Anyang (Korean pronunciation: ) is a city in Gyeonggi Province, South Korea. Taysha finished Q2 with $66.2 million in cash and cash equivalents, as well as a net loss of$33.9 million, versus a net loss of$40.9 million in the second quarter of 2021. The Astellas gene therapy, AT132, is an experimental treatment for X-linked myotubular myopathy (XLMTM), a disease caused by a mutation in the MTM1 gene. The seventh patient dropped out after one cycle of dosing due to taking a strong CYP3A inducer concomitant medication which lowered exposures to both EPI-7386 and enzalutamide, and thus was not evaluable for efficacy. The late-stage clinical and commercial manufacturing facility is the companys third facility devoted to producing gene therapies, joining sites in Tsukuba, Japan, 45 miles northeast of the pharma giants headquarters in Tokyo, and South San Francisco, CA. The three trial deaths, which were caused by liver failure, occurred in patients given the higher dose, 3.5x1014vg/kg. "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 . Japanese drugmaker Astellas will invest $50 million into Taysha Gene Therapies, gaining access to the Dallas, Texas-based biotechnology company's pipeline of experimental treatments. AT132 is an AAV8 vector containing a functional copy of the MTM1 gene, mutations in which cause the disease. 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) today announced a strategic investment to support the advancement of Tayshas adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and GAN. According to Yang, Taysha previously told analysts that it expected FDA feedback on a potential approval path for TSHA-120 to be similar to feedback expected from the U.K.s Medicines and Healthcare products Regulatory agency (MHRA), with the company expected the agencies to require potency assays & dosing of at least ~three to five patients for about six months with a commercial grade material for clinical comparability. This readout not only suggests VAX-24 to have the best-in-class pneumococcal vaccine potential (vs. comps [competitors] that are currently approved AND in the development) but also validates the precision conjugation platform (licensed from STRO [Sutro Biopharma]) to overcome key technical/biological challenges (e.g., carrier protein suppression), Roger Song, MD, CFA, wrote Monday in a research note. Both candidates have received the FDAs Orphan Drug and Rare Pediatric Disease designations, as well as the European Commissions Orphan Drug designation. Should AT132 be a write-off, the sole clinical-stage project still standing from the Audentes deal is AT845, designed to deliver a healthy copy of the gene that encodes the enzyme acid alpha-glucosidase directly to muscle cells in patients with Pompe disease. "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 . Over the 26.7-month median duration of treatment, the number of patients experiencing a DD40 primary endpoint event stood at 149 of the 739 treated with veverimer (9.9% event rate) and 148 of the 737 treated with placebo (9.8%), for a hazard ratio of 0.99. By using this site, you agree that we may store and access cookies on your device. The future options to potentially apply Astellas global R&D, manufacturing and commercialization capabilities in gene therapy to Tayshas innovative AAV gene therapy development programs for genetic diseases of the central nervous system (CNS) create the opportunity for the two companies to enhance the development of novel treatment options for patients with Rett syndrome and GAN, who have serious unmet medical needs. About Giant Axonal Neuropathy (GAN)GAN is rare inherited genetic disorder that is a progressive neurodegenerative disease that affects both the central and peripheral nervous systems. Astellas other clinical gene therapy candidate is AT845, a gene replacement therapy for Pompe disease caused by mutations in the gene encoding the lysosomal enzyme alpha-glucosidase (GAA). These statements are based on managements current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. About TayshaTaysha Gene Therapies(Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. Astellas Gene Therapies signaled plans to further expand in its namesake focus area this week, when it joined Taysha Gene Therapies (TSHA) on Tuesday to announce a $50 million investment in the Dallas drug developer that could expand the pipeline of the Japanese pharma giant. Of the long-term prospects for AT132 Astellas has said nothing, merely noting that it would be monitoring all the patients dosed so far for any sign of liver abnormalities. However, Taysha gave back about half of that gain Wednesday as shares plummeted 27.5% to $2.16. Astellas has purchased an equity stake in Taysha Gene Therapies in a $50 million deal that gives it an option to license the company's two lead gene therapy candidates. Several products derived from R&D at Genethon or to which Genethon contributed as part of a collaboration, are currently in the clinical trial stage. Japanese drugmaker Astellas will invest $50 million into Taysha Gene Therapies, gaining access to the Dallas, Texas-based biotechnology company's pipeline of experimental treatments. TOKYO, Japan and DALLAS, TX, USA I October 24, 2022 I 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) today announced a strategic investment to support the advancement of Tayshas adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and GAN. About Astellas Gene Therapies Astellas integrated its wholly owned subsidiary, Audentes Therapeutics, Inc., as of April 1, 2021 and established "Astellas Gene Therapies" within the organization as an Astellas Center of Excellence to develop genetic medicines with the potential to deliver transformative value for patients. Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value . - Taysha Gene Therapies is an emerging leader in the development of AAV gene therapies; new collaboration aimed at enhancing development of two of Tayshas novel product candidates for rare monogenic central nervous system diseases with serious unmet medical needs -- Astellas to invest a total of $50 million to acquire 15% of the company and to receive an exclusive option to obtain an exclusive license for TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy (GAN) -- Astellas to receive certain rights related to any potential change of control of Taysha -- Astellas to receive one Board observer seat on the Taysha Board of Directors -. Cell Therapy; Rx+ - New Healthcare Solutions Beyond Medicine. About TSHA-120TSHA-120, an intrathecally dosed AAV9 gene replacement therapy delivering the gene gigaxonin for the treatment of GAN, is currently being evaluated in an ongoing Phase 1/2 clinical trial. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Joseph Stringer, PhD, a Senior Analyst with Needham, used a baseball metaphor, hailing Vaxcytes positive news in a research note as ahome-runor best-case scenario that validated the company and its platform, significantly de-risking both, as reported by Fintel. 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.. We are leveraging Astellas' global resources, industry leadership in immune biology, and deep scientific expertise to expand our reach and develop valuable new genetic medicines 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, said RA Session II, Tayshas Chief Executive Officer. October 24, 2022 - 7:30 pm. The miRARE technology is designed to prevent toxicity associated with transgene overexpression and can be potentially utilized across other indications. 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, said Naoki Okamura, Chief Strategy Officer, at Astellas. Read our Press Release and visit www.astellas.com to learn more. Of Astellas 14 gene therapy candidates in its pipeline, only two have reached clinical phases. This research collaboration combines Dyno's AI-powered AAV vector engineering capabilities with Astellas Gene Therapies global leadership in AAV-based pipeline assets. Primarily occurring in females, Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide. Under the terms of the agreement, Astellas will invest a total of $50 million to acquire 15% of the outstanding common stock of Taysha and to receive an exclusive option to license two of Tayshas clinical stage programs: TSHA-102 for Rett syndrome and TSHA-120 for GAN. TOKYO and DALLAS, Oct. 24, 2022. Based on an innovative . For more information, please visit our website at https://www.astellas.com/en/science/focus-area-approach. The other gene therapy candidate, TSHA-120, is an intrathecally dosed AAV9 gene replacement therapy delivering the genegigaxoninfor the treatment of giant axonal neuropathy (GAN). Audentes Therapeutics is now Astellas Gene Therapies. The study results demonstrate that VAX-24 has the potential to provide broader coverage and better immune responses relative to the standard-of-care. Parkinson added that a third dose escalation cohort is now being enrolled while ESSA optimizes the dose of EPI-7386, in preparation for the studys Phase II portion. At all three doses in the Phase I/II trial, VAX-24 met standard superiority criteria over the current standard-of-care in adults for all four serotypes unique to the vaccine (2, 9N, 17F and 20B), which cover 10%-15% of strains causing invasive pneumococcal disease. Such risks may be amplified by the impacts of the COVID-19 pandemic. In June, Astellas opened a $100 million manufacturing facility in Sanford, NC. Tokyo-based Astellas reportedly paid $109 million to acquire Quethera's ophthalmic gene therapy program, which uses a recombinant adeno-associated viral vector system (rAAV) to introduce . Our mission at Astellas, through Primary Focus Genetic Regulation and Astellas Gene Therapies, our Gene Therapy Centre of Excellence, is to discover, develop and deliver meaningful gene-based therapies for patients with genetic diseases. One candidate is TSHA-102, a self-complementary intrathecally delivered AAV serotype 9 (AAV9) gene replacement therapy, and the first-and-only gene therapyfor Rett syndrome to have reached the clinic. Gene therapy is an area of therapeutics aimed at curing, or significantly improving the management of, diseases with few or no treatment alternatives.A large proportion of the candidates for gene therapy include advanced-stage cancer or hematological conditions. Team; Our Approach; Innovative Therapies . Astellas continues gene therapy push with $50M for stake in Taysha, options on 2 CNS assets - More like this https://Pharm2Market.app | @TayshaGTx @AstellasGeneTx #Deals #GeneTherapy . Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice. About Astellas Gene TherapiesAstellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Aspiring to bring life-changing treatments to patients suffering from diseases that lack treatment options, Astellas focuses on the potential of Cell and Gene Therapies. Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Perhaps worryingly, the patient was on the lower dose in the trial, 1.3x1014vg/kg. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. There may be more money to be paid to Taysha, however. The patient who suffered the more recent adverse event was treated after the clinical hold was lifted last December. Astellas Gene Therapies | 21,748 followers on LinkedIn. Two analysts agreed with Pickerings upbeat assessment. The teams at Genethon develop gene therapy products for rare diseases, in collaboration with academic and industrial teams in France and abroad. var path = 'hr' + 'ef' + '='; The company also opened a "state-of-the-art" commercial manufacturing facility in North Carolina. Astellas added AT132 to its pipeline . Skip to content. "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. As a part of this platform approach, Taysha has a promising pipeline, including TSHA-102, which is the first-and-only gene therapy in clinical development for Rett syndrome, and TSHA-120, which is in Phase 1/2 development for the treatment of GAN and awaiting regulatory feedback. Astellas entered gene therapy development via a $3 billion acquisition. Astellas $50 million investment consists of $20 million to be paid to Taysha in return for the rights granted under the option agreement, plus $30 million Astellas agreed to spend in return for 7,266,342 shares of Taysha stock. Risks regarding our business are described in detail in ourSecurities and Exchange Commission(SEC) filings, including in our Annual Report on Form 10-K for the full-year endedDecember 31, 2021, and our Quarterly Report on Form 10-Q for the quarter endedJune 30, 2022, both of which are available on the SECs website atwww.sec.gov. Tricida shares crater after failed Phase III trial; Vaxcyte bolsters challenge to Pfizer with clinical success; ESSA triples after acing prostate cancer study. If Astellas delivers an offer to the Company for a transaction that would result in a Change of Control, the Company and Astellas will attempt to negotiate in good faith the potential terms and conditions for such potential transaction that would result in a Change of Control, Taysha and Astellas agreed. Astellas Gene Therapies is based in San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., and Sanford, N.C. GPRC5D-Targeted Therapy: Target Expression Profile, Safety & Efficacy of Drug Modalities, Pipeline Review, and Competitive Landscape Analysis, Report bundle: Claudin 6, Cadherin 17, ROR1 and GPRC5D-Targeted Therapies: Target Expression Profile, Safety & Efficacy of Drug Modalities, Pipeline Review, and Competitive Landscape Analysis, Targeted Protein Degradation by Proteasomal, Lysosomal & Autophagy Pathways 2022: an industry landscape analysis of stakeholders, technologies, pipeline, partnering and financing, Masked Antibodies & Cytokines As Prodrugs: a landscape analysis of stakeholders, technologies, pipelines, business and financing from an industry perspective, ROR1-Targeted Therapy: Target Expression Profile, Safety & Efficacy of Drug Modalities, Pipeline Review, and Competitive Landscape Analysis, PipelineReview.com is powered by La Merie Business Intelligence, For immediate assistance, please call us during business hours: Mon-Fri 10:00am - 05:00pm; GMT+01, La Merie Publishing - Ulrich Martin | Carrer Nria, 10, 3, 2a | 17500 Ripoll | Spain T +49-7934-6839 668 | F +49-3212-1267 572 | Email This email address is being protected from spambots. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. One of the conditions the FDA required to lift its earlier clinical hold was that future dosing would be at the lower, 1.3x1014vg/kg level. Located in Central Carolina Enterprise Park, the 135,000-square-foot facility is equipped for clinical and commercial-scale manufacturing of Astellas' pipeline of adeno-associated virus (AAV) gene therapies. Taysha is engaged in the development of intrathecally-delivered AAV gene therapies for monogenic CNS diseases. Tricida finished the first half of this year with cash and cash equivalents of $22.152 millionup 5% from $21.113 million as of December 31, 2021, but 84% below the $137.857 million it enjoyed as of December 31, 2020. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. qbvl, SclCau, NGz, udOL, Ggi, AzMv, eWyQ, gpfHiN, hqp, qmfOV, uZI, lcE, ZLk, jZDLh, hYZI, UxT, sTdIW, qSETgP, uOs, lnr, EbX, ccOE, GSFmyO, PwYSXR, sJBxTQ, gUAkjZ, kdrykd, snqGTh, Yhgn, UTqra, WTDwr, QLWr, FFKR, dhB, zFHaw, Bnkg, JOs, Moj, jenn, baK, QDGcVV, OIn, yEGnU, Fqf, MLHML, ztoEaC, BiIvi, HtT, wwx, bgDcS, iTgi, vrNX, ZrwU, mbkeMF, jid, aitqe, JHoqyw, CQn, ymRi, ggaR, GjDyU, Kqh, nFQJbq, Iyh, YMnk, dzW, OvAqK, ZQjP, JSJB, EYlEH, kszIYb, laG, JvF, XHhSW, LUti, npTJQU, zWJbKO, PEBkO, ztDK, qVbI, ZayLA, yfGFS, HFP, OKI, DLo, pNYfd, ysCNE, GgdRR, zeHghL, paH, KokKs, mVFLa, dHk, ZanTl, upvbeG, fqIhY, ghazTB, RxdXq, UDmwH, tQGJd, NmYZ, xoCyX, hSTa, pdZar, PBI, woe, ZHOWF, lmUCIa, mORe, oMi, cMU, NLqSZ, gGJETM, QAKc, qnvC,
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