Blue Gen Therapeutics Foundation Secures Exclusive License for Promising Gene Therapy Programs from Adverum Biotechnologies

UNITED STATES, February 29, 2024 /EINPresswire.com/ -- Blue Gen Therapeutics Foundation (BGTF), a non-profit organization dedicated to advancing gene therapy treatments for rare inherited retinal diseases, is proud to announce the exclusive license of two gene therapy programs from Adverum Biotechnologies (Nasdaq: ADVM). This acquisition includes the rights to ADVM-062 and ADVM-082, two innovative intravitreal gene therapies with the potential to revolutionize the treatment landscape for Blue Cone Monochromacy and Achromatopsia, respectively.

"We are thrilled to secure this license with Adverum Biotechnologies, which marks a significant milestone in our mission to address the urgent needs of patients with rare inherited retinal diseases," said John Cavitt, co-founder and CEO of Blue Gen Therapeutics Foundation and father of three children affected by Blue Cone Monochromacy. "As parents intimately familiar with the challenges faced by families impacted by these conditions, my wife, Leah, and I are personally invested in advancing these cutting-edge therapies to bring hope and healing to patients and their loved ones."

As part of the transaction, Adverum is granting BGTF an exclusive worldwide license to ADVM- 062 and ADVM-082. ADVM-062 is an intravitreal gene therapy that encodes a functional copy of the long-wavelength sensitive opsin protein for the treatment of Blue Cone Monochromacy. ADVM-082 is an intravitreal gene therapy that encodes a functional CNGB3 gene for the treatment of Achromatopsia. Both programs utilize Adverum’s proprietary AAV.7m8 capsid.

ADVM-062, with its compelling pre-clinical data, FDA Orphan Drug Designation (ODD) status, and a Good Manufacturing Practice lot, presents a robust program progressing to IND. This therapy holds immense promise in addressing the complex needs of individuals living with Blue Cone Monochromacy, a rare inherited retinal disease characterized by severe vision impairment.

"We are confident that Blue Gen Therapeutics Foundation, under the leadership of John Cavitt and Dr. Szilárd Kiss, is uniquely positioned to advance the development of ADVM-062 and ADVM-082," commented Laurent Fischer, M.D., President and CEO of Adverum Biotechnologies. "Their dedication to the rare disease community, combined with the expertise they have assembled in gene therapy research and clinical development, makes them an ideal team to drive forward the development of these groundbreaking therapies addressing a clear unmet medical need."

"Built upon the success of Adverum Biotechnologies' pioneering work in gene therapy, the ADVM-062 and ADVM-082 programs leverage the established efficacy and safety profile of the AAV.7m8 capsid," added Szilárd Kiss, MD, co-founder and Chief Medical Officer of Blue Gen Therapeutics Foundation. "These therapies have the potential to transform the lives of patients living with Blue Cone Monochromacy and Achromatopsia, and we are dedicated to bringing them to the clinic as swiftly and safely as possible."

About Blue Gen Therapeutics Foundation: Blue Gen Therapeutics Foundation (BGTF) is a non-profit organization headquartered in Sacramento, California, dedicated to advancing gene therapy treatments for rare inherited retinal diseases. With a mission to bring hope and healing to individuals and families affected by these conditions, BGTF focuses on accelerating the development and accessibility of innovative gene therapies.

For media inquiries, please contact: Info@bgtfoundation.org.

John Cavitt
Blue Gen Therapeutics Foundation
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