BAX 335

consists of a self-complementary transgene flanked by AAV2-derived inverted terminal repeats, liver-specific transthyretin promoter/enhancers, and the codon-optimized complementary DNA (cDNA) encoding the hyperactive FIX (R338L) Padua variant

Also Known As:

Networked: 2 relevant articles (0 outcomes, 1 trials/studies)

Bio-Agent Context: Research Results

Experts

1.	Monahan, Paul E: 2 articles (11/2021 - 09/2018)
2.	Rottensteiner, Hanspeter: 2 articles (11/2021 - 09/2018)
3.	Scheiflinger, Friedrich: 2 articles (11/2021 - 09/2018)
4.	Bilic, Ivan: 1 article (11/2021)
5.	Chapin, John C: 1 article (11/2021)
6.	Escobar, Miguel A: 1 article (11/2021)
7.	Ewenstein, Bruce: 1 article (11/2021)
8.	Josephson, Neil C: 1 article (11/2021)
9.	Konkle, Barbara A: 1 article (11/2021)
10.	McPhee, Scott W J: 1 article (11/2021)

Related Diseases

1.

Hemophilia B (Haemophilia B) 11/01/2021 - "This phase 1/2, open-label dose-escalation study investigated BAX 335 (AskBio009, AAV8.sc-TTR-FIXR338Lopt), an adeno-associated virus serotype 8 (AAV8)-based FIX Padua gene therapy, in patients with hemophilia B. " 11/01/2021 - "BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression." 09/21/2018 - "The description of hyper-functional factor IX (FIX) Padua triggered the development of BAX 335, an AAV8-based hemophilia B gene therapy vector designed to compensate for low FIX protein expression levels by expressing the FIX Padua variant, thereby reducing the exposure to viral vector. "

Related Drugs and Biologics

1.	Proteins (Proteins, Gene)
2.	Factor IX (Coagulation Factor IX)