Adverum receives orphan drug designation for blue cone monochromacy treatment
ADVM-062, a novel gene therapy candidate, received orphan drug designation from the FDA for the treatment of blue cone monochromacy, according to a press release from Adverum Biotechnologies.
The therapy is designed to deliver a copy of the OPN1LW gene to the foveal cones with a single intravitreal injection.
“We are excited to introduce ADVM-062, which builds on our ophthalmology and gene therapy expertise to address the significant unmet medical needs of patients living with blue cone monochromacy,” Laurent Fischer, MD, president and CEO of Adverum, said in the release.
Blue cone monochromacy, an X-linked recessive hereditary condition that affects one to nine in 100,000 males worldwide, can cause loss of visual acuity and photophobia, as well as other visual impairments.
Adverum plans to present preclinical data to support proof of concept of ADVM-062 in the first half of 2022 and submit an investigational new drug application by the end of the year.
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