Replay targets genetic brain disorders with new gene therapy company, Kaleibe

Replay targets genetic brain disorders with new gene therapy company, Kaleibe

Replay, a genome-writing company, today announced the launch of Kaleibe, an HSV gene therapy company targeting genetic brain disorders.

This is the third of Replay’s product companies to launch since the company’s inception in July and will leverage Replay’s high-carrying herpes simplex virus (HSV) delivery vector, synHSV, to target genetic disorders of the brain.

Kaleibe co-founders include Richard Wade-Martins, whose research at the University of Oxford focuses on the molecular mechanisms underlying neurodegenerative diseases, and Howard J Federoff, former Distinguished Professor of Neurology at the University of California, Irvine, and former CEO of a regenerative medicine company. , Aspen Neuroscience, Inc. Joe Glorioso, the inventor of synHSV, is also a co-founder of Kaleibe.

Initial development programs will focus on genetic Parkinson’s disease (PD) and Friedreich’s ataxia (FRDA). These diseases have a high unmet medical need and known genetic causes. The target genes, 33kb and 135kb, respectively, far exceed the payload capacity of adeno-associated virus (AAV) vectors (5kb). Affecting more than 6 million people worldwide, PD is the most common movement disorder and the second most common neurodegenerative disorder in the world, after Alzheimer’s disease.

Limited treatment options

It is estimated that around 15% of cases are hereditary, with a loss of functional genes that can be targeted by genomic drugs. FRDA is a genetic, progressive, neurodegenerative movement disorder affecting approximately 1 in 50,000 people, with a typical age of onset between 10 and 15 years. It leads to impaired muscle coordination (ataxia) which changes over time. Treatment options are limited and involve managing the symptoms and complications of the disease.

The launch of Kaleibe follows product company launches Eudora and Telaria focused on eye and skin conditions, respectively. Replay’s distinctive corporate structure separates technology development from product development within disease therapeutic area focused product companies.

Adrian Woolfson, Executive Chairman, President and Co-Founder of Replay, said, “HSV’s natural neurotropism allows the virus to establish latent infection in neurons and enables robust transgene expression in multiple regions of the brain. It was therefore rational for us to apply our proprietary delivery platform, synHSV, to target genetic disorders in the brain, where we believe HSV offers the promise of safer and more durable gene delivery than approaches based on the AAV. The high payload capacity of HSV further enables the expression of genomic genes, thereby deploying natural regulatory sequences and capturing altered splice forms.

New treatments

Lachlan MacKinnon, CEO and Co-Founder of Replay, said, “The launch of Replay’s third product company highlights the strength and broad applicability of our synHSV technology, as well as our ability to leverage its ability to payload by delivering cassettes up to 150 KB in length. With Kaleibe, we are fortunate to have leading experts in neuroscience and clinical neuroscience and neurology as co-founders, leveraging synHSV to bring new treatments to patients with debilitating brain disorders.

Wade-Martins said, “Replay’s mission to write and deliver big DNA resonates with my own research interest in this area. Genetic Parkinson’s disease and Friedreich’s ataxia are diseases characterized by mutations in particularly important genes, making them particularly difficult targets for AAV-based gene therapies. With the potential to deliver up to 30 times the payload of AAV, Replay’s synHSV platform facilitates the delivery of large genes – termed “big DNA” – with the promise of a differentiated and compelling therapeutic option for these disorders. incurable to date.

Significant impact

Federoff said, “Kaleibe aims to shed light on genetic brain disorders that have high unmet medical need and are associated with a significant negative impact on patients’ quality of life. Replay’s differentiated approach brings together the necessary elements to position the company for success. I look forward to working with the team to leverage Replay’s synHSV technology platform to develop novel treatments that address genetic mechanisms beyond the scope of other technologies that have the potential to have clinical impact. meaningful and lasting.

Glorioso added, “Our next-generation HSV delivery platform has several distinct advantages over existing gene delivery platforms and has the potential to disrupt them. The defined genetic causes of the neurological disorders studied at Kaleibe offer us the opportunity to introduce profound and long-awaited technological innovation into the treatment landscape.

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