XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition.
Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health. Bile is a liquid made in the liver that helps digest fat. Ongoing liver health checks may help with the routine care of people with XLMTM.
There is a need to understand liver problems that develop in individuals with XLMTM over time. The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study.
This study is about collecting information only. This is known as an observational study. The individual's doctor decides on treatment, not the study sponsor (Astellas).
In this study, boys under 18 diagnosed with XLMTM will be followed for about 1 year. The health of their liver and gallbladder will be checked about every 6 weeks. This can be done at home, if preferred. A scan called a Fibroscan (also known as transient elastography) will check for signs of scarring in the liver (fibrosis) and the build-up of lipids. It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study.
This study will help understand liver, gallbladder, and bile duct issues in individuals with XLMTM over time. The goal is to improve their care and provide information to use in future clinical studies.

Start Date31 Dec 2024

Sponsor / Collaborator

Astellas Gene Therapies, Inc.

NCT06483802 / Not yet recruitingPhase 1

A Multicenter, Open-Label, Dose Escalation, Phase 1b Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2016 for Friedreich Ataxia Cardiomyopathy

Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech. It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems. Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families. This type of gene therapy treats a genetic condition by providing a healthy copy of the gene.
At the time this study started, there was no approved treatment for heart problems in people with Friedreich Ataxia.
In this study, ASP2016 is being tested in humans for the first time. The people taking part are adults with Friedreich Ataxia who have heart problems.
The main aims of the study are to check the safety of ASP2016 and how people cope with (tolerate) ASP2016. ASP2016 is given as a slow injection into a vein. This is called an infusion. People will also take tablets of a medicine called prednisolone. This is taken to stop the immune system interfering with ASP2016.
Each person in the study will be given 1 single infusion of ASP2016. Different small groups will receive lower or higher doses of ASP2016. Each person will stay overnight in the clinic for at least 1 night after their infusion.
For the first few months, people will visit the clinic regularly. There may be the option of home visits by a study nurse at some visits. At the 6-month and 12-month visits extra tests, procedures, and scans will be done. One of these is an ECHO (echocardiogram) scan. This is like an ultrasound scan for the heart. Another is an endomyocardial biopsy. A tiny piece of their heart tissue is removed (biopsy). A flexible hollow tube (catheter) goes into the blood vessels up to the heart. Then, a small device on the end of the catheter takes a tiny piece of heart tissue (about the size of a pencil tip). Another is a cardiac MRI. This takes pictures of the inside of the heart using a powerful magnet. Another is a cardiopulmonary exercise test (CPET). This involves moving a specially designed set of bicycle pedals using hands and arms. This will check how the lungs, heart and muscles are affected during exercise.
After the 12-month visit, people will visit the clinic every few months for up to a few years.

Start Date30 Sep 2024

Sponsor / Collaborator

Astellas Gene Therapies, Inc.

NCT06150820 / RecruitingNot Applicable

A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease

Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the cells of the body. This causes muscle weakness and other symptoms. Pompe disease can happen at any age, but in late-onset Pompe disease, symptoms generally start from 12 months old onwards.
The standard treatment for people with Pompe disease is to receive regular infusions of the GAA enzyme. This is known as enzyme replacement therapy. However, people can build up antibodies against the GAA enzyme over time.
Gene therapy is used to treat conditions caused by a faulty gene. It works by replacing the faulty gene with a working gene inside the cells of the body. The working gene is delivered into the cells using certain viruses as carriers (vectors). Viruses are often used as carriers as they can easily get inside cells. The genetic material of the original virus is replaced with the working gene, so only the working gene gets inside the cells. A common virus used as a carrier in gene therapy is the adeno-associated virus (or AAV). This is like an adenovirus, which causes the common cold.
The original type of AAV does not cause any harm to humans. However, people that have previously been infected with the original type of AAV may have built up antibodies against AAV. These antibodies may stop the AAV carrier with the working gene getting inside the cells.
Researchers want to learn more about antibody levels against AAV and the GAA enzyme in people with late-onset Pompe disease. They also want to learn about other substances in the blood that provide more information about late-onset Pompe disease. These are known as biomarkers.
In this study, older teenagers and adults with late-onset Pompe disease will take part. They will not have had gene therapy using AAV. There will be 2 groups - those who have never had enzyme replacement therapy, and those who have had enzyme replacement therapy for 6 months or more. No study treatment will be given during the study, but blood and urine samples will be taken for testing.
The main aims of the study are to check antibody levels against AAV8 (a type of AAV) in people with late-onset Pompe disease who had not received any treatment using AAV, to check antibody levels against the GAA enzyme in people previously treated with GAA as part of enzyme replacement therapy, to check levels of biomarkers for Pompe disease, and to check for medical problems.
In the study, people will visit the study clinic several times. Some visits may be in the person's home. The first visit is to check if they can take part. Those who can take part will have a medical examination, and have their vital signs checked. Vital signs include blood pressure, heart rate, breathing rate and temperature. Blood samples will be taken to check antibody levels against the GAA enzyme and against AAV8. Blood and urine samples will also be taken to check for biomarkers for Pompe disease. Blood and urine samples will be taken about every 4 months for up to 2 years.

Start Date01 Feb 2024

Sponsor / Collaborator

Astellas Gene Therapies, Inc.

100 Clinical Results associated with Astellas Gene Therapies, Inc.

0 Patents (Medical) associated with Astellas Gene Therapies, Inc.

Literatures (Medical) associated with Astellas Gene Therapies, Inc.

01 Mar 2024·Molecular Therapy - Methods & Clinical Development

AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia

Article

Author: Chang, Joshua C ; Purushothaman, Pravinkumar ; Johnson, Caitlin A ; Halawani, Sarah ; Ryan, Molly R ; Liu, Su ; Davis, Crystal ; Ngaba, Lucie V ; Medicherla, Bala ; Meng, Hui ; Bolan, Fria ; Champe, Mark ; Lutz, Cathleen ; Gonzalo-Gil, Elena ; Stark, Marie C ; Urbinati, Fabrizia ; Fonck, Carlos ; Lynch, David R ; Lawlor, Michael W

Friedreich's ataxia (FRDA) is an autosomal-recessive disorder primarily attributed to biallelic GAA repeat expansions that reduce expression of the mitochondrial protein frataxin (FXN). FRDA is characterized by progressive neurodegeneration, with many patients developing cardiomyopathy that progresses to heart failure and death. The potential to reverse or prevent progression of the cardiac phenotype of FRDA was investigated in a mouse model of FRDA, using an adeno-associated viral vector (AAV8) containing the coding sequence of the FXN gene. The Fxn^flox/null::MCK-Cre conditional knockout mouse (FXN-MCK) has an FXN gene ablation that prevents FXN expression in cardiac and skeletal muscle, leading to cardiac insufficiency, weight loss, and morbidity. FXN-MCK mice received a single intravenous injection of an AAV8 vector containing human (hFXN) or mouse (mFXN) FXN genes under the control of a phosphoglycerate kinase promoter. Compared to vehicle-treated FXN-MCK control mice, AAV-treated FXN-MCK mice displayed increases in body weight, reversal of cardiac deficits, and increases in survival without apparent toxicity in the heart or liver for up to 12 weeks postdose. FXN protein expression in heart tissue was detected in a dose-dependent manner, exhibiting wide distribution throughout the heart similar to wild type, but more speckled. These results support an AAV8-based approach to treat FRDA-associated cardiomyopathy.

13 Apr 2021·Neurology

A phase I/II open-label gene replacement clinical study for late onset Pompe disease (4399)

Author: Mozaffar, Tahseen ; Po Foo, Cheryl Wong ; Conner, Edward ; Day, John ; Bachtell, Nathan

14 Apr 2020·Neurology

ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment (1053)

Author: Sarah Neuhaus ; Francesco Muntoni ; James Dowling ; Tina Duong ; Suyash Prasad ; Carsten G. Bönnemann ; Laurent Servais ; Barbara Smith ; Ana Buj-Bello ; Perry B. Shieh ; Astrid Blaschek ; Michael Murtagh ; Lindsay N. Alfano ; Nancy Kuntz ; Robert J. Graham ; Wolfgang Müller-Felber ; Victoria MacBean ; Michael W. Lawlor ; Fulvio Mavilio ; Minal Jain ; Mojtaba Noursalehi ; Jun Lee ; Salvador Rico ; Emma S. James ; Alan H. Beggs ; Martin (Casey) Childers

Objective: ASPIRO is an ongoing, open-label, randomized study (NCT03199469) evaluating safety/efficacy of AT132, an investigational gene therapy for delivery of functional MTM1 gene copies. Background: XLMTM, a rare monogenic disease caused by mutations in the MTM1 gene, is characterized by profound muscle weakness, respiratory failure, and early death. Design/Methods: XLMTM patients, ≤7 years old, were randomized to treatment or delayed-treatment control and enrolled into ascending dose cohorts to receive a single AT132 infusion. Safety/efficacy data are available for 12 patients with 4–72 weeks’ follow-up: 6, Cohort 1 (1x1014 vg/kg); 4, Cohort 2 (3x1014 vg/kg); 2, untreated controls. Muscle biopsy data are available for 9 treated patients: 6, Cohort 1; 3, Cohort 2. Results: Since study initiation (Sep17) there were 82 AEs considered related/possibly related (14 serious AEs; 68 non-serious). At baseline, all patients required 12–24 hours/day ventilatory support, and missed critical motor milestones. As of 7Aug19, clinically meaningful changes from baseline in CHOP-INTEND and MIP scores were observed in all but one treated patient. Treated patients also achieved important motor milestones: ability to sit unassisted, raise self to stand, and walk supported or independently. There was significant and rapid reduction in ventilator use in all but one treated patient, with 7 treated patients reaching ventilator independence. These improvements contrast with the two untreated ASPIRO controls and the INCEPTUS contemporaneous external control subjects (NCT02704273) in whom gains of motor skills and achievement of ventilator independence were not observed. Muscle biopsies demonstrated robust dose-dependent tissue transduction and myotubularin expression with considerable improvement in histopathology. Conclusions: AT132 has shown a manageable safety profile across dose cohorts. Clinically meaningful improvements in neuromuscular and respiratory function continue through 72 weeks post-dose. An optimal dose (3x1014 vg/kg) has been selected for the study’s confirmatory phase. Updated data will be presented at the 2020 AAN Annual Congress. Disclosure: Dr. Shieh has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes, Sarepta, Pfizer, Genentech, Avexis, Biogen, Catalyst, Argenyx, Alexion, CSL Behring, Grifols. Dr. Shieh has received research support from Sarepta, Pfizer, Audentes, Avexis, Biogen, PTC, Roche, Sanofi, Reveragen, Acceleron.Dr. Kuntz has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for Argenyx, Audentes, AveXis, Biogen, Cytokinetics, PTC, Roche, and Sarepta.. Dr. Kuntz has received research support from Clinical trial research contracts with Audentes, AveXis, Biogen, Pfizer, Roche, and Sarepta.. Dr. Smith has nothing to disclose. Dr. Dowling has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Dynacure, RYR1 Foundation, MD Canada, MDA USA, Deep Genomics. Dr. Mueller-Felber has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes, Avexis, Biogen, Cytokinetics, PTC, Roche, Sanofi-Aventis. Dr. Bonnemann has nothing to disclose. Dr. Servais has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Avexis, Inc., Biogen, Biophytis, Cytokinetics, Dynacure, Roche, Santhera, Sarepta Therapeutics. Dr. Servais has received research support from Avexis, Inc., Biogen, Dynacure, and Roche. Dr. Muntoni has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for AveXis, Biogen, Cytokinetics, Novartis, Pfizer, PTC, Roche, Sarepta, Summit, and Wave. Dr. Muntoni has received research support from Principal Investigator for ongoing Ionis Pharmaceuticals, Inc./Biogen and Roche clinical trials; funding from Muscular Dystrophy UK, SMA Europe, and SMA Trust UK. Dr. Blaschek has nothing to disclose. Dr. Neuhaus has nothing to disclose. Dr. Alfano has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Acceleron Pharma. Dr. Alfano has received research support from Sarepta Therapeutics. Dr. Beggs has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics, Dynacure, Roche Pharmaceuticals, Guidepoint. Dr. Beggs has received research support from NIH, MDA, Joshua Frase Foundation, Lee and Penny Anderson Family Foundation, AFM-Telethon. Dr. Buj-Bello has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Childers has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics, AskBio. Dr. Duong has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AMO, Audentes, AveXis, Biogen, Cytokinetics, Santhera, Sarepta. Dr. Duong has received research support from AveXis, Biogen, Cytokinetics, Genzyme, Ionis, Roche, Santhera, Sarepta. Dr. Graham has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Jain has nothing to disclose. Dr. James has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Lawlor has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics, Solid Biosciences, Dynacure, AGADA Biosciences. Dr. Lawlor has received research support from Audentes Therapeutics, Solid Biosciences. Dr. Lee has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. MacBean has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Mavilio has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Murtagh has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Noursalehi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Prasad has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics. Dr. Rico has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Audentes Therapeutics.

News (Medical) associated with Astellas Gene Therapies, Inc.

10 Oct 2024

·www.biospace.com

Astellas Cuts 24 Universal Cells Roles in Seattle, Transfers 12 to Japan

iStock, JHVEPhoto Astellas is opening a second location of Universal Cells, its wholly owned subsidiary, at a research campus in Japan and transferring 12 roles from Universal’s Seattle location. Astellas Pharma will eliminate 24 roles at Universal Cells, its wholly owned subsidiary in Seattle, and transfer 12 of them to a new Universal site opening at the pharma’s research campus in Tsukuba, Japan, according to Fierce Pharma . Employees at the Tsukuba location, which will reportedly be Universal’s second facility, will fill the transferred roles. Regarding the role changes and new site, an Astellas spokesperson told Fierce the company regularly reviews its organizations and operations to increase efficiency and leverage new technologies and innovation. The spokesperson added that the move should expand Universal Cells’ gene editing capabilities and evolve it into a center of excellence for cell therapy R&D. Astellas acquired Universal for $102.5 million in 2018. The subsidiary produces engineered stem cell lines designed to avoid rejection and be used in allogeneic cell therapy treatments. This week’s news marks the second major milestone for an Astellas business in recent months. Astellas Gene Therapies is in the process of closing its San Francisco biomanufacturing facility , a move that will affect about 100 employees. The closure is expected to be complete in March 2025, with all programs and projects shifting to Astellas Gene Therapies’ Sanford, North Carolina, site. In addition, the company announced in September that it’s opened the Astellas Life Sciences Center in Cambridge, Massachusetts. About 400 Astellas employees will work in the facility, which was designed to accelerate innovation across the state by connecting incubators, biotechnology start-ups and academia, according to the announcement.

Cell TherapyAcquisitionGene Therapy

09 Oct 2024

·pipelinereview.com

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications

Astellas receives an exclusive option to license AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development AviadoBio receives $20 million equity investment, up to $30 million in upfront payments and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option LONDON, UK and TOKYO, Japan I October 8, 2024 I AviadoBio Ltd. (“AviadoBio”) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN). FTD is a devastating form of early-onset dementia that typically leads to death within three to 13 years from diagnosis. People with FTD commonly experience a rapid decline in executive function (attention control, working memory, problem-solving etc.), uncharacteristic behaviors, loss of language, apathy, and reduced mobility. It is an important cause of dementia in those under the age of 65 and is often underrecognized, and misdiagnosed. 1,2,3,4 Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option. Lisa Deschamps, CEO, AviadoBio “As we complete dosing of the first cohort of patients in our Phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia. This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas’ global capabilities in development and commercialization of gene therapies. Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases.” Adam Pearson, Chief Strategy Officer, Astellas “We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases. AVB-101 represents a truly innovative approach to the treatment of FTD-GRN and has the potential to be part of the next generation of gene therapy products through the creation of this agreement. Genetic regulation remains a cornerstone of our primary focus strategy at Astellas and this agreement helps us to continue to provide potential solutions for patients in need.” About AviadoBio At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc Ventures. For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio . About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. 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. 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. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at https://www.astellas.com/en . 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. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan. SOURCE: Astellas Pharma

License out/inGene Therapy

08 Oct 2024

·www.prnewswire.com

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications

Astellas receives an exclusive option to license AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development AviadoBio receives $20 million equity investment, up to $30 million in upfront payments and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option LONDON and TOKYO, Oct. 8, 2024 /PRNewswire/ -- AviadoBio Ltd. ("AviadoBio") and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN). FTD is a devastating form of early-onset dementia that typically leads to death within three to 13 years from diagnosis. People with FTD commonly experience a rapid decline in executive function (attention control, working memory, problem-solving etc.), uncharacteristic behaviors, loss of language, apathy, and reduced mobility. It is an important cause of dementia in those under the age of 65 and is often underrecognized, and misdiagnosed. 1,2,3,4 Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option. Lisa Deschamps, CEO, AviadoBio "As we complete dosing of the first cohort of patients in our Phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia. This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas' global capabilities in development and commercialization of gene therapies. Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases." Adam Pearson, Chief Strategy Officer, Astellas "We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases. AVB-101 represents a truly innovative approach to the treatment of FTD-GRN and has the potential to be part of the next generation of gene therapy products through the creation of this agreement. Genetic regulation remains a cornerstone of our primary focus strategy at Astellas and this agreement helps us to continue to provide potential solutions for patients in need." About AviadoBio At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King's College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients. AviadoBio's investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), SV Health Investor's Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc Ventures. For more information, please visit and follow us on X @AviadoBio and LinkedIn at AviadoBio. About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. 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. 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. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at . 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. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan. Astellas Cautionary Notes In 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. These statements are based on management's current assumptions and beliefs in light of the 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. 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. 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. Onyike CU and Diehl-Schmid J. Int Rev Psychiatry. 2013;25(2):130–137; Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310; Onyike CU. Neuroepidemiology. 2011;37:166–167; Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122; SOURCE Astellas Pharma Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

License out/inGene Therapy

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Pipeline Snapshot as of 24 Dec 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Preclinical

Phase 1 Clinical

Phase 2 Clinical

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

Volrubigene Ralaparvovec ( UGT1A1 )	Crigler-Najjar Syndrome More	Phase 2 Clinical
Zocaglusagene nuzaparvovec ( Glycogen )	Glycogen Storage Disease Type II More	Phase 2 Clinical
scAAV9.U7.ACCA ( DAG1 )	Muscular Dystrophy, Duchenne More	Phase 2
ASP-2016 ( FXN )	Friedreich Ataxia More	Phase 1
AT-720	Neuromuscular Diseases More	Preclinical

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