On December 1, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Dyno Therapeutics, Inc. (President and CEO: Eric Kelsic, Ph.D., "Dyno") reported an option and license agreement was signed on November 23 to develop next-generation adeno-associated virus (AAV) vectors for gene therapy directed to skeletal and cardiac muscle using Dyno’s CapsidMap platform (Press release, Astellas, DEC 2, 2021, View Source [SID1234596344]).

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Dyno’s CapsidMap platform represents a transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids – the cell-targeting protein shells of viral vectors – designed to optimize tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability. Unlike traditional approaches, CapsidMap is uniquely well-suited to simultaneously optimize capsids for delivery across multiple organs, with the goal of enabling more effective whole-body treatment for many diseases.

With the establishment of the Astellas Gene Therapies Center of Excellence following the 2020 acquisition of Audentes Therapeutics Inc., Astellas is a leader in genetic medicines, working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. This research collaboration combines Dyno’s AI-powered AAV vector engineering capabilities with Astellas Gene Therapies global leadership in AAV-based pipeline assets.

"Through our efforts in gene therapy and the Astellas Gene Therapies Center of Excellence, Astellas strives to identify, develop and deliver transformative gene-based therapies for patients with genetic diseases who currently have few or no effective treatment options. Our principal focus is on developing adeno-associated virus delivered therapies for the treatment of well-defined serious diseases," said Naoki Okamura, Chief Strategy Officer and Chief Financial Officer, Chief Business Officer at Astellas. "We are dedicated to delivering novel approaches and utilizing new technologies that can deliver transformational value for patients."

"We are so happy to be partnering with Astellas, a world leader in developing gene therapies. Dyno and Astellas each bring unique strengths to this collaboration, together enabling more rapid creation of new therapies for patients with great unmet need," said Dyno’s CEO and co-founder Eric D. Kelsic, Ph.D. "This partnership demonstrates the flexibility of Dyno’s platform to precisely design the delivery properties of gene therapy vectors towards multi-organ and disease-specific profiles, applying the scientific insights we are rapidly learning across all our partnered and internal vector engineering programs using CapsidMap."

Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities, including manufacturing, of gene therapy product candidates using the novel capsids. Dyno will receive an $18 million upfront payment and be eligible to receive additional payments during the research phase of the collaboration as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential value of future milestone and royalty payments to Dyno exceeds $235 million per product and over $1.6 billion in total value.

On December 2, 2021 Clarity Pharmaceuticals (ASX: CU6) ("Clarity"), a clinical-stage radiopharmaceutical company developing next-generation products to address the growing needs in oncology, and Cardinal Health (NYSE: CAH), reported that the companies have entered into an agreement covering Clarity’s Targeted Copper Theranostics (TCT) platform (Press release, Clarity Pharmaceuticals, DEC 2, 2021, View Source [SID1234596385]).

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Cardinal Health will provide cGMP product manufacturing of ready-to-use TCT for Clarity’s U.S.-based clinical trials from its Center for Theranostics Advancement in Indianapolis, Indiana. TCT are investigational radiopharmaceuticals that pair copper-64 (64Cu) and copper-67 (67Cu) for diagnosis and therapy.

With more than 40 years of experience in the radiopharmaceutical industry, Cardinal Health provides radiopharmaceutical development support, manufacturing, preparation, commercialization, and business and practice management solutions across the care delivery continuum from origin to patient administration. Its Center for Theranostics Advancement is helping shape the future of precision medicine by providing radiopharmaceutical innovators the solutions they need at every stage of the product lifecycle.

Clarity’s Executive Chairman, Dr Alan Taylor, said, "We are pleased to have further strengthened and expanded our U.S. manufacturing capabilities, ensuring seamless product supply for our clinical trials, including those in cancer indications with large patient populations. Cardinal Health has an intricate understanding of the supply chain requirements for copper-based products and the capability to mass-produce these products.

"This is a testament to Clarity’s pairing of copper isotopes fitting into the ‘goldilocks zone’ of half-lives when considering biological half-life of the targeting moieties, centralized manufacture, and broad distribution. The half-life of copper-64 is nearly identical to that of iodine-123, for which Cardinal Health has an established and robust distribution system across the U.S. For the therapeutic isotope copper-67, centralized distribution is well proven in an isotope of comparable half-life, yttrium-90, or the much shorter half-life of samarium-153."

Cardinal Health Nuclear & Precision Health Solutions President Mike Pintek, said, "We are excited to be working together with Clarity on the manufacturing and pharmacy dispensing of the TCT platform of investigational products. The 64Cu and 67Cu isotope pairing holds great promise for the field of radiopharmaceuticals as they are ideally suited for central manufacturing of cGMP ready-to-use products; they will fit easily within our broad distribution network. We are very pleased to enter this agreement and hope it will be another avenue for us to help improve access to next-generation radiopharmaceuticals for diagnosis and treatment of a range of cancers."

Dr Alan Taylor said, "Clarity’s TCT platform is clearly differentiated in the radiopharmaceutical field as all products, once approved by the FDA, can be provided as ready-to-use to patients in any location in the U.S. from a single manufacturing site. The manufacturing agreement with Cardinal Health, in addition to the recent manufacturing agreement signed with Evergreen Theragnostics and other partners, will enable Clarity to create redundancies in the supply of our products – ensuring that we can provide a reliable, seamless supply of TCT that is unique for a company in the radiopharmaceutical field. This agreement further enables Clarity to continue to pursue our ultimate goal of improving treatment of children and adults with cancer."

This announcement has been authorized for release by the Executive Chairman of Clarity Pharmaceuticals.

On December 2, 2021 Novartis reported that holds an investor event to provide a comprehensive overview of the company’s progress in advancing its industry-leading R&D engine (Press release, Novartis, DEC 2, 2021, View Source [SID1234596404]).

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Vas Narasimhan, CEO of Novartis, said "Novartis has transformed to become a focused medicines company, building depth in our core therapeutic areas and strength across key technology platforms. We expect to continue delivering strong operational performance, with 4%+ CAGR through to 2026*, driven by the momentum of our multi-billion dollar in-market growth drivers. Up to 20 new assets with significant sales potential could be approved by 2026, which will fuel the next phase of growth and address major unmet needs. We are building the foundation for long-term differential growth by investing in advanced technology platforms and data science. Novartis remains disciplined and shareholder focused in its capital allocation priorities, as we continue to deliver on our strategy".

New announcements at R&D Day 2021:

Cosentyx, our largest medicine by sales, showed topline results in moderate to severe hidradenitis suppurativa (HS), a potential new indication. Two Phase 3 studies (SUNRISE and SUNSHINE) met their primary endpoint, with more patients treated with Cosentyx achieving a HS Clinical Response (HiSCR), compared with placebo, at week 16. The safety of Cosentyx in HS was consistent with the therapy’s known safety profile. The trials are ongoing to 52 weeks and are expected to complete in H2 2022. Regulatory filings are planned for 2022.

Novartis presents T-Charge, a next generation CAR-T cell therapy platform, expected to increase CAR-T potency and have important process efficiencies to reduce turnaround time. In first-in-human trials to be presented at ASH (Free ASH Whitepaper) 2021, lead candidates YTB323 and PHE885 showed 75% Complete Response in Diffuse Large B-Cell Lymphoma (DLBCL) at three months and 100% Best Overall Response (BOR) in multiple myeloma, respectively. Novartis is developing T-Charge as the foundational platform for a wave of potentially transformative CAR-T cell therapies.2,3

Phase 3 study starts planned or ongoing across 5 core therapeutic areas include:

Cardio-Renal: Leqvio (CVRR-LDL-C), pelacarsen (CVRR-Lp(a)), iptacopan (C3G; IgAN)
IHD: Cosentyx (HS; GCA; lupus nephritis), ligelizumab (CSU; food allergy; CINDU), ianalumab (Sjögren’s syndrome), remibrutinib (CSU)
Neuroscience: Zolgensma (SMA IT), remibrutinib (MS)
Oncology: Kisqali (HR+/HER2- BC adjuvant), 177Lu-PSMA-617 (mCRPC, pretaxane; mHSPC), canakinumab (adjuvant NSCLC), NIS793 (PDAC), JDQ443 (NSCLC, 2/3L)
Hematology: Iptacopan (aHUS; PNH), Scemblix (CML 1L), sabatolimab (HR-MDS), and YTB323 (2L DLBCL)
(for abbreviations, see below)

Novartis announces a global co-development and co-commercialization agreement with UCB to bring disease-modifying therapies to people living with Parkinson’s Disease. The agreement covers UCB0599, a potential first-in-class, small molecule, alpha-synuclein misfolding inhibitor currently in Phase 2 clinical development. In addition, upon completion of the ongoing Phase 1 program, there is an opt-in to co-develop UCB7853, an anti-alpha-synuclein antibody. Both assets could transform care for 10 million people living with Parkinson’s Disease worldwide given the lack of disease-modifying therapies.4,5

Novartis also provides a comprehensive overview of its mid- and late-stage pipeline assets in five core therapeutic areas, highlighting: Leqvio, pelacarsen, iptacopan, Cosentyx, ligelizumab, remibrutinib, ianalumab, LNA043, branaplam, Zolgensma, Kisqali, 177Lu-PSMA-617, sabatolimab, JDQ443, TNO155, Scemblix and NIS793.

Additionally, Novartis highlights the continued expansion of its pipeline and capabilities in advanced technology platforms that are expected to drive multiple waves of biopharmaceutical innovation. These include: T-Charge, Targeted Protein Degradation, Cell Therapy, Gene Therapy, Radioligand Therapy and xRNA.

On December 2, 2021 Leucid Bio ("Leucid" or the "Company"), a biotech company pioneering next-generation cell therapies for hard-to-treat cancers, reported that it has entered a strategic collaboration with Lonza to utilize Lonza’s Cocoon Platform for the Company’s forthcoming Phase I clinical trial, through to commercial manufacturing (Press release, Lonza, DEC 2, 2021, View Source [SID1234596422]).

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Under the terms of the collaboration, Leucid will utilise Lonza as its preferred manufacturer, aiming to deliver high quality cell therapies quickly and cost-effectively to patients in a decentralized manufacturing model. Leucid is, in turn, one of Lonza’s preferred collaborators on the Cocoon Platform, and both companies will work together to optimise and streamline the manufacturing process for Leucid’s CAR-T therapies. As a preferred collaborator, Leucid will have early access to new Cocoon Platform technologies.

Founded to translate 20 years of pioneering CAR-T research led by Dr John Maher at King’s College London, Leucid has developed a proprietary engine that builds upon Dr Maher’s novel parallel CAR-T model which positions these molecules in a more natural biological configuration. The Company’s technology confers properties on the CART-cells that enable them to consistently outperform previous generations of CAR-T therapies in pre-clinical studies. As a result, this leads to enhanced T-cell potency and persistent long-term response with reduced toxicity.

Artin Moussavi, Chief Executive Officer of Leucid Bio, said: "Following on from our successful Series A financing, we are delighted to be working with Lonza and its Cocoon Platform. This agreement will allow us to accelerate our path to the clinic, treating more patients sooner, potentially at point-of-care, and help to solve the key issues facing CAR-T therapeutics."

John Maher, Chief Scientific Officer of Leucid Bio, added: "This is a fantastic opportunity to collaborate with Lonza, enabling us to develop a state-of-the-art scalable manufacturing process. This will significantly help Leucid to efficiently transition our CAR-T pipeline for maximum patient benefit."

Nicholas Ostrout, Head of Commercial Development within Personalized Medicine at Lonza, commented: "We look forward to working with Leucid to bring its revolutionary, novel cancer immunotherapies to the clinic and patients. Use of the Cocoon Platform, coupled with the array of process development, manufacturing expertise and tools that Lonza brings, will provide a smooth route to commercial approval. By leveraging the Cocoon Platform’s unique capabilities, this collaboration aims to illustrate the promise and feasibility of manufacturing autologous immunotherapies in a decentralized model, into approved routine use."

CAR T-cell therapy is a revolutionary technology in which the patient’s own immune cells are reprogrammed so they can recognise and destroy cancer cells. This has proven to be a powerful therapy for refractory blood cancers, but has not yet been very effective for the treatment of solid tumours.

Leucid’s LEU-011 programme is a NKG2D-targeted CAR T-cell therapy in pre-clinical development for the treatment of solid tumours and haematological malignancies. The NKG2D receptor is an activating immune receptor that triggers cell death upon recognition of human NKG2D ligands expressed on transformed, infected or damaged cells. LEU-011 has potential for the treatment of multiple cancer types as NKG2D ligands are expressed on more than 80% of human tumour cells.

On December 2, 2021 Integra Therapeutics, a biotechnology company that is creating next-generation gene writing tools to boost the efficiency and safety of advanced therapies, reported the company has completed its first round of funding for €4.5 million with Advent France Biotechnology (France), Invivo Capital (Spain) and Takeda Ventures (USA) (Press release, Integra Therapeutics, DEC 2, 2021, View Source [SID1234654526]).

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Integra Tx was founded in late 2020 as a spin-off of Pompeu Fabra University (UPF) based on technology developed in the Translational Synthetic Biology Lab led by Dr Marc Güell (EMBO Young Investigator, National Research Award for Young Talent and co-founder of two biotech firms).

The great potential of this novel gene writing technology lies in the fact that it resolves some of the main technical limitations of gene therapy: it can be used to paste DNA sequences of any size gene with high precision. Advanced therapies are one of the pillars of the medicine of the future and are aimed at both preventing and treating genetic and oncological diseases that, for now, are incurable.

The funds raised in this round will allow Integra Tx to complete the prototype of the new gene writing technology platform, carry out preclinical validation using in vivo and ex vivo models, and manage its patent portfolio in 2022 and 2023. After that, the company plans to open a Series A round to seek regulatory approval and carry out clinical trials with patients.

"We’re very proud to be transferring our scientific knowledge and technological skills in gene editing from the lab to society. We thank all our investors for their commitment to Integra Tx and to making advanced therapies safer and more effective, and getting them to patients that urgently need them," says Dr Avencia Sánchez-Mejías, co-founder and CEO of Integra Tx. Sánchez-Mejías joined the UPF Translational Synthetic Biology Lab in 2018 after doing research at the Institute of Biomedicine of Seville, the National University of Singapore and the University of Miami Miller School of Medicine.

Dr Marc Güell, co-founder and CSO of Integra Tx, explains, "the Integra Tx technology platform is very promising because it is an evolution of the CRISPR-Cas techniques. We’ve found a way to merge them with transposase and integrase proteins that have a great capacity for gene transfer and to not depend on viral vectors for transporting the components into the cell, which is a step forward in making these therapies safer." Plus, it has applications both in vivo (directly in patients) and ex vivo (outside of patients).

Matthieu Coutet, Managing Partner at Advent France Biotechnology, adds: "We decided to invest in Integra Tx because we believe in its seasoned scientific and management team. Its founders have shown a strong passion and ambition to move Integra Tx’s technology forward, combined with a proven expertise in gene editing and advanced therapies."

Dr Luis Pareras, Managing Partner at Invivo Capital, says: "We’re thrilled with the possibilities Integra Tx’s plataform can develop to solve the problem of cargo size in gene therapy. We’re also very pleased with the international syndicate supporting this seed round, in yet another example of technology transfer opportunity and the competitiveness of the biotech ecosystem in Spain."

Miles Gerson, Takeda Ventures Partner and Senior Investment Director, says: "Takeda Ventures is very excited to support Integra Tx and their next generation gene writing platform with many potential applications to benefit patients."