BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration & License Agreement

BioMarin | May 03, 2020

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR will receive an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.  The company did not disclose financial terms.  BioMarin management reiterated its 2020 GAAP net income guidance of $20 to $80 million, inclusive of this collaboration. The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM).  Additionally, the companies will collaborate on several of DiNAQOR's other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms.  Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR. "With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer at BioMarin.  "This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies." "We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyophathies," said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin.  "We believe there is tremendous potential in combining our experience in gene therapy research and development with DiNAQOR's in-depth knowledge of genetic heart diseases.

Spotlight

This technical note demonstrates the comprehensive
characterization and confident identification of glucuronide
metabolites from hepatocyte incubations of midazolam. An
orthogonal fragmentation mechanism was applied to generate diagnostic fragment ions for confident identification of glucuronide metabolites

Spotlight

This technical note demonstrates the comprehensive
characterization and confident identification of glucuronide
metabolites from hepatocyte incubations of midazolam. An
orthogonal fragmentation mechanism was applied to generate diagnostic fragment ions for confident identification of glucuronide metabolites

Related News

MEDTECH, INDUSTRIAL IMPACT

argenx and Genmab Enter Partnership to Advance Antibody Therapies in Immunology and Oncology

Globenewswire | April 18, 2023

argenx SE and Genmab A/S announced that argenx and Genmab have entered into a collaboration agreement to jointly discover, develop and commercialize novel therapeutic antibodies with applications in immunology, as well as in oncology therapeutic areas. The multiyear collaboration will leverage the antibody engineering expertise and knowledge of disease biology of both companies to accelerate the identification and development of novel antibody therapeutic candidates with a goal to address unmet patient needs in immunology and cancer. “Our core mission is to innovate on behalf of patients by translating immunology breakthroughs into novel pipeline candidates. We do this through a model of co-creation which has led to eight molecules demonstrating human proof-of-concept in our pipeline,” said Tim Van Hauwermeiren, Chief Executive Officer, argenx. “Through our collaboration with Genmab, we are bringing together our combined antibody discovery, development and commercialization expertise to unlock insights on the disease pathways that we will address. This allows us to broaden our capabilities and maximize the opportunity to generate novel therapeutic antibodies within autoimmunity or cancer.” “Genmab is entering the therapeutic area of immunology and inflammation as a steppingstone to achieving its vision that by 2030, our knock-your-socks-off “KYSO” antibody medicines will be transforming the lives of people with cancer and other serious diseases,” said Jan van de Winkel, Ph.D., Chief Executive Officer, Genmab. “By partnering with argenx, we will be able to combine our deep knowledge of the biology and therapeutic power of antibodies and have an opportunity to address patients’ needs in oncology as well as in immunology and inflammation.” About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first-and-only approved neonatal Fc receptor (FcRn) blocker in the U.S., the EU and UK, and Japan. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.

Read More

INDUSTRIAL IMPACT, MEDICAL

Bicycle Therapeutics Announces a Strategic Collaboration with Novartis to Discover, Develop and Commercialize Bicycle® Radio-Conjugates

Businesswire | March 29, 2023

Bicycle Therapeutics plc a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle®) technology, today announced that it has entered into a strategic collaboration agreement with Novartis to develop, manufacture and commercialize Bicycle® radio-conjugates (BRCs) for multiple agreed upon oncology targets. “This collaboration builds on the groundbreaking clinical work we have been doing in the toxin conjugate field and provides new and additional validation for this unique technology,” said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. “We look forward to working closely with Novartis to pioneer the discovery and development of potential new cutting-edge radiopharmaceutical cancer treatments based on Bicycles. We believe the properties of Bicycles make them well suited for the development of precision guided radiopharmaceuticals and represents the next leg in the application of our proprietary discovery platform in oncology.” Under the terms of the agreement, Bicycle will utilize its proprietary phage platform to discover Bicycles to be developed into BRCs. Novartis will be responsible for further development, manufacture and commercialization of the BRCs. Novartis will fund all pre-clinical and clinical development and commercialization activities. Bicycle will receive a $50 million upfront payment and is eligible for development and commercial-based milestone payments totaling up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties on Bicycle-based medicines commercialized by Novartis. About Bicycle Therapeutics Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, referred to as Bicycles, for diseases that are underserved by existing therapeutics. Bicycles are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycles attractive candidates for drug development. Bicycle is evaluating BT5528, a second-generation Bicycle Toxin Conjugate targeting EphA2; BT8009, a second-generation BTC targeting Nectin-4, a well-validated tumor antigen; and BT7480, a Bicycle TICA™ targeting Nectin-4 and agonizing CD137, in company-sponsored Phase I/II trials. In addition, BT1718, a BTC that targets MT1-MMP, is being investigated in an ongoing Phase I/IIa clinical trial sponsored by the Cancer Research UK Centre for Drug Development. Bicycle is headquartered in Cambridge, UK, with many key functions and members of its leadership team located in Cambridge, MA.

Read More

INDUSTRY OUTLOOK

TriLink BioTechnologies Introduces Latest mRNA Capping Technology: CleanCap® M6 analog

Globenewswire | May 15, 2023

TriLink BioTechnologies a Maravai LifeSciences company and global provider of life science reagents and services, has launched its newest cap analog, CleanCap® M6 . The latest CleanCap analog is expected to help developers and researchers maximize the impact of their mRNA therapeutics and vaccines while reducing overall manufacturing costs, bringing life-changing medicines to market faster. With the introduction of the CleanCap M6 technology, legacy capping methods like enzymatic and ARCA may be a thing of the past. By combining the modification of 3’OMe on the m7-Guanosine and the additional methyl modification on the +1 Adenosine, the CleanCap M6 analog is expected to improve mRNA potency with the highest protein expression of any CleanCap analog yet. Combined with technology’s single-pot reaction capabilities that shrink manufacturing times and increase mRNA yields with a purity profile of >95%, CleanCap analogs are novel mRNA Cap structures, which may improve the benefits of an mRNA therapeutic or vaccine. “TriLink’s portfolio of CleanCap mRNA capping technology is expected to be the new go-to capping method in the very near future - it is the future,” shared Kate Broderick Ph.D., Chief Innovation Officer at Maravai LifeSciences. “What excites me most about this novel analog is its ability to make mRNA an even more powerful therapeutic than we have seen previously.” The company’s first CleanCap analog launched in 2017 and this capping technology has been incorporated in one of the first commercially approved COVID-19 vaccines. With the introduction of CleanCap M6, TriLink has developed its most robust analog to date. Studies have shown that the innovative product increases mRNA expression by 30% or more versus enzymatic capping methods and is estimated to reduce manufacturing costs by 20-40%. “mRNA is clearly an emerging therapeutic modality where mRNA vaccines and therapeutics are expanding rapidly,” added Drew Burch, EVP of TriLink’s Nucleic Acid Products division. “CleanCap M6, and the entire CleanCap portfolio, joins a strong roster of synthesized nucleic acids, NTPs, and more that we expect to continue to grow.” In addition to the CleanCap mRNA capping portfolio, TriLink has scale-up expertise and unique mRNA, oligonucleotide & plasmid production capabilities for companies focused on therapeutic, vaccine, diagnostic breakthroughs, leaning on its 25-plus years of experience to provide superior CDMO services. Data and customer insights garnered from a market assessment conducted by a third-party consulting firm on behalf of TriLink BioTechnologies in November 2022. About Maravai Maravai is a leading life sciences company providing critical products to enable the development of drug therapies, diagnostics, and novel vaccines. Maravai’s companies are leaders in providing products and services in the fields of nucleic acid synthesis and biologics safety testing to many of the world’s leading biopharmaceutical, vaccine, diagnostics, and cell and gene therapy companies.

Read More