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.

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The Black Death, bubonic plague, killed as many as 200 million people in the 1300’s. It is relatively rare now, but the first case in 26 years has recently been reported in the United States. The affected boy was hospitalized and given antibiotics and is now back at home recovering. Drugs have reduced the death rate from the illness to about eleven percent, down from 65 percent when left untreated.


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INDUSTRIAL IMPACT

Canopy Biosciences Launches Next-Generation ChipCytometry™ Instrument for Spatial Biology with Sub-Cellular Resolution

Canopy Biosciences | March 04, 2022

Canopy Biosciences, a Bruker Company, announced the commercial launch of the CellScapeTM system, the next generation in ChipCytometryTM instrumentation, advancing the cutting-edge for quantitative in situ spatial phenotyping. ChipCytometry delivers single-cell targeted spatial proteomics for complex whole-tissue analysis of the tumor microenvironment, as well as deep immune profiling for applications in immunology, neuroscience, and infectious disease. The new benchtop CellScape system builds on the existing core strengths of the original ChipCytometry instrument, the ZellScannerONE™, which has enabled spatial biology research with sub-cellular resolution and a large field of view suitable for whole slide pathology imaging, on both tissue samples and cell suspensions since 2016. The CellScape system , with its multiplexed fluidics integration, adds complete walk-away automation, improved optical performance, sub-cellular resolution and massively increased throughput for whole slide imaging of millions of cells, while maintaining the key features of the core ChipCytometry technology, including high-plex phenotyping with single-cell resolution, very high dynamic range imaging for detection and quantification of both high- and low-expressing targets, and compatibility with standard commercially available fluorescently labelled antibodies, requiring no proprietary antibody conjugation. This powerful combination of features will significantly improve researchers’ workflow, accelerate spatial biology discovery, and drive broader adoption of high-plex spatial omics for translational and clinical applications. “The ChipCytometry platform has been a great tool for us in developing high content staining approaches for in-depth spatial immune profiling of human tissue samples. The improvements to throughput and automation of the CellScape system gives the opportunity to scale up studies and explore larger cohorts – and accelerate studies aimed at improving patient care.” Paul Klenerman, Ph.D., Professor of Gastroenterology at The University of Oxford “Following the acquisition of Canopy Biosciences by Bruker in 2020, we were able to leverage Bruker’s advanced fluorescence microscopy expertise to design the CellScape instrument as an integrated, multiplexed imaging system that is highly optimized for the application of high-throughput quantitative spatial omics with sub-cellular resolution,” added Thomas Campbell, Ph.D., Product Manager at Canopy Biosciences. “The improved optical performance of the system builds on the high resolution and high dynamic range that was already best-in-class with our ChipCytometry technology.” About the CellScape Instrument Through enhanced optics and automation, the throughput of CellScape is markedly improved compared to the previous generation of ChipCytometry instrumentation. Combined with multi-sample automated processing, which is available standard with every CellScape, the platform will have among the highest throughput of any highly multiplexed spatial proteomics system available today. CellScape will also be available with an optional FalconFastTM configuration, which provides an even greater increase in throughput, bringing into reach large-scale clinical studies that have previously not been possible at a high plex. About Canopy Biosciences, a Bruker Company Canopy Biosciences was formed in 2016 and rapidly built a comprehensive portfolio of products and services for spatial biology, multi-omics, and bioprocessing. Canopy offers its ChipCytometry technology for precise spatial multiplexing in cells and tissue samples, along with other technologies of ultrasensitive DNA sequencing (RareSeq), RNA-Seq, and gene expression analysis for services. Canopy Biosciences is headquartered in St. Louis, Missouri, with a CLIA site in California, and a site in Germany to serve researchers at universities, research institutions, and biotechnology and pharmaceutical companies worldwide. Canopy was acquired by Bruker Corporation in 2020.

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MEDICAL

Light Bio Partners with Ginkgo Bioworks to Optimize the Brightness of Glowing Plants through Bioluminescence Engineering

Light Bio | April 18, 2022

Light Bio, Inc., a company that is creating bioluminescent plants for home and garden, and Ginkgo Bioworks (NYSE: DNA), the leading horizontal platform for cell programming, today announced a collaboration to leverage Ginkgo's plant cell assays for bioluminescence engineering. Through the agreement, Ginkgo aims to help improve the luminescent output and efficiency of the enzymes within Light Bio's glowing ornamental plants. The scientists at Light Bio have discovered a new type of bioluminescence mechanism and are using synthetic biology to engineer plants to efficiently emit light. The original light emission pathway Light Bio uses in its glowing plants comes from bioluminescent mushrooms, and the company is now focused on expanding its engineering capabilities to support the creation of glowing plants. We are excited to collaborate with Ginkgo toward additional multifold output and efficiency improvements powered by Ginkgo's plant cell engineering capabilities across both individual enzyme and metabolic pathway improvements, Light Bio has already achieved strong light emission results on our glowing plant luminescence to date." Keith Wood, CEO, Light Bio. Nothing captures the wonder of biology quite like seeing glowing plants in person, Our platform enables developers to imagine with biology and to design all types of cells, from bacteria and yeast to mammalian and plant cells. We're so excited to bring this unique application onto our platform and to help Light Bio bring the magic of glowing plants to people." Jason Kelly, CEO, Ginkgo Bioworks. About Light Bio Light Bio creates novel biodesigns in living light. Through consumer-led applications of synthetic biology, Light Bio is opening new markets with products that have never existed before. Led by a team with deep experience in the development and commercialization of bioluminescence products, the company is sustained by its proprietary technologies backed by a strong patent portfolio. About Ginkgo Bioworks Ginkgo is building a platform to enable customers to program cells as easily as we can program computers. The company's platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo has also actively supported a number of COVID-19 response efforts, including K-12 pooled testing, vaccine manufacturing optimization and therapeutics discovery.

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CELL AND GENE THERAPY

Bicycle Therapeutics Announces Continued Clinical Progress and Updates to Management Team

Bicycle Therapeutics | January 06, 2022

Bicycle Therapeutics plc, a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide technology, announced continued progress in its ongoing Phase I/II clinical trials of BT8009, BT5528 and BT7480. “Last quarter, we reported interim clinical results from ongoing trials of two of our Bicycle toxin conjugates, BT8009 and BT5528, demonstrating preliminary anti-tumor activity in two tumor types, and announced the first patient dosed in the Phase I clinical trial of BT7480, our first tumor-targeted immune cell agonist to enter the clinic. In our trial of BT8009, we are pleased to confirm the preliminary activity and see that these patients remain on trial. We look forward to presenting interim BT8009 Phase I results at a medical meeting and initiating the BT5528 expansion cohorts this year. We are also pleased with our progress advancing BT7480 in the ongoing Phase I clinical trial and look forward to sharing additional details regarding the potential for Bicycles beyond our toxin conjugates as we strive to become a leader in the development of targeted oncology therapeutics.” Kevin Lee, Ph.D., Chief Executive Officer Bicycle Toxin Conjugates® (BTCs) BT8009 and BT5528 In the ongoing Phase I portion of the Phase I/II clinical trial of BT8009, a second-generation BTC™ targeting Nectin-4, four out of 11 patients were previously reported to have a partial response under Response Evaluation Criteria in Solid Tumors version 1.1, including one out of four (25%) in the 2.5mg/m2 dose and three out of seven (43%) at the 5.0 mg/m2 cohorts. All four patients previously reported as responders have since received at least one subsequent scan, and all have been confirmed as ongoing RECIST 1.1 responses. One patient in the 5.0 mg/m2 cohort, who previously was reported to have a partial response with an 89% tumor reduction, has now received two subsequent scans, which each showed that total tumor volume has been reduced by 100%, constituting a confirmed complete response. All four patients previously reported to have a RECIST 1.1 clinical response remain on therapy. The tolerability profile of the 2.5mg/m2 and 5.0 mg/m2 cohorts remains consistent with that previously reported. Dose escalation in the BT8009 Phase I trial remains ongoing, with patients currently being dosed at 7.5mg/m2 weekly or every-other-week. Bicycle intends to present interim Phase I results from the ongoing clinical trial at a medical meeting this year. The Phase I/II trial of BT5528, Bicycle’s second-generation BTC targeting EphA2, is also ongoing, with plans remaining on track to initiate the expansion cohorts this year, with an expected recommended Phase II dose of 6.5mg/m2 every-other-week. Bicycle tumor-targeted immune cell agonist™ (Bicycle TICA™) BT7480 Bicycle also initiated a Phase I clinical trial of BT7480, a novel, fully synthetic Bicycle TICA targeting Nectin-4 and agonizing CD137, in the fourth quarter of 2021, and dose escalation in that trial remains ongoing. BT7480 and other Bicycle TICAs, including a novel NK-cell-engaging molecule, were the subjects of four posters at SITC in November 2021. Management Team Updates Bicycle is also announcing the expansion of and transition in its management team. Michael Skynner, Ph.D., the company’s Chief Operating Officer has been appointed to the newly created position of Chief Technology Officer, effective January 3, 2022, to focus on leading and overseeing the growth of Bicycle’s proprietary phage display discovery platform in oncology, as well as on creating innovative opportunities for the platform outside of oncology. Dr. Skynner joined the company in January 2016 as Vice President, Operations and Discovery and had served as COO since March 2018. Alistair Milnes, who has served as the company’s Vice President, Human Resources and Communications since January 2021, has assumed the COO role. Mr. Milnes previously led human resources and communications at multinational energy and mineral companies. Both Dr. Skynner and Mr. Milnes are based in the United Kingdom. “We are delighted to announce our recent management team appointments, with Mike Skynner becoming our new CTO and Alistair Milnes moving to the COO role. Mike has been an invaluable contributor to Bicycle’s success to date and has led our platform discovery efforts since joining in early 2016. Following recent promising clinical progress, we believe it is time to focus on accelerating the growth of our proprietary oncology pipeline and on identifying innovative ways to potentially capitalize on our unique technology beyond our current therapeutic focus. I am confident that Mike can help us achieve these objectives.” Dr. Lee added, “I am similarly enthusiastic about Alistair’s appointment as COO and believe his operational experience and track record of successfully identifying, recruiting, and retaining key talent at large, multinational companies will be instrumental in helping guide Bicycle through our next phase of growth.” 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 Lexington, MA.

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INDUSTRIAL IMPACT

Aanika Biosciences First Biotech Company to Receive Bermuda Monetary Authority’s Approval for Reinsurance Innovation Hub

Aanika Biosciences, | February 25, 2022

Aanika Biosciences, an emerging biotech company using microbial based technologies to protect the safety and security of our food system, announced today it has received acceptance into the Bermuda Monetary Authority’s (BMA) Innovation Hub. The program will allow reinsurers to explore how the application of Aanika’s traceability and prevention technology can help reduce the magnitude and size of claims while lowering premiums and expanding protection for the food and agriculture industry. Reinsurance is an almost $500 billion global industry that allows insurance carriers to offload portfolio risks to reinsurers and hedge funds. Aanika estimates its technology could help in the underwriting of nearly $100 billion in premiums linked to contamination, recalls, crop insurance as well as non-safety related claims linked to ESG practices and general product liability. Bermuda is one of the largest hubs for the reinsurance market in the world and the center of innovation in the space. Prior to co-founding Aanika, CEO Vishaal Bhuyan spent more than a decade in finance focused on investing, managing and co-authoring multiple books on insurance-linked derivatives related to human longevity, overfishing and water scarcity. “Climate change will wreak havoc on our food system – potentially accelerating contamination outbreaks, diseases and natural disasters that are damaging and outright destroying crops around the world. ,” says Bhuyan. “ The insurance industry is not yet equipped to handle the volatility of risk and magnitude of loss - and is operating with a massive blind spot. In recent years insurers have adopted machine learning, satellite data and blockchain technologies to increase efficiency and more accurately price risks, but this will be the first use of synthetic biology to better prepare and protect themselves and their policyholders.” The proof-of-concept initiative approved by the BMA will enable Aanika to partner with insurance carriers to develop ‘test policies’ for the U.S. food and agricultural market that mandate applying the microbial technology in their supply chain. This improved ability to distinguish identical commodities from each other will increase transparency throughout the claims process beyond what is possible with digital technologies alone. Aanika co-founder and Chief Science Officer Dr. Ellen Jorgensen says this is a unique and unprecedented opportunity to unlock the potential of synthetic biology. “We need stable, scalable, actionable solutions now to build a more resilient global food supply, which means finding inventive ways of looking at the problem,” says Dr. Jorgensen. “The Aanika science team has developed a way to adapt nature to meet this challenge, and it will be exciting to see how leveraging it in an unconventional way like reinsurance can have immediate as well as long-term benefits.” By tapping into the reinsurance industry, Aanika will be able to commercialize biological innovations in ways that have never been done before. Aanika’s goal is to capture a fee on every policy that is essentially microscopically tethered to the product while using its growing pipeline of “secondary functions” to further enhance the stability and shelf life of said product – reducing the probability and magnitude of a claim. About Aanika Biosciences Aanika Biosciences was co-founded in 2018 by Vishaal Bhuyan after he personally experienced the consequences of ordering fresh, ethically sourced seeds and receiving stale, contaminated products instead. He made it his mission to create a safer food supply by finding a way to track, trace and authenticate products. Aanika’s customized microbial-based tags help companies gain valuable insights about their supply chains, help their customers make better consumption choices, and create a more sustainable world. About Bermuda Monetary Authority The Bermuda Monetary Authority (Authority or BMA) regulates Bermuda’s financial services sector. The Authority was established by statute in 1969. Its role has evolved over the years to meet changing needs in the financial services sector. Today it supervises, regulates and inspects financial institutions operating in the jurisdiction. It also issues Bermuda’s national currency, manages exchange control transactions, assists other authorities with the detection and prevention of financial crime, and advises Government on banking and other financial and monetary matters.

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Spotlight

The Black Death, bubonic plague, killed as many as 200 million people in the 1300’s. It is relatively rare now, but the first case in 26 years has recently been reported in the United States. The affected boy was hospitalized and given antibiotics and is now back at home recovering. Drugs have reduced the death rate from the illness to about eleven percent, down from 65 percent when left untreated.

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