2022-07-05 | Press release | Mogrify and Astellas Announce Collaboration to Research In Vivo Regenerative Medicine Approaches to Treat Sensorineural Hearing Loss

CAMBRIDGE, United Kingdom and TOKYO, July 5, 2022 /PRNewswire/ — Mogrify Limited (CEO: Darrin M. DisleyPh.D., “Mogrify®“), a biopharmaceutical company that is transforming the lives of patients with a new class of live Reprogramming Therapies, and Astellas Pharma Inc. (TSE: 4503), (President and Chief Executive Officer: Kenji YasukawaPh.D., “Astellas”), a leader in regenerative medicine, today announced that it has entered into a collaborative research agreement on live regenerative medicine approaches to treat sensorineural hearing loss.

Using Mogrify’s proprietary direct cell reprogramming platform, the collaboration will seek to identify novel combinations of transcription factors involved in cell differentiation to generate novel cochlear hair cells. As part of this collaboration, Astellas Gene Therapies, a division of Astellas, covers the research costs of the work and brings its expertise in genetic medicine based on adeno-associated viruses (AAV) and its translational capacities to carry out preclinical experiments. models. Mogrify will leverage its bioinformatics platform, screening and validation process to characterize potential therapeutic factors.

It is estimated that 1.57 billion people worldwide suffer from hearing loss1and US data suggests that more than 10% have severe to profound sensorineural hearing loss in at least one ear2. This degree of hearing loss significantly reduces quality of life and, in the absence of currently available drug treatments, represents a significant unmet need.

Dr. Louise Modis, CSO, Mogrify, said:Mogrify’s human regulatory network-centric approach is well positioned to identify superior factor combinations, increasing the efficiency of direct conversion to the target cell type in the ear. Combined with Astellas’ abilities to gene therapy and neurosensory researchthis givesa clear path for the development of a novel live reprogramming therapy forSensorineural hearing loss.”

dr. Mathew PlecherSenior Vice President, Division Head of Gene Therapy Research and Technical Operations, Astellas, said: “In this collaboration, we will seek tocombine the unique delivery attributes of AAV-based gene therapy, with our extensive otology translation capabilities developed through our “Targeted Therapeutics for Auditory Regeneration” and “Direct Reprogramming (Transdifferentiation)” initiatives. Thanks to this collaboration,we will seek to address a significant unmet need in the field of sensorineural hearing loss.

1. Collaborators with hearing loss. Prevalence of hearing loss and years lived with disability, 1990-2019: results from the 2019 Global Burden of Disease Study. Lancet. 2021 Mar 13;397(10278):996-1009.

2.Goman AM, Lin FR. Prevalence of severe hearing loss in United States. Am J Public Health. (2016);106(10):1820-1822.

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Mogrify has developed a proprietary suite of platform technologies that use a systematic approach to big data to direct cellular reprogramming (Rackham et al.Nature Genetics, 2016) and maintenance of cellular identity (Kamaraj et al., Cellular Systems, 2020).

Platforms, MOGRIFY® and epiMOGRIFY®developed over a 12-year period via a multinational research collaboration, deploys next-generation sequencing, gene regulation and epigenetic network data to enable prediction of the transcription factors and growth factors needed to produce any target human cell type from any source human cell type.

The platforms can be used to improve existing methods of direct stem cell programming or bypass developmental pathways altogether, affecting direct transdifferentiation between one mature cell type and another mature cell type.

Mogrify applies its proprietary, award-winning platforms to generate the functional cell types needed to transform patients’ lives, delivering a new class of live reprogramming of therapies in the fields of ophthalmology, otology, metabolism and other degenerative diseases.

Uniquely positioned to address a regenerative medicine market estimated to be worth 150 billion US dollars by 2028, Mogrify is commercializing its technology through a combination of live rescheduling therapy development, co-development partnerships, as well as leveraging the platform in other therapeutic and non-therapeutic applications.

Situated at Cambridge, United Kingdomthe Company has raised more than 40 million US dollars funding from Parkwalk, Ahren Innovation Capital, 24Haymarket, Trend Investment Group, Dr. Darrin M. Disley,OBE, Dr. jonathan milner and the University of Bristol Enterprise Fund III, as well as strategic investors; Stellas Venture Management.

Follow Mogrify on Twitter @Mogrify_EN and LinkedIn @Mogrify

Astellas Pharma Inc. is a pharmaceutical company with operations in more than 70 countries around the world. We promote the focus area approach which is designed to identify opportunities for the continued creation of new medicines to treat diseases with high unmet medical need by focusing on biology and modality. Additionally, we are also looking beyond our core purpose of Rx to create Rx+® healthcare solutions that combine our expertise and knowledge with state-of-the-art technology in different areas from external partners. Through these efforts, Astellas is at the forefront of change in healthcare to turn innovative science into value for patients. For more information, please visit the Astellas website.

About Astellas Gene Therapies

Astellas incorporated its wholly-owned subsidiary, Audentes Therapeutics, Inc. effective April 1, 2021 and established “Astellas Gene Therapies” within the organization as an Astellas Center of Excellence for developing genetic medicines with the potential to deliver transformative value to patients. Based on an innovative scientific approach and industry-leading in-house manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy and vectored RNA knockdown and also hope to advance other Astellas gene therapy programs to clinical investigation. We are based at San Franciscowith manufacturing and laboratory facilities at South of San Francisco and Sanford, North Carolina.

Cautionary Notes (Astellas)

In this press release, statements made regarding current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. These factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in the launch of new products, (iv) Astellas’ inability to effectively market existing and new products, (v) Astellas’ inability to continue to effectively research and develop customer-accepted products in highly competitive markets, and (vi) violations of Astellas’ intellectual property rights by third parties. Information about pharmaceutical products (including products under development) that is included in this press release is not intended to constitute advertising or medical advice.

Dr. Mathew Pletcher, Senior Vice President, Gene Therapy Research and Technical Operations Division Head, Astellas

Mogrify and Astellas Announce Collaboration

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SOURCEAstellas Pharma Inc.

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