Sacramento, California – February 29, 2024
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.