Takeda/Evozyne Partnership Headlines Hot Week for Gene Therapy Deals


Chicago-based Evozyne inked a partnership with Takeda Pharmaceutical to develop next-generation gene therapies for up to four rare disease targets. The companies had a previous, separate agreement signed in January 2021.

Under the terms of the new collaboration and license agreement, Evozyne will develop novel protein sequences for gene therapies. Takeda has the option for an exclusive license to develop and commercialize the sequences as part of its gene therapy program. Takeda is paying Evozyne “double-digit million dollars in upfront and research funding payments.” Evozyne is also eligible for milestones up to $400 million, as well as tiered royalties on net sales of any products coming out of the collaboration.

“Our continued work with Takeda affirms Evozyne’s unique capability to provide advanced, targeted novel proteins that have remarkable potential in numerous applications,” said Jeff Aronin, Evozyne’s co-founder and chief executive officer. “Through these types of innovative collaborations, Evozyne plays a critical role in accelerating the development of gene therapies to potentially help people living with serious medical conditions.”

It's not unusual for companies to partner on gene therapies. Another announcement Wednesday was a gene therapy deal between Germany’s ViGeneron GmbH and Regeneron Pharmaceuticals. They will develop and commercialize a gene therapy product using ViGeneron’s novel engineered recombinant adeno-associated virus vectors (vgAAVs) to treat an inherited retinal disease. Regeneron is paying ViGeneron an undisclosed upfront payment and research funding, while Regeneron picks up an option for an exclusive license for the product. ViGeneron is up for various milestones and royalties.

On Tuesday, France’s Cytoo and Astellas presented results from their gene therapy collaboration at the 2nd Gene Therapy for Muscular Disorders Summit in Boston. The two companies partnered on an AAV gene therapy to treat Duchenne muscular dystrophy (DMD). The collaboration between the companies evaluated the activity of several AAV-mediated exon skipping candidates using Cytoo’s quantitative cell profiling assay, which uses artificial intelligence to create profiles that phenotypically differentiate between healthy and patient cells.

Although not a collaboration, 4D Molecular Therapeutics announced Monday that it had dosed the first patient in its Phase I/II trial of 4D-710, an AAV vector-based, aerosol-delivered gene therapy for cystic fibrosis.

Dr. Robert Fishman, M.D., 4DMT’s chief medical officer and pulmonology therapeutic area head, said, “To date, our platform has produced five clinical-stage product candidates that incorporate three different proprietary and novel capsids. We are seeking to unlock the full potential of genetic medicines through our platform and to fulfill the promise of transformative biotherapeutics to benefit patients.”

Takeda’s deal with Evozyne is part of its strategic plan to grow its gene therapy pipeline. The original deal between the companies focused on developing proteins to be used in gene therapies for inherited metabolic diseases. Evozyne delivered its first protein to Takeda within six months and then went on to design a library of novel proteins with better functionality. It is also developing functional “miniaturized genes” that might get around obstacles of engineered viruses presently used in gene therapy.

For example, the deal with Cytoo and Astellas focuses on DMD gene therapy using exon-skipping technology, which is similar to what is used by Sarepta’s Exondys 51 and other DMD drugs. The dystrophin gene that is damaged in DMD patients is the largest gene in the human body, and as such, doesn’t fit into the viral vectors used in most gene therapies. So researchers are developing several different approaches to work around this, including gene skipping, where only part of a gene is translated and creates a truncated form of the necessary protein, or Evozyne’s approach to miniaturization.

Evozyne’s technology platform leverages deep-learning-based computational models and high-throughput gene synthesis and assays to build novel, adaptive proteins. It has a six-step process moving from statistical analysis to microfluidic assays, and an analytical model that can take a large amount of protein data and select useful information about the target protein. Their algorithm then creates a model that associates the protein’s sequence to its function. This allows the company to design a protein with a significantly greater set of information than those performed with traditional directed evolution.

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