Newly formed gene therapy company has genetic Parkinson’s disease in its sights

By Jane Byrne

- Last updated on GMT

© GettyImages/Dr_Microbe
© GettyImages/Dr_Microbe

Related tags Gene therapy AAV

Replay, the genome writing company formed in July, has launched its third product company, Kaleibe, which will use HSV gene therapy to target genetic brain disorders.

The establishement of Kaleibe follows the recent inauguration of Replay’s two other companies, Eudora and Telaria, which are focused, respectively, on diseases of the eye and skin.

Kaleibe will look to leverage Replay’s high payload capacity herpes simplex virus (HSV) delivery vector, synHSV, which was developed at the University of Pittsburgh by Kaleibe co-founder, Professor Joe Glorioso, to target genetic brain disorders such as genetic Parkinson’s disease (PD) and Friedreich’s ataxia (FRDA).

Experts in neuroscience and neurology, Professors Richard Wade-Martins and Howard J Federoff, are also co-founders of Kaleibe.

Initial disease targets

Genetic PD and FRDA have a high unmet medical need and known genetic causes, said the developers. The target genes, 33kb and 135kb, respectively, far exceed the payload capacity of adeno-associated virus (AAV) vectors (5kb), they added.

Wade-Martins said that because genetic PD and FRDA are diseases characterized by mutations in particularly sizeable genes, this makes them especially difficult targets for AAV-based gene therapies. “With the potential to deliver up to 30x the payload of AAV, Replay’s synHSV platform facilitates the delivery of large genes – so called ‘big DNA’ – with the promise of a differentiated and compelling therapeutic option for these, to date, intractable disorders.”

PD is the second most common neurodegenerative disorder globally, after Alzheimer’s disease. It is estimated that approximately 15%​ of cases are inherited, with a loss of functional genes that can be targeted by genomic medicines.

FRDA is a genetic, progressive, neurodegenerative movement disorder affecting around 1 in every 50,000 individuals, with a typical age of onset between 10 and 15 years. It leads to impaired muscle coordination (ataxia) that progresses over time. Treatment options for FRDA are limited and involve managing the symptoms and complications of the disorder.

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