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

KingFisher Ready DNA Ultra 2.0 prefilled plates are ready-to-use reagents using MagMAX technology, which means fewer pipetting steps and faster, more consistent extractions. Simply peel off the cover, add sample, and load plate onto a KingFisher.

Spotlight

KingFisher Ready DNA Ultra 2.0 prefilled plates are ready-to-use reagents using MagMAX technology, which means fewer pipetting steps and faster, more consistent extractions. Simply peel off the cover, add sample, and load plate onto a KingFisher.

Related News

DIAGNOSTICS

GRAIL Announces Strategic Collaboration With AstraZeneca to Develop Companion Diagnostic Tests to Enable the Treatment of Early-Stage Cancer

GRAIL | June 06, 2022

GRAIL, LLC, a healthcare company whose mission is to detect cancer early when it can be cured, today announced a broad strategic collaboration with AstraZeneca (LSE/STO/Nasdaq: AZN) to develop and commercialize companion diagnostic (CDx) assays for use with AstraZeneca’s therapies. The collaboration will initially focus on developing companion diagnostic tests to identify patients with high-risk, early-stage disease, with plans to embark on numerous studies across multiple indications over the next several years. The parties are additionally planning to use GRAIL’s technology to enable recruitment of patients with early-stage cancer for AstraZeneca’s clinical studies. GRAIL has developed a novel approach to detect cancer signals in blood, regardless of whether or not the patient has clinical symptoms. We are excited to embark on this work with AstraZeneca to transform cancer outcomes through a broad, strategic collaboration, Through our collaboration, we hope to provide critical information to improve the identification of patients who may be eligible for clinical trials and change clinical paradigms for the treatment of early-stage cancers." Sir Harpal Kumar, President, Biopharma Business and Europe at GRAIL. Across the projects within this collaboration, GRAIL will use its methylation platform to perform testing for patients enrolled in AstraZeneca’s clinical trials. GRAIL will seek regulatory approval in key markets for the liquid biopsy companion diagnostics. Identifying and treating cancer early is at the heart of this strategic collaboration. Combining GRAIL’s innovative blood-based methylation profiling platform with AstraZeneca’s leadership in Oncology, we hope to accelerate the adoption of circulating tumor DNA across clinical trials and make our cancer medicines available at an earlier stage of disease when there is greater potential to transform patient outcomes, and even cure.” Susan Galbraith, Executive Vice President of Oncology R&D, AstraZeneca. GRAIL is developing its proprietary methylation platform for use in multiple post-diagnostic settings to identify patients at high risk of future cancer recurrence, detect the presence or absence of residual disease, and to inform treatment decisions, including identifying patients with cancer who may be eligible for neoadjuvant or adjuvant therapy. GRAIL’s technology platform is designed to optimize circulating tumor DNA testing to enable detection of early stage disease and minimal residual disease (MRD). About GRAIL GRAIL is a healthcare company whose mission is to detect cancer early, when it can be cured. GRAIL is focused on alleviating the global burden of cancer by developing pioneering technology to detect and identify multiple deadly cancer types early. The company is using the power of next-generation sequencing, population-scale clinical studies, and state-of-the-art computer science and data science to enhance the scientific understanding of cancer biology, and to develop its multi-cancer early detection blood test. GRAIL is headquartered in Menlo Park, CA with locations in Washington, D.C., North Carolina, and the United Kingdom. GRAIL, LLC, is a subsidiary of Illumina, Inc. (NASDAQ:ILMN) currently held separate from Illumina Inc. under the terms of the Interim Measures Order of the European Commission dated 29 October 2021.

Read More

Novartis Acquires Vedere Bio, a Novel Optogenetics AAV Gene Therapy Company

Vedere Bio | October 29, 2020

Vedere Bio, Inc. (Vedere Bio), a stealth-stage company focused on advancing photoreceptor-protein-based optogenetic therapies that are delivered to the retina intravitreally to restore functional vision, announced today that it has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and will be eligible for up to $130 million in milestone payments, for a total of $280 million. Based on technology from the laboratories of Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, and technology directed at enhanced ocular gene therapy delivery arising jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania, Vedere Bio was formed in the Atlas Venture incubator in June 2019. The company was launched with a $21 million Series A financing and began lab operations at LabCentral in Cambridge, MA where it advanced its lead programs from concept to development candidate within one year. Immediately prior to the acquisition, certain earlier-stage vision restoration and vision preservation assets leveraging the company's ocular gene therapy toolbox were spun out into a newly formed entity – Vedere Bio II, Inc.

Read More

DIAGNOSTICS

Avantor® to Collaborate with GeminiBio to Bring Custom Hydrated Solutions and Cell Culture Media to Biopharma Customers

Avantor | June 24, 2022

Avantor, Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries has entered into an agreement with GeminiBio to deliver custom hydrated solutions and cell culture media to the biopharma industry. Custom hydrated solutions and cell culture media are critical components of the bioproduction workflow, especially for emerging modalities such as gene therapy. This collaboration will complement Avantor's proven ability to serve manufacturing platforms with GeminiBio's small-to-medium scale offering, enabling customers to utilize custom cGMP products through the full development lifecycle – including early-stage research, scale-up and commercialization. This new capability along with Avantor's recent acquisitions and site investments, support a growing portfolio of proprietary offerings, demonstrating Avantor's commitment to supporting customer workflows in both upstream and downstream processing for the biopharma industry. With the imperative to meet patient needs quickly, our customers require custom solutions to accelerate their bioproduction goals, Customized media is critical in optimizing cell line productivity, and hydrated solutions offer efficiency to biomanufacturers as they optimize their downstream processes. Our collaboration with GeminiBio will drive increases in product yields, helping bring efficiency and speed to our customers as they scale. Together, we can drive science forward to bring therapies to the patients who need them most." Dr. Ger Brophy, Executive Vice President, Biopharma Production for Avantor. Enabling customers to optimize their upstream and downstream liquid manufacturing workflows by offering custom solutions, built around a strong quality foundation, has been an essential part of GeminiBio's success. Collaborating with Avantor, who shares our focus on quality, will allow us to expand our ability to address customer needs for both small and large volume process liquids." Brian Parker, CEO of GeminiBio. About Avantor Avantor®, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries. We set science in motion to create a better world.

Read More