Astellas and Taysha Gene Therapies Announce Strategic

– Taysha Gene Therapies is an rising chief within the improvement of AAV gene therapies; new collaboration aimed toward enhancing improvement of two of Taysha’s novel product candidates for uncommon monogenic central nervous system illnesses with severe unmet medical wants –
– Astellas to speculate a complete of $50 million to amass 15% of the corporate and to obtain an unique choice to get hold of an unique license for TSHA-102 for Rett syndrome and TSHA-120 for big axonal neuropathy (GAN) –
– Astellas to obtain sure rights associated to any potential change of management of Taysha –
– Astellas to obtain one Board observer seat on the Taysha Board of Directors –

TOKYO and DALLAS, Oct. 24, 2022 (GLOBE NEWSWIRE) — Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) and Taysha Gene Therapies, Inc. (NASDAQ: TSHA, CEO: RA Session II, “Taysha”) immediately introduced a strategic funding to assist the development of Taysha’s adeno-associated virus (AAV) gene remedy improvement applications for the remedy of Rett syndrome and GAN. The future choices to probably apply Astellas’ international R&D, manufacturing and commercialization capabilities in gene remedy to Taysha’s modern AAV gene remedy improvement applications for genetic illnesses of the central nervous system (CNS) create the chance for the 2 corporations to boost the event of novel remedy choices for sufferers with Rett syndrome and GAN, who’ve severe unmet medical wants.

Under the phrases of the settlement, Astellas will make investments a complete of $50 million to amass 15% of the excellent frequent inventory of Taysha and to obtain an unique choice to license two of Taysha’s scientific stage applications: TSHA-102 for Rett syndrome and TSHA-120 for GAN. In addition, Taysha has granted Astellas sure rights associated to any potential change of management of Taysha. Definitive agreements can be executed upon Astellas’ train of any such possibility, and any change of management transaction would require approval by Taysha’s stockholders.

Taysha is engaged within the improvement of intrathecally-delivered AAV gene therapies for monogenic CNS illnesses. As part of this platform strategy, Taysha has a promising pipeline, together with TSHA-102, which is the first-and-only gene remedy in scientific improvement for Rett syndrome, and TSHA-120, which is in Phase 1/2 improvement for the remedy of GAN and awaiting regulatory suggestions.

Astellas is constant to construct its functionality to deliver novel gene therapies to sufferers, following the acquisition of Audentes (now Astellas Gene Therapies, California) in January 2020 and the development of a state-of-the-art industrial GMP manufacturing facility in North Carolina, which was opened in June of this 12 months.

“Gene therapy is the corner stone of Astellas’ Primary Focus, Genetic Regulation^*1; our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options,” stated Naoki Okamura, Chief Strategy Officer, at Astellas. “Taysha is an industry leader in CNS gene therapies and this partnership fits strategically with our long-term vision of expanding Astellas’ gene therapy capabilities, allowing the company to impact the lives of a broader range of patients with urgent unmet medical needs.”

“We are excited to enter this strategic investment with Astellas, a premier biopharmaceutical company with global R&D, manufacturing and commercial capabilities,” stated RA Session II, Taysha’s Chief Executive Officer. “We believe this investment not only further validates the potential of our technology platform, but also reinforces the therapeutic and market opportunity of our two lead clinical assets.”

To additional strategically align Astellas and Taysha, in reference to its fairness funding, Astellas will obtain one Board observer seat on Taysha’s Board of Directors, enabling Taysha to leverage Astellas’ gene remedy scientific and industrial experience as Taysha advances TSHA-120 and TSHA-102.

*1: Astellas has established a Focus Area Approach for its analysis and improvement technique. For extra info, please go to our web site at https://www.astellas.com/en/science/focus-area-approach.

About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 gene alternative remedy beneath improvement for the remedy of Rett syndrome. TSHA-102 makes use of the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to control transgene expression genotypically on a cell-by-cell foundation. The miRARE know-how is designed to forestall toxicity related to transgene overexpression and might be probably utilized throughout different indications. TSHA-102 has acquired Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission.

About Rett Syndrome
Rett syndrome is a extreme genetic neurodevelopmental dysfunction attributable to a mutation within the X-linked MECP2 gene important for neuronal and synaptic perform within the mind. Primarily occurring in females, Rett syndrome is likely one of the commonest genetic causes of extreme mental incapacity worldwide. Patients have regular early improvement, with symptom onset usually starting between 6 to 18 months of age. Rett syndrome is characterised by fast developmental regression that results in mental disabilities, lack of speech, lack of purposeful use of fingers, lack of mobility, seizures, cardiac impairments and respiration points. Currently, there are not any permitted therapies that deal with the underlying reason for this progressive illness.

About TSHA-120
TSHA-120, an intrathecally dosed AAV9 gene alternative remedy delivering the gene gigaxonin for the remedy of GAN, is at present being evaluated in an ongoing Phase 1/2 scientific trial. TSHA-120 has acquired Orphan Drug and Rare Pediatric Disease designations from FDA and Orphan Drug Designation from the European Commission.

About Giant Axonal Neuropathy (GAN)
GAN is uncommon inherited genetic dysfunction that may be a progressive neurodegenerative illness that impacts each the central and peripheral nervous techniques. The illness is attributable to loss-of-function mutations within the gene coding for gigaxonin, which leads to dysregulation of intermediate filament turnover, an essential structural element of the cell. Children with GAN current earlier than the age of 5 with signs together with unsteady gait, frequent falls, and motor weak point. Currently, there are not any permitted therapies for GAN, which leads to loss of life for sufferers of their late teenagers or early twenties.

About Taysha
Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS illness. With a singular give attention to growing healing medicines, we goal to quickly translate our therapies from bench to bedside. We have mixed our crew’s confirmed expertise in gene remedy drug improvement and commercialization with the world-class UT Southwestern Gene Therapy Program to construct an intensive, AAV gene remedy pipeline targeted on each uncommon and large-market indications. Together, we leverage our totally built-in platform—an engine for potential new cures—with a objective of dramatically enhancing sufferers’ lives. More info is offered at www.tayshagtx.com.

Forward-Looking Statements (Taysha)
This press launch comprises forward-looking statements inside the that means of the Private Securities Litigation Reform Act of 1995. Words similar to “anticipates,” “believes,” “expects,” “intends,” “projects,” and “future” or related expressions are meant to determine forward-looking statements. Forward-looking statements embrace statements regarding the potential of our product candidates, together with TSHA-120 in GAN and TSHA-102 in Rett syndrome, to positively affect high quality of life and alter the course of illness within the sufferers we search to deal with, the potential advantages of Taysha’s collaboration with Astellas, the potential for Astellas to train any of the choices granted to it by Taysha, our analysis, improvement and regulatory plans for our product candidates, the potential for these product candidates to obtain regulatory approval from the FDA or equal international regulatory companies, and whether or not, if permitted, these product candidates shall be efficiently distributed and marketed, and the potential market alternative for these product candidates. Forward-looking statements are based mostly on administration’s present expectations and are topic to numerous dangers and uncertainties that would trigger precise outcomes to vary materially and adversely from these expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements don’t represent ensures of future efficiency, and you’re cautioned to not place undue reliance on these forward-looking statements. Risks relating to our business are described intimately in our Securities and Exchange Commission (“SEC”) filings, together with in our Annual Report on Form 10-Ok for the full-year ended December 31, 2021, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, each of which can be found on the SEC’s web site at www.sec.gov. Additional info shall be made out there in different filings that we make sometimes with the SEC. Such dangers could also be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements communicate solely as of the date hereof, and we disclaim any obligation to replace these statements besides as could also be required by legislation.

About Astellas
Astellas Pharma Inc. is a pharmaceutical firm conducting business in additional than 70 nations world wide. We are selling the Focus Area Approach that’s designed to determine alternatives for the continual creation of latest medication to deal with illnesses with excessive unmet medical wants by specializing in Biology and Modality. Furthermore, we’re additionally wanting past our foundational Rx focus to create Rx+^® healthcare options that mix our experience and information with cutting-edge know-how in several fields of exterior companions. Through these efforts, Astellas stands on the forefront of healthcare change to show modern science into worth for sufferers. For extra info, please go to our web site at https://www.astellas.com/en.

About Astellas Gene Therapies
Astellas Gene Therapies is an Astellas Center of Excellence growing genetic medicines with the potential to ship transformative worth for sufferers. Our gene remedy drug discovery engine is constructed round modern science, a validated AAV platform, and trade main inner manufacturing functionality with a selected give attention to uncommon illnesses of the attention, CNS and neuromuscular system. Astellas Gene Therapies may also be advancing extra Astellas gene remedy applications towards scientific investigation. Astellas Gene Therapies is predicated in San Francisco, with manufacturing and laboratory amenities in South San Francisco, Calif., and Sanford, N.C.

Astellas Cautionary Notes
In this press launch, statements made with respect to present plans, estimates, methods and beliefs and different statements that aren’t historic details are forward-looking statements concerning the future efficiency of Astellas. These statements are based mostly on administration’s present assumptions and beliefs in gentle of the data at present out there to it and contain identified and unknown dangers and uncertainties. Various components may trigger precise outcomes to vary materially from these mentioned within the forward-looking statements. Such components embrace, however are usually not restricted to: (i) adjustments normally financial circumstances and in legal guidelines and laws, referring to pharmaceutical markets, (ii) foreign money trade fee fluctuations, (iii) delays in new product launches, (iv) the lack of Astellas to market present and new merchandise successfully, (v) the lack of Astellas to proceed to successfully analysis and develop merchandise accepted by clients in extremely aggressive markets, and (vi) infringements of Astellas’ mental property rights by third events.

Information about pharmaceutical merchandise (together with merchandise at present in improvement) which is included on this press launch is just not meant to represent an commercial or medical recommendation.

Contacts for inquiries or extra info:

Astellas Pharma Inc.

Portfolio Communications
Cassie Hogenkamp
+1-847-942-0980
[email protected]

Corporate Advocacy & Relations
+81-3-3244-3201

Taysha Gene Therapies, Inc.

Company Contact:
Kimberly Lee, D.O.
Chief Corporate Affairs Officer
Taysha Gene Therapies
[email protected]

Media Contact:
Carolyn Hawley
Canale Evoke
[email protected]