01-18-2022

Adverum Biotechnologies Granted Orphan Drug Designation by FDA for Gene Therapy Candidate for the Treatment of Blue Cone Monochromacy

Adverum Biotechnologies (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate being developed as a potential single intravitreal (IVT) administration for blue cone monochromacy (BCM) by delivering a functional copy of the OPN1LW gene.

“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. We are pleased the FDA has recognized the patient need by granting Orphan Drug Designation and we look forward to advancing ADVM-062 towards the clinic and generating important data,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies.

BCM affects approximately 1 to 9 in 100,000 males, worldwide. This X-linked recessive hereditary condition is caused by mutations in either the L or the M opsin gene(s) and can manifest in loss of visual acuity, photophobia, myopia and infantile nystagmus that can persist into adulthood. Consequently, individuals with BCM have visual impairments to important aspects of daily living such as facial recognition, learning, reading, and daylight vision. Currently, there is no cure for BCM.  

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