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BioTech 365
MARCH 1, 2021
04, an Investigational Gene Therapy for Cystinosis AVR-RD-04 is the third AVROBIO investigational … Continue reading → AVROBIO Receives Orphan Drug Designation from the European Commission for AVR?RD?04,
Pharmaceutical Technology
SEPTEMBER 22, 2022
Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.
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Pharmaceutical Technology
JULY 14, 2022
Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II (MPSII) or Hunter syndrome. AVR-RD-05 had previously obtained rare pediatric disease designation from the FDA.
BioPharma Reporter
SEPTEMBER 23, 2021
This month sees the launch of a new gene therapy player, Opus Genetics, a company backed and spun out by leading patient group Foundation Fighting Blindnessâ venture arm, the Retinal Degeneration Fund (RD Fund).
STAT News
AUGUST 23, 2022
Image adapted from Pernkopf’s Atlas 3 rd Edition. Suddenly, as I was observing a beautiful illustration of the venous drainage of the brain, a name at the bottom of the screen stunned me. Read the rest…
Pharmaceutical Technology
MAY 23, 2023
In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration. The company will also provide knowledge transfer and other related services to support the programme’s transition.
BioSpace
DECEMBER 7, 2022
AVROBIO is planning a Phase II/III registrational trial for its Gaucher Disease Type 3 (GD3) gene therapy candidate, AVR-RD-02, in 2023.
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