PR Newswire | October 05, 2023
Capsida Biotherapeutics Inc. and Kate Therapeutics announced a strategic partnership to leverage Capsida's expertise and adeno-associated virus (AAV) manufacturing capabilities to enable KateTx's initial internal portfolio of muscle and heart disease programs.
Under the terms of the agreement, Capsida will provide GMP manufacturing of KateTx's gene therapies as the medicines advance through preclinical and clinical development. In return, Capsida will receive undisclosed funding for the term of the agreement.
KateTx's publicly disclosed internal programs include myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy, which are two of the leading causes of adult-onset muscular dystrophy.
"Capsida possesses end-to-end expertise – which includes AAV capsid engineering, preclinical, clinical, and state-of-the-art manufacturing," said Peter Anastasiou, chief executive officer (CEO) of Capsida Biotherapeutics. "We are thrilled to be able to leverage our established capabilities in AAV manufacturing and support a peer company to reach patients in a complementary space, while continuing to advance our own internal and partnered pipeline in CNS and ophthalmology."
"KateTx is leading the way in developing capsid and cargo combinations to address muscle and heart diseases in ways that have not been previously possible," said Kevin Forrest, Ph.D., president and CEO of Kate Therapeutics. "Capsida's exceptional AAV manufacturing capabilities allow us to build our pipeline, de-risk our programs, and advance them into the clinic in the most rapid manner possible, so we can bring much-needed therapies to patients quickly."
"This collaboration between Capsida and KateTx leverages complementary expertise and overlapping needs across both innovative companies to advance programs in a capital-efficient manner. Capsida has established first-in-class engineered AAV manufacturing capabilities applicable across broad therapeutic areas, and this partnership with KateTx aims to bring these next-generation gene therapies to patients sooner than with traditional approaches," said Beth Seidenberg, M.D., co-founding managing director at Westlake Village BioPartners, member of the board of Capsida, and chair of the board at KateTx.
About Capsida Biotherapeutics
Capsida Biotherapeutics is an integrated gene therapy company developing treatments for rare and common diseases across all ages. Capsida's new class of engineered adeno associated virus (AAV)-based delivery technologies enables a broad range of therapeutic modalities, including DNA, RNA, gene editors, vectorized antibodies and other genetic cargoes, to be systemically delivered to specific organ systems while simultaneously limiting exposure to non-targeted organs. Capsida's proprietary capsid engineering and broad IP estate allows for identification and development of capsids with tropism for any tissue or cell type. The company's initial focus has been delivering therapeutic cargoes intravenously to the central nervous system (CNS) with high transduction levels to the desired tissues and cells. The company is developing a pipeline of both wholly owned programs in CNS and partnered programs with AbbVie (CNS and broad range of ophthalmic disorders), Lilly (CNS), and CRISPR Therapeutics (CNS). Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech.
About Kate Therapeutics
Kate Therapeutics (KateTx) is a patient-focused biotechnology company developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined muscle and heart diseases. The company is applying novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation. These breakthroughs have the potential to improve the efficacy and safety of gene therapies and enable the pursuit of a broader set of targets that are otherwise difficult to drug with current technologies.
globenewswire | September 12, 2023
Nurix Therapeutics, Inc. a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with hematologic malignancies and solid tumors, today announced that it has entered into a multi-year, multi-target strategic collaboration agreement with Seagen Inc. to advance a new class of medicines called Degrader-Antibody Conjugates (DACs) for use in cancer. The collaboration between the two companies will focus on an innovative approach to combine two powerful technologies to target cancer—antibody-drug conjugation (ADC) and targeted protein degradation (TPD)—with the goal of creating drugs with new mechanisms of action as well as improved specificity and anti-cancer activity.
“By combining the tissue and tumor specificity of antibodies with highly potent and catalytic targeted degradation of cancer driver proteins, we believe that DACs may represent a next generation of cancer medicine for a wide range of solid tumors and hematologic malignancies,” said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. “With Seagen, our strategic goal is to advance ADC technology to the next level to provide patients with new DAC drugs that deliver greater anti-tumor efficacy and safety compared to currently available agents.”
“The targeted protein degrader modality provides unique advantages over payloads currently employed across the ADC field,” said Gwenn M. Hansen, Ph.D., chief scientific officer of Nurix. “This collaboration is a new application of our DELigase technology, and we are delighted to work with Seagen, a pioneer in the development and commercialization of ADC therapeutics, to create a new generation of drugs to fight cancer.”
Under the terms of the agreement, Nurix will receive an upfront payment of $60 million and has the potential to receive up to approximately $3.4 billion in research, development, regulatory and commercial milestone payments across multiple programs. In addition, Nurix will be eligible for mid-single to low double digit tiered royalties on future sales, and Nurix retains an option for U.S. profit sharing and co-promotion on two products arising from the collaboration. As part of the multi-year collaboration, Nurix will use its proprietary DELigase platform to develop a suite of targeted protein degraders against multiple targets nominated by Seagen that are suitable for antibody conjugation. Seagen will be responsible for conjugating these degraders to antibodies to make DACs and advancing these DAC drug candidates through preclinical and clinical development and commercialization. Given the potential to conjugate multiple antibodies to unique degraders, several DAC drugs may be developed and commercialized within this collaboration.
With the receipt of the $60 million upfront payment, Nurix expects that its existing cash, cash equivalents and marketable securities, excluding any future potential milestones from collaborations, will be sufficient to fund its operating activities into the second quarter of 2025.
About Nurix Therapeutics, Inc.
Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative medicines based on the modulation of cellular protein levels as a novel treatment approach for cancer and other challenging diseases. Leveraging extensive expertise in E3 ligases together with proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform, to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin-proteasome system to selectively decrease or increase cellular protein levels. Nurix’s wholly owned, clinical stage pipeline includes targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates activation of multiple immune cell types including T cell and NK cells. Nurix is headquartered in San Francisco, California.
businesswire | October 19, 2023
Global genomics solutions provider Integrated DNA Technologies (IDT), an operating company in the Life Sciences segment of Danaher Corporation announced the completion of its new Therapeutic Oligonucleotide Manufacturing facility in Coralville, Iowa. The milestone marks a significant achievement in the company’s 35-year-history—its entrance into the therapeutics space—and enables IDT to manufacture products for research use through to current good manufacturing practice (cGMP) grade cell and gene therapy reagents to provide researchers with a single partner that can help them rapidly transition from the lab to therapeutic development.
The 41,000-square-foot-site will produce cGMP cell and gene therapy reagents, including single guide RNAs (sgRNAs) and donor oligos for homology-directed repair (HDR) with additional offerings to follow. These new capabilities and offerings will be supported with comprehensive documentation and testing, a support team, and regulatory guidance to help accelerate researchers’ path to the clinic.
“An increasing number of customers are seeking out IDT as a trusted partner for their CRISPR genome editing needs, and are asking us to be the provider that can help them bridge the gap from lab to clinic,” said Demaris Mills, president, IDT. “Now, with our new cGMP manufacturing facility, IDT can provide a complete CRISPR workflow—from design to analysis—that supports cell and gene therapy developers in all stages of therapeutic development, with the same support and expertise they have come to know from IDT. These new manufacturing capabilities, which have been informed by our decades of oligonucleotide synthesis manufacturing expertise, evolves IDT’s business model from Research Use Only to cGMP, and enables us to help more people.”
Danaher Vice President and Group Executive Chris Riley added, “The future of genomic medicine hinges on the industrialization of biology to make life-saving therapies more accessible to people. As a pioneer in genome editing, IDT’s continued investments will enable customers to rapidly move from clinical development to commercialization. This new facility is another significant milestone in IDT’s innovation journey, one we envision will have a profound impact in genomic medicine for years to come.”
The Therapeutic Oligo Manufacturing facility features ISO 8 cleanrooms, purification suites, chemical distribution and storage rooms, quality control labs, analytical lab space for product testing, ancillary and office spaces and shell space for future expansion. Manufacturing is performed in accordance with ICH Q7 cGMP standards for consistent and reliable quality. The controlled-access building features environmental controls for temperature, humidity and air pressure throughout, supported by an environmental program and continuous monitoring system. The addition of this new facility expands IDT’s global manufacturing footprint and enables the company to provide a range of manufacturing capabilities, including Research Use Only (RUO), large scale RUO, Engineering Run and cGMP (ICH Q7).
A Decade of CRISPR Innovation
Since 2015, IDT has continued to accelerate the pace of genomics with its complete portfolio of Alt-R™ CRISPR genome editing solutions. Its portfolio includes several first-to-market research products as well as solutions to meet researchers’ complete workflow—from design to analysis—to enable greater quality, simplicity, and cost efficiency. Last year, IDT launched the Alt-R™ HDR Donor Blocks, an improved solution for increasing homology-directed repair (HDR) rates in large fragment knock-in experiments, and expanded access to its Alt-R™ Custom CRISPR gRNA Libraries for drug discovery. In 2021, IDT also unveiled its rhAmpSeq™ CRISPR Analysis System, an end-to-end solution for characterizing and quantifying the full array of on- and off-target genome editing events in CRISPR research products.
IDT’s research and development teams have also been at the forefront of CRISPR innovation, with achievements that include the development of multiple proprietary CRISPR reagents such as IDT’s Alt-R™ S.p. HiFi Cas9 Nuclease and the Alt-R L.b. and A.s. Cas12a (Cpf1) Ultra enzymes. Notably, their groundbreaking research and collaborations with scientists around the globe has been published in more than 200 scientific journals as peer-reviewed articles, and is helping to shape the future of genomics and biotechnology.
IDT collaborated with M.A. Mortenson Company, a team led by OPN Architects and Barr Engineering Co. on the facility design and construction of its cGMP facility.
For more than 35 years, Integrated DNA Technologies, Inc. (IDT) has been empowering genomics laboratories with an oligonucleotide manufacturing process unlike anyone else in the industry, with the most advanced synthesis, modification, purification, and quality control capabilities available. Since its founding in 1987, IDT has progressed from a leading oligo manufacturer to a genomics solutions provider supporting key application areas such as next generation sequencing, CRISPR genome editing, synthetic biology, digital PCR, and RNA interference. IDT manufactures products used by scientists researching many forms of cancer and most inherited and infectious diseases.