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New Company Creating High-Capacity Gene Therapies to the Brain

Herpes simplex virus illustration

Herpes simplex virus (Bernard Heymann, NIH)

14 Dec. 2022. A new business is underway developing gene therapies with higher capacities than most current technologies to treat neurodegenerative disorders. The company known as Kaleibe is formed by Replay Bio, a six-month old enterprise in San Diego that spins off businesses to advance genomic processes it calls big DNA.

Replay Bio seeks to provide gene therapies with more capacity to deliver larger payloads, as well as off-the-shelf cell therapies, with a common set of genomic and computational tools. The company acts as the parent organization for a group of new businesses, each developing treatments for specific or related targets. Since beginning operations in July 2022, Relay Bio started Eudora to develop treatments for genetic diseases affecting the retina, and Telaria for therapies addressing rare inherited skin disorders.

Kaleibe is Replay Bio’s newest spin-off enterprise, developing gene therapies for inherited forms of Parkinson’s disease and Friedreich’s ataxia. In Parkinson’s disease, certain nerve cells in the brain fail to produce the chemical  dopamine that controls muscular movement, and nerve endings stop producing norepinephrine that controls vital bodily functions. In some cases, Parkinson’s disease can be traced to inherited genetic conditions. Friedreich’s ataxia is a rare, inherited degenerative disorder caused by a defect in the FXN gene. While Parkinson’s disease affects mainly older individuals, Friedreich’s ataxia symptoms usually begin appearing in childhood.

Three Relay Bio companies using synHSV vectors

To deliver gene therapies for these conditions, Kaleibe plans to use an altered benign herpes simplex virus, one of Relay Bio’s genomic tools. The synthetic herpes virus, called synHSV by Relay Bio, is based on research by microbiologist Joseph Glorioso at University of Pittsburgh. Relay Bio says the modified herpes simplex virus has 30 times the capacity of adeno-associated viruses or AAVs, the current workhorses for delivering gene therapies. Glorioso is listed as a co-founder of Kaleibe, as well as Eudora and Telaria, Relay Bio’s first two spin-off companies that also deliver their gene therapies with synHSV vectors.

Adrian Woolfson, chairman and president of Relay Bio, says in a company statement, “HSV’s natural neurotropism allows the virus to establish a latent infection in neurons and enables robust transgene expression across multiple brain regions.” Woolfson adds, “The high payload capacity of HSV, furthermore, allows for the expression of genomic genes, thereby deploying natural regulatory sequences and capturing alterative splice forms.”

Kaleibe’s scientific founders include Richard Wade-Martins, professor of molecular neuroscience at University of Oxford in the U.K. and Howard Federoff, professor of neurology and neuroscience at Georgetown University in Washington, D.C. Wade-Martins’s lab at Oxford studies molecular mechanisms of age-related neurodegenerative diseases, and heads the university’s Parkinson’s disease research center. Federoff also studies neurodegenerative diseases, and is founder and CEO of other start-up biotechnology companies including Aspen Neuroscience in San Diego.

“Replay’s mission to write and deliver big DNA resonates with my own research interest in this area,” notes Wade-Martins. “Genetic Parkinson’s disease and Friedreich’s ataxia are diseases characterized by mutations in particularly sizable genes, making them especially difficult targets for AAV-based gene therapies.” Glorioso adds, “The defined genetic causes of the neurological disorders under investigation at Kaleibe provide us with the opportunity to introduce profound and long overdue technological innovation into the treatment landscape.”

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