Biointaxis S.L.
A spin-off biotech company developing a state-of-the-art gene therapy for Friedreich’s ataxia
| IP Type | Status | Time | Countries | Link | Coverage |
|---|---|---|---|---|---|
| Patent | Approved | 25 years |
|
https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2019076973 | 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. |
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Under evaluation at least till: 8th April
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