A Step-Change in Treating Ciliopathies

Axovia’s mission is to develop disease-transformative therapies that target key aspects of ciliopathies, addressing the devastating impact of the disease head-on for patients, their families and caregivers.

The Axovia Charter

The scientific team at Axovia is committed to:

  • Designing treatments that will reduce the ciliopathic disease burden and increase patients’ quality of life
  • Developing disease-transformative adeno-associated virus-based gene therapies that treat key aspects of the devastating disease
  • Intervening early to prevent disease development and progression

Accelerating Disease-Transformative Treatments

Initially discovered by Laurence and Moon in 1886, it took another century before Professor Beales analyzed the underlying characteristics of BBS and published a definitive set of diagnostic criteria in 1999. The discovery of the first BBS gene followed in 2000 and now stands at 22 genes – some 20 years later.

Bardet-Biedl Syndrome Discovery Timeline

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Discovery Timeline

1886

The disease was first discovered by Laurence and Moon.

1999

Professor Beales established the first diagnostic criteria for BBS.

2000

The first BBS gene was discovered with Professor Beales as part of the team.

2003

Primary cilia dysfunction identified as the causation of BBS.

2010

The first national specialist clinic for BBS opens in the UK, funded by the National Health Service.

2017

At UCL, Prof. Beales, Dr. Victor Hernandez and team received funding from Apollo Therapeutics, enabling them to develop preclinical evidence for a viable gene therapy for BBS.

2020

Axovia Therapeutics was formed to work towards developing the first in-human therapies for BBS, starting with BBS1.

It’s Time for a Novel Approach

Our goal is to build on the discoveries of the disease and accelerate the development of transformative treatments for patients – as early as possible. The initial goal is to halt the progression of BBS by targeting the most debilitating aspects of the disease.

While modern diagnostic tools have made definitive, early identification of the disease easier, there remains a real need for a treatment to address the BBS disease trajectory through the design of novel gene therapies.