AGTC-401 is under clinical development by Applied Genetic Technologies and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect AGTC-401’s likelihood of approval (LoA) and phase transition for Achromatopsia took place on 10 Feb 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their AGTC-401 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

AGTC-401 overview

Gene therapy is under development for the treatment of achromatopsia. It is administered through subretinal route as an injection. The therapeutic candidate uses a recombinant adeno-associated virus serotype 5 (rAAV5) vector expressing a human CNGB3 gene. It is developed based on Adeno-Associated virus (AAV) vector production technology platform.

Applied Genetic Technologies overview

Applied Genetic Technologies (AGTC) is a pharmaceutical company. The company Invovled in development of eye disease cures. AGTC develops treatments for patients that have diseases caused by broken genes. It offers products such X-linked retinitis pigmentosa, achromatopsia, bionic sight, age-related macular degeneration,non-syndromic hearing loss and deafness,frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company’s achromatopsia provides treatment for inherited conditions that are associated with visual acuity loss, extreme light sensitivity resulting in daytime blindness, and total loss of color discrimination. AGTC’s x-linked retinoschisis offers treatment in retinal degeneration affecting young males, presenting with poor vision by school age. The company also offers ophthalmology development programs and proof-of-concept data in multiple indications. AGTC is headquartered in Alachua, Florida, the US

Quick View AGTC-401 LOA Data

Report Segments
  • Innovator
Drug Name
  • AGTC-401
Administration Pathway
  • Intraocular
Therapeutic Areas
  • Genetic Disorders
Key Developers
  • Sponsor Company: Applied Genetic Technologies
  • Originator: Icagen
Highest Development Stage
  • Phase II


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.