The startup develops next-generation gene therapies utilizing Adeno-Associated Virus (AAV) vectors to deliver healthy genes to patients with rare diseases affecting the Central Nervous System. This approach aims to provide significant therapeutic benefits by addressing the underlying genetic causes of these conditions.
Funding
$3.5M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare genetic diseases affecting the central nervous system lack effective treatments, leading to progressive neurological deficits and limited life expectancy for affected individuals. Current management strategies often focus on symptomatic relief rather than addressing the underlying genetic cause.
Solution
Blackfin Bio is developing gene therapies that utilize adeno-associated virus (AAV) vectors to deliver functional genes to patients with rare central nervous system (CNS) disorders. Their approach aims to correct the genetic defects responsible for diseases like Hereditary Spastic Paraplegia (SPG47) and dopamine deficiencies. The company's lead candidate, BFB-101, is an AAV vector expressing the AP4B1 gene for the treatment of SPG47, a rare form of AP-4 Hereditary Spastic Paraplegia. BFB-201, another pipeline candidate, is designed to deliver a gene fusion cassette for optimal dopamine synthesis, targeting rare neurological diseases caused by dopamine deficiency. These gene therapies are designed as a one-time treatment administered directly to the brain, offering the potential to halt or reverse disease progression.
Target Audience
The primary target audience includes patients with rare genetic diseases affecting the central nervous system, particularly those with Hereditary Spastic Paraplegia (SPG47) and dopamine deficiencies, as well as their families and caregivers.
Features
- Utilizes AAV vectors for efficient gene delivery to the central nervous system.
- BFB-101: AAV vector expressing the AP4B1 gene for SPG47, with demonstrated potential in restoring AP-4 function in vitro and improving motor function in AP4B1 mutant mice.
- BFB-201: AAV-based gene therapy delivering a single gene fusion cassette for dopamine synthesis, targeting sepiapterin reductase deficiency (SRD), 6-pyruvoyl tetrahydropterine synthase deficiency (PTPSD), dihydropterine reductase (DHPR) and tyrosine hydroxylase (TH) deficiency.
- Designed for a single, lifetime dose administration to the brain.
- Holds Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for BFB-101 in the treatment of SPG47.