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Direct administration of AAV-based Gene Therapies to the central nervous system (CNS) can increase transduction and expression in the CNS. However, even with direct administration, vector "escapes" the CNS and distributes to peripheral organs.
Lead Product(s): AAV-based Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 16, 2022
Details:
Lonza has worked with Affinia Therapeutics to achieve a four-fold improvement in yield for the company’s lead capsid, Anc80L65. Lonza and Affinia Therapeutics are partnering with the goal of ensuring flexible manufacturing capacity for the next several years.
Lead Product(s): Anc80L65
Therapeutic Area: Genetic Disease Product Name: Anc80L65
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Lonza Group
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement September 08, 2021
Details:
Proceeds to support continued development of proprietary platform for rationally designed adeno-associated virus vectors (AAV) and advance programs into the clinic.
Lead Product(s): AAV-based gene therapies
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: EcoR1 Capital
Deal Size: $110.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing May 04, 2021
Details:
Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.
Lead Product(s): AAV capsid-gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: UndisclosedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Vertex Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 27, 2020
Details:
The proceeds will be used to advance the company’s platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.
Lead Product(s): Gene therapies
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: UndisclosedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Atlas Venture
Deal Size: $60.0 million Upfront Cash: Undisclosed
Deal Type: Series A Financing March 31, 2020
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