Drug intelligence / Profile preview

rAAV-16L1

Development stage
Unknown
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intramuscular, Intravenous, Subretinal, Topical
01

Overview

Recombinant adeno-associated virus (rAAV) vector, likely engineered for gene delivery or gene therapy purposes. Recombinant AAVs are non-pathogenic viral vectors in which the native viral genes are replaced with a therapeutic gene of interest, while retaining the inverted terminal repeats necessary for packaging and vector function. Once administered, rAAV vectors deliver single-stranded DNA, which is converted to double-stranded episomes in the host cell nucleus, resulting in long-term and stable expression of the therapeutic gene without integrating efficiently into the host genome[1][2][3]. rAAV vectors can be tailored based on the choice of AAV serotype capsid and the transgene cassette; the "16L1" designation likely refers to a specific vector construct, cargo, or serotype variant, but no authoritative information is available confirming a well-recognized or marketed rAAV-16L1 construct in the literature or public databases.

Other names
rAAV-16L1rAAV16L1rAAV 16L1

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