discrimination. XLRP is an inherited disorder that causes boys to develop night blindness by age 10 and progresses to legal blindness by the 40’s.

AGTC uses gene therapy to replace the damaged gene with a normal functional gene, resulting in the patient producing proteins to treat their illness. The company uses the Adeno-Associated Virus (AAV) technology that delivers “good healthy” copies of the gene, replacing defective copies. Per AGTC’s website :

“AGTC has developed an AAV vector manufacturing platform comprised of a proprietary, patented production method and proven downstream purification operations integrated into a GMP-compliant format capable of AAV vector generation at a commercial scale.  The manufacturing platform is flexible and efficient, accommodating multiple genetic targets and AAV variants, or serotypes, while producing vector in sufficient quantity for treatment of both confined-space and systemic disease candidates.  AGTC is currently partnering with SAFC Pharma to make its proprietary production system available to potential academic and corporate partners.”  

AGTC’s FDA & EU Orphan Drug Designations

# Indication FDA ODD EU ODD 2015 Clinical Trials
1 X-Linked Retinoschisis (XLRS) May 2007 Yes Initial Clinical Data Mid-2015
2 Achromatopsia (ACHM) Feb 2011 Yes CNGB3 Gene: Initial Clinical Data Late 2015CNGA3 Gene: IND-enabling studies 2nd half 2015
3 X-Linked Retinitis Pigmentosa.(XLRP) No No Additional Pre-Clinical Studies 2014-2015

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Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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