Biointaxis S.L. 

A spin-off biotech company developing a state-of-the-art gene therapy for Friedreich’s ataxia


Antoni Matilla (See Profile)




Prototyping, preclinical



Unique product in reversing both neurological and cardiac deficits in FRDA

Unique in providing phisological expression of product for efficient treatment in FRDA

International patent (national phases)

Outstanding preclinical data

IP Type Status Time Countries Link Coverage
Patent Approved 25 years
  • Australia
  • Austria
  • Belgium
  • Canada
  • China
  • Denmark
  • Finland
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Russia
  • South Korea
  • Spain
  • Switzerland
  • USA
  • United Kingdom The present invention provides gene therapies for the treatment of Friedreich’s ataxia. Specifically, the present invention provides a nucleic acid, cloning vector and transfer vector for the production of an adeno-associated virus (AAV) vector. The nucleic acid comprises (i) a nucleic acid sequence encoding frataxin, (ii) a phospho-glycerate-kinase (PGK) promoter, and (iii) a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The present invention also provides a pharmaceutical composition which comprises the AAV vector or nucleic acid. Also, the AAV vector, nucleic acid or pharmaceutical composition can be used as a medicament, specifically as a medicament for the treatment of Friedreich’s ataxia.

This information is confidential, do not share or copy.

Under evaluation at least till: 8th April

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