Astellas Pharma

Novel therapies to reboot cells and organs

Cell & Gene Therapy

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 Taysha Gene Therapies is a gene therapy developer that manufactures gene-based treatments for medical conditions such as neurodegenerative diseases, neurodevelopmental diseases, and genetic epilepsies. The company uses edited genes via its AAV9 Capsid platform (a adeno-associated virus vector), which is used to make engineered genes replace mutated genes. Taysha&rsquo;s solution is administered intrathecally to directly deliver gene therapeutics into the cerebrospinal fluid for more optimal distribution. The company is listed on the Nasdaq and trades under the symbol TSHA.
 
 
 In February 2024, the company transferred the IP rights for several gene therapy programs to other organizations under a loan and security agreement. This move was in order to extend the company&rsquo;s cash runway into 2026. 
 
 
 As of May 2024, the company had two gene therapy candidates in development, both for the treatment of Rett syndrome. Both candidates were in Phase I/II of clinical testing. 
 
 
 
 
 Key customers and partnerships
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 Taysha and pharmaceutical company Catalent partnered in November 2020 to support the development and manufacturing capabilities of Tysha&rsquo;d gene therapeutics. Through the partnership Tysha would be able to use Catalent&rsquo;s manufacturing facilities to produce its gene therapies.
 
 
 
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Poseida Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies for cancer and rare diseases. The company's technology platform includes its proprietary non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-Specific Gene Editing System, and nanoparticle and hybrid gene delivery technologies. Their gene therapy pipeline includes P-KLKB1-101 for hereditary angioedema and P-FVIII-101 for hemophilia A. The company's cell therapy portfolio features allogeneic CAR-T cell therapy candidates targeting both solid and liquid tumors.&nbsp;As of November 2024, the company had 10 programs in its pipeline, comprising eight CAR-T programs and two genetic medicines, with three programs in Phase I clinical trials.Key customers and partnershipsThe company has established strategic collaborations with major pharmaceutical companies. In August 2022, Poseida partnered with Roche to develop allogeneic cell therapies for blood malignancies. In May 2024, the company entered into a research collaboration with Xyphos Biosciences, an Astellas Pharma subsidiary, to develop new allogeneic cell therapies for cancer. Additionally, Poseida has formed a partnership with Takeda.

UK-based AviadoBio is a developer of gene-based therapeutics that target neurodegenerative disorders. Specifically, the company develops therapeutics that help treat patients with conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). AviadoBio’s platform uses therapeutics based on gene supplementation and gene knock-down and is based on research carried out at King’s College London and the UK Dementia Research Institute.As of May 2024, the company had five therapeutic candidates; its lead candidate, AVB-101 for the testing of frontotemporal dementia with GRN mutations, was in its early clinical testing phase. The company’s other candidates were all in discovery stages for the treatment of ALS and Frontotemporal disorders. In April 2024, the company announced that the first patient had been treated with its AVB-101 candidate for frontotemporal dementia with GRN mutations as a part of its Phase I/II clinical trial.

A concentration of wealth and a willingness to adopt age-in-place technologies present new opportunities.

Age Tech

Catalia Health offers a personal healthcare companion device, “Mabu”, for homecare, to help patients adhere to medication in an engaging manner. The device and complementing platform Mabu Care Insights Platform combines robotics, artificial intelligence (AI), and Internet of Things (IoT) technologies.The device learns the interests and challenges of its users and creates customized conversations. The conversation platform can be deployed through multiple interfaces such as the Mabu device, mobile apps, text, and web. Catalia Health primarily targets elders with chronic illnesses, such as rheumatoid arthritis, late-stage kidney disease, and congestive heart failure. Key customers and partnershipsIn September 2019, Catalia Health launched a pilot project with Pfizer to test the impact of regular engagement with AI on a patient’s treatment journey. Moreover, in February 2020, it partnered with language and technology solutions provider, TrasPerfect DataForce, to launch a Spanish version of Mabu.

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Poseida Therapeutics specializes in gene editing to develop novel therapeutics for a range of liquid and solid tumors using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops autologous (using cells from the patients themselves) and allogeneic (using cells from other individuals) gene-edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells.&nbsp;Poseida operates a suite of platforms including non-viral piggyBac DNA Modification System for gene insertion, Cas-CLOVER for site-specific gene editing, and gene delivery technologies (in vivo and ex vivo) based on nanoparticle and adeno-associated virus (AAV) vectors. The company has demonstrated low to almost no off-target activities in the company’s preclinical studies using its Cas-CLOVER platform, which it considers to be an advantage over other existing gene editing systems.As of March 2024, the company has six programs in its CAR-T pipeline focused on oncology, of which three have reached Phase I clinical trials. It also has three gene therapies to treat Hemophilia A, Ornithine transcarbamylase deficiency (OTCD), and Phenylketonuria, all of which were in the preclinical stages . The company fully owns two of its CAR-T therapies for solid tumor indications and all of its gene therapies, while its remaining cell therapies are a part of a collaboration with Roche.&nbsp;Key customers and partnershipsThe company announced in August 2022 that it had entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. The global collaboration covered the research and development of multiple existing and novel "off-the-shelf" cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications.

Therapeutics that target specific areas/tissues to minimize treatment side effects.

Tissue Targeting Therapeutics

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 Poseida Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapeutics with the potential to cure various diseases. The company utilizes proprietary genetic engineering platform technologies, including the non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System, and nanoparticle- and AAV-based gene delivery technologies. These platforms, either alone or in combination, enable Poseida to engineer a portfolio of product candidates designed to overcome limitations of current cell and gene therapeutics.
 
 
 
 
 Poseida's pipeline includes both autologous and allogeneic CAR-T cell therapy programs targeting various indications. The company's P-PSMA-101 is an autologous CAR-T program for patients with metastatic castrate-resistant prostate cancer. In the allogeneic space, Poseida is developing P-BCMA-ALLO1, P-MUC1C-ALLO1, and P-CD19CD20-ALLO1, leveraging its proprietary technology to create off-the-shelf CAR-T therapies. The company's approach focuses on generating T-stem cell memory (Tscm) cells, which are associated with improved clinical responses and tolerability profiles.
 
 
 
 
 In gene therapy, Poseida is advancing P-OTC-101, a liver-directed program for the treatment of urea cycle disease caused by ornithine transcarbamylase (OTC) deficiency. The company's hybrid delivery approach aims to achieve durable expression at lower doses, potentially offering a more effective and tolerable treatment option. Additionally, Poseida is developing P-FVIII-101, a liver-directed gene therapy for Hemophilia A in partnership with Takeda, utilizing the piggyBac DNA Delivery System and biodegradable nanoparticle delivery.
 
 
 
 
 Poseida continues to innovate with emerging technologies such as the Site-Specific Super PiggyBac DNA Delivery system for highly specific DNA integration, and a TCR-T platform combining piggyBac and Cas-CLOVER technologies for off-the-shelf TCR-T product candidates. The company is also exploring a CAR 3.0 approach using genetically modified hematopoietic stem cells to create next-generation anti-cancer therapeutics.
 
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AviadoBio mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.

Poseida Therapeutics is a clinical-stage biopharmaceutical company utilizing genome engineering capabilities to develop targeted therapeutics for patients with high unmet medical needs. The company develops a pipeline of autologous and allogeneic chimeric antigen receptor T cell or CAR-T, product candidates focusing on the treatment of hematological malignancies and solid tumors.

Taysha Gene Therapies is a developer of treatments to eradicate severe & life-threatening monogenic diseases of the central nervous system. Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we are able to rapidly translate our treatments from bench to bedside. We have combined our team’s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to quickly and efficiently build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications.

The National Comprehensive Cancer Network® (NCCN®), a not-for-profit alliance of 26 of the world's leading cancer centers devoted to patient care, research, and education, is dedicated to improving the quality, effectiveness, and efficiency of cancer care so that patients can live better lives. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. As the arbiter of high-quality cancer care, NCCN promotes the importance of continuous quality improvement and recognizes the significance of creating clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers.  World-renowned experts from NCCN Member Institutions diagnose and treat patients with a broad spectrum of cancers and are recognized for dealing with complex, aggressive, or rare cancers. NCCN Member Institutions pioneered the concept of the multidisciplinary team approach to patient care and conduct innovative research that contributes significantly to understanding, diagnosing, and treating cancer. NCCN programs offer access to expert physicians, superior treatment, and quality and safety initiatives that continuously improve the effectiveness and efficiency of cancer care.

LabCentral developing novel cancer treatments: Conkwest, Inc, headquartered in DelMar, California, is opening its research laboratory at LabCentral. Conkwest is an innovative life sciences company that is developing and commercializing cell based treatments for cancer and viral infections.

Founded in 2017 to build on the award-winning ultrasound technology invented at the University of California, Berkeley, iota Biosciences is working to improve human life by pioneering cutting-edge bioelectronics. Since launch, driven by passion and tenacity, with an unwavering commitment to quality, iota has progressed from ideation to functional technology at a significant pace. We are committed to maintaining this momentum and bringing our groundbreaking approaches to the patients who need them. Using innovative science and technology, our mission is to create and develop state-of-the-art diagnostic and therapeutic bioelectronic devices that are safe and effective. We are working to transform the way doctors manage and treat disease to make a life-changing difference for patients. Today, we are proud and privileged to be powered by Astellas, a leading global pharmaceutical company that brings experience, reach and rigor, with an ethical and transformational vision aligned with our own. Together, we are creating innovative technologies that will change patients’ lives.

Catalia Health brings together artificial intelligence, psychology, and medicine to deliver lasting change. We create effective patient engagement and generate unique data of value to our customers. Our cloud-based platform delivers unique conversations to patients to help address their ongoing healthcare issues in an effective way. These messages are delivered through mobile apps, a web interface, and our uniquely engaging (and patented) interactive robotic coach.

Mitobridge is discovering and developing small molecule therapeutics that improve mitochondrial functions. Building upon the emerging scientific findings linking mitochondrial dysfunction with disease pathologies, the Company is progressing innovative approaches to the treatment of diseases with a high unmet medical need. Their strategy is to establish proof of concept in rare diseases and then expand into more common diseases. Mitobridge has assembled a strong team with expertise in advancing ground-breaking therapeutics into the clinic. Their scientific founders and advisors include leaders in mitochondrial biology, metabolism, and aging with experience in translating novel discoveries into next-generation medicines.  The Company was founded in October of 2013 with financing from MPM Capital, Longwood Fund, and Astellas Pharma and a shared vision for the promise of mitochondrial-targeted therapeutics.

Telsar Pharma Inc., a new company that will develop an Astellas drug for a common inflammatory bowel disease.

CoMentis is a biotech company focused on the research and development of small molecule drugs to treat neurovascular diseases such as Alzheimer's disease (AD), age-related macular degeneration (AMD) and other cognitive disorders. Originally founded in 2004 as Athenagen, Inc., the company was re-named CoMentis following its August 2006 merger with Zapaq, Inc., which created a leading neurovascular disease franchise.

FibroGen is a research-based biotechnology company using its expertise in connective tissue growth factor (CTGF) and hypoxia-inducible factor (HIF) biology to discover, develop, and commercialize novel therapeutics for serious unmet medical needs. The company applies its pioneering expertise in hypoxia-inducible factor (HIF), connective tissue growth factor (CTGF) biology, and clinical development to advance innovative medicines for the treatment of anemia, fibrotic disease, and cancer. FibroGen's product Roxadustat is an oral small-molecule inhibitor of HIF prolyl hydroxylase activity in worldwide Phase 3 clinical development for the treatment of anemia in chronic kidney disease (CKD), with a New Drug Application currently under review in China by the State Drug Administration or SDA (formerly the China Food and Drug Administration).

At Astellas, our vision is to be at the forefront of healthcare change by turning innovative science into meaningful VALUE for patients. As a specialty global pharmaceutical company, we focus on accelerating the discovery, development and commercialization of ground-breaking innovations that could redefine expectations of care for difficult-to-treat diseases. Together with our partners, we aim to lead in the areas where we can deliver the greatest value, with a focus on immuno-oncology, gene therapy, mitochondria, blindness and regeneration and targeted protein degradation. Astellas operates its business in approximately 70 countries around the world. It has approximately 14,000 employees, and its core products include anticancer agents like XTANDI, XOSPATA, and PADCEV; VEOZAH for the treatment of vasomotor symptoms due to menopause; Betanis / Myrbetriq / BETMIGA for the treatment of overactive bladder (OAB); and Prograf, an immunosuppressant for organ transplantation. In its FY2022 financial results, revenue achieved about 1,500 billion yen (ca.$11 billion*), and R&D expenses were about 280 billion yen (ca.$2.0 billion*). *The exchange rate for the yen, 1USD: 142yen

<div> Astellas Pharma Inc. (TSE: 4503) is a pharmaceutical company headquartered in Tokyo, Japan. The company was formed in 2005 through the merger of Fujisawa Pharmaceutical and Yamanouchi Pharmaceutical. Astellas conducts business in over 70 countries worldwide, focusing on developing innovative therapies in areas with high unmet medical needs. Astellas' product portfolio and pipeline span multiple therapeutic areas, including oncology, urology, immunology, nephrology, and neuroscience. Some of the company's key products include XTANDI (enzalutamide) for prostate cancer, PADCEV (enfortumab vedotin) for urothelial cancer, and zolbetuximab, an investigational therapy for gastric and gastroesophageal junction cancers. The company employs a &quot;Focus Area Approach&quot; to identify opportunities for creating new drugs by concentrating on biology and modality. Astellas is also exploring &quot;Rx+&quot; healthcare solutions that combine its pharmaceutical expertise with cutting-edge technologies from external partners. Astellas continues to expand its oncology portfolio through internal research and development as well as strategic collaborations. Recent initiatives include partnerships with YASKAWA Electric Corporation to create an innovative cell therapy ecosystem, a joint venture with Takeda and Sumitomo Mitsui Banking for incubating early drug discovery programs, and a collaboration with Sony Corporation to develop a novel antibody-drug conjugate platform. The company is actively pursuing regulatory approvals for its pipeline candidates across major markets including the US, Europe, Japan and China. </div>

Sangamo to receive $20M from Astellas Pharma in capsid license agreement

Astellas Venture Management

Companies producing cells, genes, tissues, and biomaterials such as exosomes and extracellular matrix for drug developers and pharmaceutical companies developing regenerative medicine

Chimeric antigen receptor T-cell (CAR-T) therapy involves genetically engineering T cells to target and kill cancer cells

Companies conducting R&D on therapies that modify or introduce new genes into the body to treat diseases

Includes therapies that use cells (other than CAR-T and stem cells) such as Adipose-Derived Regenerative Cells (ADRCs), cardiosphere-derived cells, and gamma delta T cells (GDT cells)

Stem cell therapy involves using stem cells usually extracted from the bone marrow and umbilical cord blood to repair a variety of diseases

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

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 Astellas Pharma, a global pharmaceutical company, has made a licensing agreement with gene therapy firm AviadoBio. This agreement gives Astellas exclusive rights to AviadoBio's AVB-101, an AAV-based gene therapy under Phase 1/2 development. Astellas is going to invest USD 20 million in equity and an upfront payment of up to USD 30 million for the licensing option to AVB-101. If Astellas decides to exercise this option, AviadoBio could earn USD 2.18 billion from license fees and milestone payments along with royalties.
 AVB-101 is intended to treat frontotemporal dementia with progranulin mutations (FTD-GRN), a dementia often misdiagnosed or undiagnosed. This alliance plays an important role in speeding up the development and global commercialization of a potential therapy for patients living with FTD-GRN and related neurological disorders.
 
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