astellas gene therapy pipeline

Designated by Astellas as one of its "strategic" pipeline candidates. Astellas Reports Update to September 1 Announcement on the ASPIRO Tricida shares crater after failed Phase III trial; Vaxcyte bolsters challenge to Pfizer with clinical success; ESSA triples after acing prostate cancer study. 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 in a deal announced Monday. . Anyang (Korean pronunciation: ) is a city in Gyeonggi Province, South Korea. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. TSHA-102 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission. Vaxcyte said VAX-24 met the studys primary safety and tolerability objectives by demonstrating a safety profile similar to Pfizers Prevnar 20TM(PCV20) for all doses studied. Audentes Therapeutics Expands Mission, Becomes Astellas Gene Therapies Excitement around viral-vector gene therapies is evident. Astellas and Taysha Gene Therapies Announce Strategic Investment to Astellas tries a different tack in gene therapy, The Kras Shp has not yet sailed for Bristol, Jazz overture rekindles hopes for orexin agonism, Biogen takes another shot at spinal muscular atrophy, Novo follows three Swiss forays into a novel inflammatory target, Lecanemab can; now the wait for details begins, SITC 2022 preview cytokines and oral checkpoints, TCT 2022 Otsukas renal denervation system shines, Preclinical; expected to enter ph1 by end 2022, Astellas Tries Different Tack Gene Therapy. In addition, Taysha has granted Astellas certain rights related to any potential change of control of Taysha. Such 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 new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas intellectual property rights by third parties. Prevnar 20, marketed in Europe as Apexxnar, won FDA approval in June 2021 and European approval in February. 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. 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. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. Astellas and Taysha Gene Therapies Announce Strategic Corporate Strategic Plan 2021 . Astellas takes a stake in Taysha and gets a chance at next big gene Currently, there are no approved treatments for GAN, which results in death for patients in their late teens or early twenties. We use cookies on this website. On Monday the company was worth $60m; today its market capis around $100m. Overview of R&D Pipeline | Astellas Pharma Inc. Only a few other trials have dosed gene therapies at higher levels than Aspiro, and one of them, the Ignite DMD study of Solid Bios SGT-001, has been halted more than once. And in December 2021, Astellas announced plans to develop next-generation AAV vectors for gene therapies targeting skeletal and cardiac muscle using Dyno Therapeutics CapsidMap platform, through a collaboration with Dyno that could generate more than $1.6 billion for the George Church, PhD-co-founded company. Only two Audentes-originated therapies appear in Astellass current pipeline, quite the step down from the 10 it obtained via the takeout (Astellas spends $3bn on a bold move into gene therapy,December 3, 2019). Astellas Pharma (4503)'s investment gives it a 15% stake in Taysha, as well as an exclusive option to obtain an exclusive license for two of Taysha's By using this site, you agree that we may store and access cookies on your device. 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 tries a different tack in gene therapy | Evaluate We are encouraged by the rapid and deep PSA responses observed in the dose escalation study of EPI-7386 in combination with enzalutamide, stated David Parkinson, MD, ESSAs President and CEO. Astellas Gene Therapies is based in San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., and Sanford, N.C. Astellas Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. Tricida proceeded with VALOR-CKD after the FDA responded to the companys New Drug Application (NDA) seeking accelerated approval for veverimer in August 2020 with a Complete Response Letter. Astellas entered gene therapy development via a $3 billion acquisition. //-->. 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. It also will help address the Japan-headquartered company's supply chain needs and provide in-house quality control and testing. 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. 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:. "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. Tricida shares continued their slide over the following two trading days, falling another 8% to 55 cents on Tuesday, then plunging another 15% to $0.467 on Wednesday. Our investigational therapies target rare diseases through three modalities: gene replacement, exon skipping gene therapy and vectorized RNA knockdown. The $50m up front also gets Astellas an exclusive option to license two Tayshaprojects: TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy, though exactly how much Astellas might pay for these is subject to negotiation. Such risks may be amplified by the impacts of the COVID-19 pandemic. Solids gene therapy, like AT-132, uses an adeno-associated virus vector, though they are slightly different, Solids being AAV9-based where Astellass project uses AAV8. The information about the major pipeline at Astellas is updated on a quarterly basis. 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. Both candidates have received the FDAs Orphan Drug and Rare Pediatric Disease designations, as well as the European Commissions Orphan Drug designation. Looking for winners in geographic atrophy | Evaluate All Rights Reserved. Astellas is taking a minority stake in Taysha Gene Therapies, an investment that also gives the Japanese pharmaceutical giant an exclusive option to license two gene therapies Other interesting approaches include gene therapy, with Novartis active here through last year's purchase of . 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. 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. 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 . miRARE technology is intended to prevent toxicity associated with transgene overexpression and can be potentially utilized across other indications. Astellas and Taysha Gene Therapies Announce Strategic Investment to