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

Percival Scientific’s incubation chambers create the ideal environment for superior butterfly emergence to populate the Christina Reiman Butterfly Wing at Reiman Gardens, Iowa State University’s public garden facility in Ames, Iowa.


Other News
INDUSTRIAL IMPACT

Empatica receives new FDA clearance for its Health Monitoring Platform and announces Series B financing

Empatica | November 23, 2022

Empatica, a digital health and AI company developing medical-grade wearables and algorithms for health monitoring and diagnostics, today announced the clearance of its Empatica Health Monitoring Platform by the U.S. Food and Drug Administration. The Empatica Health Monitoring Platform is a full-stack remote health monitoring and data collection solution for research and healthcare professionals, built on data collected by Empatica's medical-grade, CE-certified EmbracePlus wearable. In addition, the Platform includes Empatica's proprietary Care software suite, secure cloud infrastructure, and clinically validated digital biomarkers. Empatica's Platform has been cleared for continuous data collection to monitor blood oxygen saturation during rest, peripheral skin temperature, activity associated with movement during sleep, and Electrodermal Activity. Each of Empatica's digital biomarkers is based on trained algorithms that analyze sensor data in one-minute intervals, and have been rigorously validated in clinical studies conducted with diverse groups of participants. Platform users can also access raw data collected by EmbracePlus' five sensors, and research-grade digital biomarkers such as Pulse Rate, Pulse Rate Variability, and Respiratory Rate. The Empatica Health Monitoring Platform is being used globally by major pharmaceutical companies to continuously gather and analyze physiological data for clinical trials evaluating the impact of novel therapeutics, with Empatica collaborating to develop digital biomarkers for use as endpoints. Researchers also have the possibility to develop their own digital biomarkers, which they can implement in their digital health applications or infrastructure using the Platform's Software Development Kit. "This clearance represents a significant step forward for our scientific community. Patients, healthcare providers, and researchers deserve digital health products that are accurate, validated in diverse populations, and intuitive to use. We are proud to have built a solution that accomplishes these goals, offering a high-quality and reliable digital health tool to scientists working to improve patient outcomes through research and clinical care." Dr. Marisa Cruz, Chief Medical Officer of Empatica Today Empatica also announced the recent closing of its Series B financing, led by Sanofi Ventures and RA Capital Management, and participation by Black Opal Ventures. The investment will enable Empatica to expand its suite of digital biomarkers, for use in patient care and as digital endpoints in clinical trials. "We are excited to team up with Empatica, their investors and partners on this journey," said Cris De Luca, Partner at Sanofi Ventures and newly-appointed Empatica board member. "By gaining higher resolution into disease symptomology through novel digital measures and digital biomarkers in clinical and real-world settings, Empatica is unlocking the possibilities of early disease detection, enhanced treatment decisions, and improving quality of life for patients around the world". Empatica Empatica Inc is a pioneer in continuous, unobtrusive remote health monitoring driven by AI. Empatica's platform and technology are used by thousands of institutional partners for research purposes, in studies examining stress, sleep, epilepsy, migraine, depression, addiction, and other conditions. Its flagship medical wearable, EmbracePlus, has been developed with key partners including HHS, USAMRDC, and the NASA-funded TRISH.

Read More

CELL AND GENE THERAPY

Orangewood Partners Makes Strategic Investment in Barrington James

Orangewood and Barrington James | October 06, 2022

Orangewood Partners a long-term focused private investment firm, announced an investment in Barrington James, a leading global recruitment firm that primarily serves the pharmaceutical, biotechnology and medical device sectors. Terms of the transaction were not disclosed. In partnering with Orangewood, Barrington James intends to enhance its global recruiting solutions, expand its client-service capabilities and support its ongoing commitment to developing and utilizing cutting-edge technology and software leading to greater access for its clients of exceptional talent on a permanent or temporary basis. Barrington James’ Founder and Chief Executive Officer, Dan Barrington, will continue to lead the business alongside the existing leadership team, who will maintain a significant investment in the company. Founded in 2002, Barrington James provides life sciences companies with recruitment solutions that combine deep industry expertise with innovative recruitment technology to make talent identification and attraction easier. The company’s specialist areas range from AI, biometrics, and clinical development to finance, medical affairs, commercial, quality assurance and computational sciences, and identifies placement opportunities ranging from contract and interim positions through to board and C-suite-level placements. Headquartered outside of London, Barrington James operates twelve offices in core life sciences clusters globally including in New York, Tokyo, Singapore, Raleigh and Greenville supporting placements across Europe, North and South America and Asia. Neil Goldfarb, Managing Partner of Orangewood, stated, “All of the whole of the life science and biotech sectors are poised for significant long-term growth, and competition for top talent in virtually every position is continuing to intensify. Barrington James deep focus on life sciences uniquely positions it to provide outstanding and highly bespoke service to its clients and capitalize further on this attractive opportunity. To that end, we are thrilled to bring Orangewood’s deep expertise in partnering with founders and track record of building healthcare-focused businesses to support Dan and his talented team. We look forward to working together as Barrington James continues to innovate and deepen its role as one of the premier life sciences focused recruiting firms.” “This is an exciting day for Barrington James, our employees and clients. Orangewood is a world-class investment firm with proven capabilities in supporting management teams and driving long-term value from its portfolio companies. We believe this partnership will enable us to continue to assist our top-class clients with their growth by providing exceptional talent to their teams, big or small. Our partnership and growth will allow us to deliver niche recruitment for new biotech’s and volume recruitment for leading pharmaceutical companies with speed and a standard of services that is second to none, on a temporary or permanent basis. I am grateful for the passion and dedication of our talented team around the world who recognize the important role that we play to support our clients whose work has never been more vital.” Dan Barrington, CEO of Barrington James K&L Gates LLP served as legal advisor to Orangewood. Fairmount Partners served as exclusive financial advisor to Barrington James and Keystone Law served as its legal advisor. Brightwood Capital Advisors, LLC acted as sole lead arranger and administrative agent for the transaction. ABOUT ORANGEWOOD Founded in 2015, Orangewood Partners is a New York-based private investment firm with a long-term approach. Orangewood focuses on growth-oriented private transactions in partnership with founder-led businesses, management teams and entrepreneurs. The firm’s senior investment team and deep bench of active operating advisors has decades of experience across a range of sectors, primarily healthcare, consumer, multi-unit and related industries. Orangewood works to build companies into strategic assets in their industries to create long-term value for investors, companies, and communities. ABOUT BARRINGTON JAMES Barrington James is an industry leading, global recruitment business that primarily serves the Pharmaceutical, Biotechnology and Medical Device sectors. With offices all over the world and hundreds of specialists dedicated to sourcing exceptional talent, we have the capacity and capabilities to deliver world-class recruitment solutions to life science companies of all sizes. We combine our reach of more than 6 million industry professionals worldwide and investment in AI platforms with expert consultants specializing in market niches to deliver a personalized approach to global recruitment for life science.

Read More

INDUSTRIAL IMPACT

Agilent Attains ‘Angel’ Level Sponsorship with My Green Lab

Agilent Technologies | December 01, 2022

cevel sponsor of My Green Lab, a nonprofit organization dedicated to building a global culture of sustainability in science. ‘Angel’ level sponsorship provides Agilent access to even more strategic counsel from My Green Lab in support of its sustainability activities and expertise that can be shared with customers. Agilent is also the proud sponsor of the My Green Lab Certification program – considered the gold standard for laboratory sustainability best practices. As a good citizen and role model for customers, Agilent is working to ensure its on-site customer demonstration laboratories globally are Green Lab Certified and have already achieved highest level certification for Waldbronn, Germany, Cheadle, UK, and Santa Clara, US sites. “Sustainability and environmental, social, and governance standards are central to our mission to advance the quality of life, and our sponsorship of My Green Lab helps us to drive this forward. Being an ‘Angel’ level sponsor will further cultivate our knowledge of creating sustainable green labs, which we can share with our customers globally and support them on their sustainability journey.” Darlene Solomon, senior vice president, and chief technology officer at Agilent The Green Lab Certification from My Green Lab is recognized by the United Nations Race to Zero campaign as a key measure of progress for pharmaceutical and medical technology companies toward a zero-carbon future. It indicates that a lab has achieved strong sustainability practices in energy use, water consumption, recycling, and waste production. About Agilent Technologies Agilent Technologies Inc. is a global leader in the life sciences, diagnostics, and applied chemical markets, delivering insight and innovation that advance the quality of life. Agilent’s full range of solutions includes instruments, software, services, and expertise that provide trusted answers to our customers' most challenging questions. The company generated of $6.85 billion in fiscal 2022 and employs 18,000 worldwide.

Read More

INDUSTRIAL IMPACT

AbCellera and Rallybio Announce Strategic Alliance to Discover, Develop, and Commercialize Novel Antibody-Based Therapeutics for Rare Diseases

Rallybio | December 02, 2022

AbCellera and Rallybio Corporation announced that they have entered into a strategic alliance to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. This multi-year, multi-target collaboration will combine AbCellera’s antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates and ultimately deliver therapies to patients. “It’s clear that we need to accelerate drug development for rare diseases to address the enormous unmet medical need for these patients. Rallybio’s team has decades of experience in sourcing, identifying, and evaluating therapeutic targets. By bringing together Rallybio’s deep expertise in rare diseases with AbCellera’s integrated technology for delivering clinical leads with unmatched precision and speed, this partnership creates a unique advantage that we believe will deliver new and transformative medicines to patients.” Carl Hansen, Ph.D., founder and CEO of AbCellera “AbCellera’s clinically validated discovery engine will strengthen our efforts in identifying novel rare disease candidates to add to our existing pipeline,” said Martin Mackay, Ph.D., Chief Executive Officer of Rallybio. “We are excited to establish this partnership and look forward to working closely with our colleagues at AbCellera as we build novel medicines together.” Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies. The collaboration will allow Rallybio to add product candidates to its existing pipeline and also provides the option for AbCellera to conduct process development and clinical manufacturing activities. The partnership’s first program will focus on addressing the significant unmet therapeutic needs of patients with rare metabolic diseases. About AbCellera Biologics Inc. AbCellera is a technology company that searches, decodes, and analyzes natural immune systems to find antibodies that its partners can develop into drugs to prevent and treat disease. AbCellera partners with drug developers of all sizes, from large pharmaceutical to small biotechnology companies, empowering them to move quickly, reduce cost, and tackle the toughest problems in drug development. About Rallybio Rallybio is a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases. Since its launch in January 2018, Rallybio has built a portfolio of promising product candidates, which are now in development to address rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, and metabolic disorders. The Company’s mission is being advanced by a team of highly experienced biopharma industry leaders with extensive research, development, and rare disease expertise. Rallybio is headquartered in New Haven, Connecticut, with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, Connecticut.

Read More

Spotlight

Percival Scientific’s incubation chambers create the ideal environment for superior butterfly emergence to populate the Christina Reiman Butterfly Wing at Reiman Gardens, Iowa State University’s public garden facility in Ames, Iowa.

Resources