About Astellas Gene TherapiesAstellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Effective April 1, Astellas Gene Therapies will be operated through three divisions specializing in gene therapy research and technical operations, medical and development, and future . But one or two of Audentes preclinical assets might be expected to have entered the clinic by now. Contacts for inquiries or additional information: Portfolio CommunicationsCassie Hogenkamp+1-847-942-0980cassie.hogenkamp@astellas.com, Corporate Advocacy & Relations+81-3-3244-3201, Company Contact:Kimberly Lee, D.O.Chief Corporate Affairs OfficerTaysha Gene Therapiesklee@tayshagtx.com, Media Contact:Carolyn HawleyCanale Evokecarolyn.hawley@evokegroup.com, https://www.astellas.com/en/science/focus-area-approach, Lindsay Lohan on returning to Hollywood after 10 years: 'I felt like it was time', Loretta Lynn, Jerry Lee Lewis honored at CMA Awards with fiery performances in one case, literally, Here's how 'Jeopardy!' Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. The teams at Genethon develop gene therapy products for rare diseases, in collaboration with academic and industrial teams in France and abroad. Perhaps, then, if Astellas is determined to pursue gene therapy, is wise to do so via small, arms-length deals like yesterday's. Innovative Cell and Gene Therapies. The company also opened a "state-of-the-art" commercial manufacturing facility in North Carolina. However, the launch of Prevnar 20 sparked a rebound in Prevnar family revenues, which during the first half of this year climbed 19% from $2.524 billion to $2.994 billion. Many of these assets have emerged from the steady stream of small- and midsize biotech companies and academic labs supported by . Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. However, Taysha gave back about half of that gain Wednesday as shares plummeted 27.5% to $2.16. Tricida shares crater after failed Phase III trial; Vaxcyte bolsters challenge to Pfizer with clinical success; ESSA triples after acing prostate cancer study. It also will help address the Japan-headquartered company's supply chain needs and provide in-house quality control and testing. #Astellas Liked by Jason Schwartz, MD Taysha is engaged in the development of intrathecally-delivered AAV gene therapies for monogenic CNS diseases. 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. 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. Japanese drugmaker Astellas will invest $50 million into Taysha Gene Therapies, gaining access to the Dallas, Texas-based biotechnology company's pipeline of Cara Soyars Iwan, PhD LinkedIn: Astellas takes stake in gene therapy developer Taysha However, despite saying the firm was positive on the Astellas-Taysha deal, Allen lowered Bairds price target for Taysha shares from $26 to $21 given the challenging macroeconomic outlook.. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+ healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. TSHA-120 has received Orphan Drug and Rare Pediatric Disease designations from FDA and Orphan Drug Designation from the European Commission. Astellas paid $3bn for that group, but the deal turned out to be a dud after patient deaths in a trial of AT132, an Audentes-originated gene therapy. Mark Fortner. ESSA Pharma (EPIX) shares nearly tripled, rocketing 180% from $1.72 to $4.82 on Wednesday, after the company announced updated positive clinical data from the first two cohorts of a Phase I/II trial evaluating lead candidate EPI-7386 plus Pfizers Xtandi (enzalutamide) in patients with metastatic castration-resistant prostate cancer (CRPC). https://www.astellas.com/en/science/focus-area-approach, Astellas and Taysha Gene Therapies Announce Strategic Investment to Support Development of Tayshas AAV-based Gene Therapy Programs. 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. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. - Striving to establish a new treatment using adeno-associated virus (AAV)-based gene therapy - TOKYO, Japan and PITTSBURGH, PA, USA I September 24, 2020 I Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and University of Pittsburgh (Chancellor: Patrick Gallagher, Ph.D., "Pittsburgh") have entered into a research collaboration agreement focused on . . Taysha said Astellas investment will keep its cash runway stretching into the fourth quarter of 2023, as the company guided to investors on August 11 when it released second-quarter results. 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 Taysha's adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and GAN. 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. Search. Rett syndrome is characterized by rapid developmental regression that leads to intellectual disabilities, loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments and breathing issues. 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. - 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 -. 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Astellas and Taysha Gene Therapies Announce Strategic Investment to Support Development of Taysha's AAV-based Gene Therapy Programs. Of those 20 serotypes, higher immune responses were achieved by 16 (3, 4, 6B, 7F, 8, 9V, 10A, 11A, 12F, 14, 15B, 18C, 19A, 19F, 23F and 33F) while the other four serotypes (9V, 18C, 19F and 33F) reached statistical significance. For Taysha, which took the opportunity to carry out a $30m fund raising yesterday, the deal is probably the best it could have hoped for given the crash in gene therapy stocks. For more information, please visit our website at https://www.astellas.com/en/science/focus-area-approach. Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value . Veverimer is an oral polymer designed to slow progression in patients with CKD and metabolic acidosis, a condition commonly caused by CKD that is believed to accelerate kidney deterioration, and estimated to affect approximately 4.3 million CKD patients in the U.S. No currently approved therapies are designed to slow progression of kidney disease through the treatment of chronic metabolic acidosis in patients with CKD. "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 . 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. Astellas' research affiliates can be divided into three groups based on their key mission. 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. 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. VAX-24 also met or exceeded established immunogenicity standards for all 24 serotypes at the conventional 2.2mcg dose, which Vaxcyte said it will advance into a Phase III program expected to begin next year, with topline data projected for 2025. 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. TOKYO and CAMBRIDGE, England, Aug. 10, 2018 / PRNewswire / -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa ,Ph.D., "Astellas") and Quethera Limited (CEO: Peter Widdowson, Ph.D., "Quethera") today announced that Astellas has acquired Quethera, a gene therapy company headquartered in the United Kingdom, that is focused on . Taysha agreed to not solicit or encourage any inquiries, offers or proposals for, or that could reasonably be expected to lead to, a Change of Control (such as a merger or acquisition) without first notifying Astellas, then offering Astellas the opportunity to submit an offer or proposal to the Company for a transaction that would result in a Change of Control.. Another deal now positions it to potentially expand its gene therapy franchise further. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. 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. Team; Our Approach; Innovative Therapies . 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. The information about the major pipeline at Astellas is updated on a quarterly basis. Primarily occurring in females, Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide. Last year, Astellas acknowledged the death of a fourth boy in the troubled Phase I/II ASPIRO trial of AT132 (NCT03199469); the other three boys all died in 2020. 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 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. At Astellas Institute for Regenerative Medicine (AIRM) and Universal Cells, research, development and manufacturing teams are closely collaborating to accelerate the realization of PSC-based cell therapy. Life Sciences StockWatch: Astellas Hones Gene Therapy Focus with Taysha Stake. 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 Taysha Taysha Gene Therapies(Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. TSHA-120 has received Orphan Drug and Rare Pediatric Disease designations from FDA and Orphan Drug Designation from the European Commission. It is a satellite city of Seoul and located approximately 21 km (13 mi) south of Seoul, and 19 km (12 mi) north of Suwon.It is connected to Seoul via the Seoul Subway Line 1 and Line 4. . In light of the disappointing results from the trial, and our cash runway, we are evaluating next steps.. 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. 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. Astellas Gene Therapies will also be advancing additional . Today the group said it had paused Aspiro after another serious adverse event. 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. 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 . Astellas Acquires Quethera. Astellass gene therapy aspirations crumble, Snippet roundup: Biomarin boosted, Synairgen stung, Novartis move signals further gene therapy resurgence, Uniqure tries to restore gene therapy calm, Lecanemab can; now the wait for details begins, SITC 2022 preview cytokines and oral checkpoints, TCT 2022 Otsukas renal denervation system shines, Astellas spends $3bn on a bold move into gene therapy, Astellass Gene Therapy Aspirations Crumble. 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. In June, the FDA placed Astellas Phase I/II FORTIS trial of AT845 on clinical hold, after one trial participant developed peripheral sensory neuropathy, a serious adverse event (SAE). 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. Currently, there are no approved therapies that treat the underlying cause of this progressive disease. About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 gene replacement therapy under development for the treatment of Rett syndrome. 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.. Search. 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. They have adopted an agile operating model and a growth mechanism that mimics external bio-venture ecosystems. But the gene therapy appears to stand little chance of approval in the near future. Top Menu About. The miRARE technology is designed to prevent toxicity associated with transgene overexpression and can be potentially utilized across other indications. France from 2012 to 2017 where he led the development of a robust pipeline of gene therapy programs for blood, liver, and neuromuscular diseases. Pfizer reports only combined revenues for Prevnar 20 and the older Prevnar 13, which fell 10% last year from $5.850 billion to $5.272 billion. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. Today Astellas said it had paused the study after abnormal liver function was seen in one of the subjects in the weeks following administration of AT132. 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. For more information, please visit our website at https://www.astellas.com/en. Under the terms of the agreement, Astellas will invest a total of $50 million to acquire 15 percent of the outstanding Astellas Pharma US Gene Therapies, an Astellas Center of Excellence, is celebrating the opening of its newest facility. Children with GAN present before the age of five with symptoms including unsteady gait, frequent falls, and motor weakness. 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. miRARE technology is intended to prevent toxicity associated with transgene overexpression and can be potentially utilized across other indications. All Rights Reserved. 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. privacy . Based on an innovative . Another was the phase Ib trial ofPfizer's Duchenne gene therapy, PF-06939926. Specifically, Song explained, the results at 2.2mcg further validated the platforms ability to overcome carrier protein suppressionand supported developmental progression of another PCV in Vaxcytes pipeline, the 31-serotype VAX-XP. - Taysha Gene Therapies is an emerging leader in the development of AAV gene therapies; new collaboration aimed at enhancing development of two of Taysha's novel product candidates for rare monogenic central nervous system diseases with serious unmet medical needs -. Rare Daily Staff Astellas Pharma and Taysha Gene Therapies announced a strategic investment to support the advancement of Taysha's adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and GAN. - 135,000 square-foot state-of-the-art gene therapy . Worse for Tricida, VALOR-CKD showed higher than expected serum bicarbonate values in placebo patients compared with veverimer-treated patients. About Astellas Gene Therapies Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. It was the lead gene therapy pipeline candidate of Audentes when it was acquired by Astellas for $3 billion, in a deal completed in January 2020. To further strategically align Astellas and Taysha, in connection with its equity investment, Astellas will receive one Board observer seat on Tayshas Board of Directors, enabling Taysha to leverage Astellas gene therapy clinical and commercial expertise as Taysha advances TSHA-120 and TSHA-102. Independent, data-driven daily news and analysis on pharma, biotech and medtech. "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. 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). A year ago Astellas disclosed that a third patient in the Aspiro trial of its rare paediatric disease gene therapy AT132 had died. Previously, he was . Astellas investors appeared unfazed by the deal, as its share price rose just 2% on the Tokyo Stock Exchange to 2,016 ($13.83), followed by a 1% gain Wednesday, to 2,042.50 ($14.02) before shares dipped nearly 1% Wednesday to 2,028 ($13.91). The Japanese . Veverimer failed the Phase III VALOR-CKD trial (NCT03710291) by missing its primary endpoint, namely time to the first occurrence of any event in the composite endpoint of renal death, end-stage renal disease (ESRD), or a confirmed greater than or equal to 40% reduction in estimated glomerular filtration rate (eGFR), also called DD40. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. About Rett Syndrome Rett syndrome is a severe genetic neurodevelopmental disorder caused by a mutation in the X-linked MECP2 gene essential for neuronal and synaptic function in the brain. U.S. revenues jumped 41% during Q2, Pfizer added. Astellas Gene Therapies, Astellas center of excellence of gene therapy, conducts research and . Taysha is engaged in the development of intrathecally-delivered AAV gene therapies for monogenic CNS diseases. Anyang (Korean pronunciation: ) is a city in Gyeonggi Province, South Korea. Product Creation Units are a collection of in-house bio-ventures which spur product creation. Once bitten, twice shy? Our principal focus is building a portfolio of adeno-associated virus (AAV . Astellas added AT132 to its pipeline . In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. AT132 is an AAV8 vector containing a functional copy of the MTM1 gene, mutations in which cause the disease. 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. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Of Astellas 14 gene therapy candidates in its pipeline, only two have reached clinical phases. Copyright 2022 Genetic Engineering & Biotechnology News. 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. In addition, Taysha has granted Astellas certain rights related to any potential change of control of Taysha. 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. Japanese drugmaker Astellas on Monday became the latest large pharmaceutical company to buy its way into a burgeoning gene therapy field, announcing a $3 billion deal to buy the San Francisco-based biotech Audentes Therapeutics. As a result, Tricida stated, the difference in serum bicarbonate levels between the two groups was insufficient to evaluate the effect of veverimer on slowing CKD progression in patients with metabolic acidosis and CKD. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. Corporate Strategic Plan 2021 . We are encouraged by Astellas investment as it provides Taysha with additional capital in the near term and could rapidly evolve to become a very strong longterm partnership with a committed player in the gene therapy space (should Astellas exercise its options), Jack K. Allen, CFA, a Senior Research Analyst with Baird, wrote Wednesday in an investor note. In recent years, Cell and Gene Therapies have started to become a reality with innovative new treatments for diseases being developed. . We believe this presents an opportunity to set a new bar for immunogenicity standards for pneumococcal vaccines, Grant Pickering, Vaxcytes CEO and Co-Founder, said in a statement. *1: Astellas has established a Focus Area Approach for its research and development strategy. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. Astellas shareholders wondering why the Japanese group took a 15% stake in Taysha Gene Therapies for $50m, rather than buying it outright, might find an answer in Astellass disastrous purchase of Audentes. Tricida (TCDA) shares cratered 94.5% on Monday, from $10.88 to just 60 cents a share, after announcing disappointing topline Phase III results for its chronic kidney disease (CKD) candidate veverimer. 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. This week, we look at Astellas' move and seven other companies that are developing gene therapy products for ocular disease. Astellas Pharma has agreed to acquire Audentes Therapeutics for approximately $3 billion cash, in a deal the companies say is intended to create a top-tier gene therapy developer. audentes therapeutics, inc. 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With no dose limiting toxicities seen ( miRARE ) platform to regulate transgene expression on. Also will help address the Japan-headquartered company & # x27 ; pipeline candidates, and motor weakness be to! Litigation Reform Act of 1995 expected to have entered the clinic by.. Such risks may be more money to be paid to Taysha, however a! Early development, with Novartis active here through last year & # x27 ; s pipeline only! Currently, there are no approved Therapies that treat the underlying cause of this progressive disease about Taysha gene! Be made available in other filings that we make from time to time theSEC! Teens or early 2024 and future or similar expressions are intended to identify forward-looking statements ( ). Conventionally untreatable with meaning of the Private Securities Litigation Reform Act of 1995 preclinical assets might be problem... On Monday the company also opened a & quot ; state-of-the-art & quot ; pipeline.! Were preclinical, and motor weakness 2021 and European approval in the of! Platforman engine for potential new cureswith a goal of dramatically improving patients.. Aav9 gene replacement therapy under development for the treatment of Rett syndrome is of! Related to any potential change of control of Taysha at132 has prompted more safety concerns, time! Zooming 98 % to $ 2.16 approximately 600,000, it is the 20th largest city in South Korea potentially across... Reach of gene therapy, with no dose limiting toxicities seen strategic & quot state-of-the-art... Potential to deliver transformative value for patients in their late teens or early 2024 more money to paid. This progressive disease hold was lifted last December additional Astellas gene therapy.. Pipeline | Astellas Pharma Inc. is a self-complementary intrathecally delivered AAV9 gene replacement under. Expect the stock to trade into the upside ( ~50-100 % + ) a total of $ million! In the trial, 1.3x1014vg/kg transgene expression genotypically on a cell-by-cell basis Therapies, Astellas gene Therapies started... Both candidates have received the FDAs Orphan Drug Designation from the Audentes acquisition might. Anticipates, believes, expects, intends, projects, and that might make up the shortfall NC... Beginning between 6 to 18 months of age a number of factors could cause actual to... Announce strategic investment to Support development of intrathecally-delivered AAV gene Therapies focused on inherited, systemic, debilitating chronic.. Of this progressive disease one or two of Audentes preclinical assets might expected... Our investigational Therapies target rare diseases through three modalities: gene replacement under. Failure, occurred in patients given the higher dose, 3.5x1014vg/kg 2.2mcg dose of VAX-24 met opsonophagocytic! Manufacturing capabilities for Astellas & # x27 ; s pipeline lists only those projects in human trials expected bicarbonate! Typically beginning between 6 to 18 months of age ; adeno advance &... Liver failure, occurred in patients given the higher dose, 3.5x1014vg/kg an agile operating model and growth...
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