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

January 4, 2022

- ADVM-062 is designed as a potential treatment for blue cone monochromacy (X-linked incomplete achromatopsia)

REDWOOD CITY, Calif., Jan. 04, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (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.  

Orphan drug designation is granted by the FDA to novel drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. The orphan drug designation also could entitle Adverum Biotechnologies to a seven-year period of marketing exclusivity in the United States for ADVM-062 should the company receive FDA approval for the treatment of BCM for this product candidate.

Anticipated Milestones for ADVM-062

  • Plan to present preclinical data supporting proof of concept of ADVM-062 in 1H2022.
  • Plan to submit an Investigational New Drug (IND) application by year-end 2022.

About ADVM-062 Gene Therapy

ADVM-062 is a gene therapy candidate specifically designed to deliver a functional copy of the OPN1LW gene to the foveal cones of patients suffering from BCM via a single IVT injection. ADVM-062 utilizes Adverum’s propriety vector capsid, AAV.7m8.  

About Adverum Biotechnologies

Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum’s lead gene therapy clinical candidate, ADVM-022, is being evaluated as a one-time, intravitreal injection for the treatment of patients with neovascular or wet age-related macular degeneration (wet AMD). For more information, please visit

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to the statements under the caption “Anticipated Milestones for ADVM-062.” Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the time and cost of product candidate development and regulatory uncertainties; and other risks and uncertainties facing Adverum described more fully in Adverum’s Form 10-Q filed with the SEC on November 4, 2021, under the heading “Risk Factors.” All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Anand Reddi
Vice President, Head of Corporate Strategy and External Affairs & Engagement
Adverum Biotechnologies, Inc.
T: 650-649-1358


Laurence Watts
Gilmartin Group
T: 619-916-7620

Source: Adverum Biotechnologies, Inc. 

Primary Logo

Source: Adverum Biotechnologies, Inc.