Our Pipeline

Pioneering Hope in Rare Diseases

Our innovative research consists of a growing portfolio of highly promising programs offering treatments and life-changing opportunities for patients worldwide.

BFB-101: Spastic Paraplegia 47 (SPG47)

SPG47 is an ultra-rare form of AP-4 Hereditary Spastic Paraplegia (AP-4 HSP) with an estimated prevalence in excess of 5,000 patients worldwide. It’s a progressive neurodevelopmental and neurodegenerative disease characterised by early childhood spasticity, microencephaly and seizures.

The science behind the disease

The AP4B1 gene helps to move things inside our cells, specifically from the trans-Golgi network to the endosomal compartment. When this gene does not work correctly, we call it a loss-of-function mutation, which causes SPG47.

A potential solution

BFB-101 is a patented AAV vector that expresses the AP4B1 gene, developed by Professor Mimoun Azzouz at the University of Sheffield with the support of Cure AP-4 and LifeArc.

It is administered via the brain as a single lifetime dose and has shown potential in restoring AP-4 function in vitro and improving motor function in AP4B1 mutant mice.

Next stages

As a late-stage pre-clinical project, we anticipate filing an IND in the U.S. for a phase I/II clinical trial in early 2025. In mid-2025, we will be ready to begin the clinical trial of BFB-101 and are in the process of securing further funding to embark on this exciting new phase of the project.

BFB-201: Dopamine Deficiency Disease

Monogenetic Disease Landscape

Dopaminergic enzyme deficiencies

  • Sepiapterin reductase
  • GTPCH-1 – CTP cyclohydrolase
  • PTPS 6-pyruvoyl tetrahydropterine synthase
  • DHPR – Dihydropterine reductase
  • AADC-Aromatic L-amino acid decarboxylase
  • TH-Tyrosine Hydroxylase

Parkinsonism syndromes

  • Lesch-Nyhan’s disease
  • Rett’s syndrome
  • Dopa responsive dystonias
  • Early onset Parkinson’s disease

Alacrita has undertaken an evaluation and has identified key targets within this landscape

Alacrita Report Key Targets

  • Sepiapterin reductase
  • PTPS 6-pyruvoyl tetrahydropterine synthase
  • DHPR – Dihydropterine reductase
  • TH-Tyrosine Hydroxylase

These conditions are clinically similar.
Could be treated as a single disorder group using a Master protocol.

Development Timeline

Development Timeline

“Very young children will benefit the most; this treatment could alter the trajectory of their lives. This is our legacy project.”

Chris Edwards

Founder and Chief Business Officer