BUSINESS INSIGHTS

EditForce and Mitsubishi Tanabe Pharma Enter into License Agreement

EditForce, Inc. | July 06, 2022 | Read time : 2 min

EditForce and Mitsubishi
EditForce, Inc. has entered into a License Agreement with Mitsubishi Tanabe Pharma Corporation to research, develop and commercialize potential gene therapy products for a specific target disease in the field of CNS by utilizing EditForce's proprietary PPR protein platform technology.

In this alliance, MTPC and EditForce aim to create potential novel pharmaceuticals for the specific CNS disease by utilizing the drug R&D know-how and global business experience of MTPC and the novel biotechnology of EditForce. MTPC will acquire the exclusive right to conduct the selection of drug candidate molecules, preclinical and clinical development, manufacturing, and commercialization worldwide.

Under the terms of the Agreement, EditForce will receive an upfront payment and milestone payments amounting to over 20 billion yen depending on the development stage and commercialization progress, and royalties based on worldwide sales after the launch.

"I am so delighted to reach the agreement with MTPC, which has an interest in our proprietary PPR protein platform technology. We look forward to working closely with MTPC to develop and deliver breakthrough pharmaceutical products with our technology to patients suffering from diseases."

Takashi Ono, President and CEO of EditForce

About EditForce, Inc.
EditForce, Inc., a Kyushu University-originated venture company developing a unique DNA/RNA editing technology (PPR platform technology (*)), was established in May 2015 by KISCO Ltd. and Prof. Takahiro Nakamura of Kyushu University (former president of EditForce and currently its scientific advisor) and funded by companies and funds that have track records of investing in life sciences and biotechnology. EditForce aims for drug discovery that applies the PPR technology through joint research with universities and private companies.

Pentatricopeptide repeat protein platform technology
PPR is a protein discovered in plants that regulates gene expression by binding to DNA and RNA in a sequence-specific manner. The PPR proteins are also found in humans and yeasts, and they have similar functions. Prof. Takahiro Nakamura and Dr. Yusuke Yagi, CTO of EditForce, have focused on the PPR proteins and elucidated the mechanism that determines sequence specificity, and established a technology for creating various PPR proteins, each of which binds to a specific target DNA or RNA sequence. Furthermore, it is possible to manipulate and modify the target genome and RNA both inside and outside the cell by fusion with effector proteins.

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation the pharma arm of Mitsubishi Chemical Group is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, "Forging the future." MTPC sets the MISSION of "Creating hope for all facing illness." To that end, MTPC is prioritizing work on "precision medicine" to provide drugs with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety, focusing on the disease areas of the central nervous system and immuno-inflammation. In addition, MTPC is working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis.

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Spotlight

DSM’s unique enzyme Tolerase® G helps digest gluten and support your life style. Gluten is a hard to digest protein, since it is rich in the amino acid proline. The human body cannot break down proline-rich proteins such as gluten efficiently and this may be why some people are sensitive to dietary gluten. As gluten is present in many different foods, following a gluten-free diet is difficult. And even with a gluten-free diet, it is almost impossible to fully avoid gluten intake.

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BUSINESS INSIGHTS

Septerna Strengthens Leadership Team to Advance Novel Small Molecule GPCR Medicines

Septerna | July 29, 2022

Septerna, a biotechnology company discovering and advancing novel small molecule medicines targeting G protein-coupled receptors announced the addition of Liz Bhatt, MS, MBA, as Chief Operating Officer, and Ran Xiao as Vice President of Finance and Business Operations to its leadership team. Septerna launched in January 2022 and is growing a pipeline of differentiated products using its GPCR Native Complex™ platform to unlock the vast untapped potential of GPCR drug targets for a wide range of diseases. We are thrilled to welcome Liz and Ran as we continue to build our exceptional team. Together, they bring deep experience in corporate, commercial, and business strategy. They also have significant operational expertise building and supporting both private and public companies,” said Jeffrey Finer, MD, PhD, Chief Executive Officer and Co-founder of Septerna and Venture Partner at Third Rock Ventures. “Since our launch in January, we’ve made rapid progress advancing our platform and early discovery programs, and now expanding our focus to strategic growth and operational excellence is absolutely critical so that we can fully realize the untapped opportunity space for new GPCR medicines.” “Septerna launched with the ambitious plan to expand the GPCR target landscape by developing and industrializing new drug discovery technologies and processes that can convert this target-rich superfamily of proteins into impactful new therapies for patients across a range of therapeutic areas. I’m excited to be working with a team of talented GPCR scientists and drug hunters and look forward to using my experience leading teams and developing corporate and product strategies to build Septerna into a robust company poised for long-term growth.” Ms. Bhatt Liz Bhatt brings more than 25 years of strategy, deal-making and company-building experience across a range of biotech and pharmaceutical companies. Liz was previously Chief Business & Strategy Officer at Applied Molecular Transport where she oversaw corporate development, portfolio and project management, and commercial strategy. Prior to AMT, Liz was Chief Operating Officer at Achaogen where she held various strategic and operational roles. Liz spent over a decade at Gilead Sciences as VP, Corporate Development leading deals across all therapeutic areas and as Senior Director, Commercial Strategy leading long-term global commercial and strategic planning, and supporting the launch of several products. Liz also held management roles at Eli Lilly and Maxygen and started her career in research at Affymax. She currently serves as an independent director on the board of eFFECTOR Therapeutics. Liz earned a BA in chemistry from Pomona College, an MS in biomedical sciences from the University of California San Diego, and an MBA from the Kellogg School of Management at Northwestern University. Ran Xiao has more than 20 years of experience in corporate accounting, finance, and business operations in the biotech industry. Prior to Septerna, Ran was the VP of Finance and Corporate Controller at Ambys Medicines, Inc, where she supported the company launch and managed various general and administrative functions through a rapid growth period. Before joining Ambys, Ran was the Corporate Controller at Corvus Pharmaceuticals, where she played a key role in the company’s initial public offering. Prior to Corvus, Ran held various managerial positions at Intermune, Natus Medical, Celera Genomics and CV Therapeutics. Ran has a BS in accounting from Shanghai University of Finance and Economics and an MBA from the Illinois Institute of Technology. Ran is a Chartered Financial Analyst. About GPCRs G protein-coupled receptors are the largest and most diverse family of cell membrane receptors, and humans have hundreds of different GPCRs, each involved in controlling specific biological functions. GPCRs on the surface of each cell bind a wide range of external signaling molecules from throughout the body, and the GPCR transmits the signal across the cell membrane to drive internal cellular mechanisms. GPCRs have been widely studied as drug targets and are the largest family of proteins targeted by approved drug products. An estimated 700 approved drugs target GPCRs, representing approximately one-third of all currently approved drugs. Despite the pharmacological success of GPCRs as a drug class to date, the large majority of potential therapeutic GPCR targets remain undrugged. About Septerna Septerna, Inc., is a biotechnology company creating broad new drug discovery opportunities across many disease areas for the abundant drug target class of G protein-coupled receptors. The company’s Native Complex™ Platform recapitulates GPCRs with their native structure, function, and dynamics outside of the cellular environment to enable new technologies for industrial-scale drug discovery for the entire GPCR target class for the first time. Septerna has an emerging pipeline of GPCR-targeted small molecule drug discovery programs, along with growth potential to reach many GPCRs that have been undruggable and unexploited to date. Septerna was launched in 2022 by scientific founders who have made groundbreaking GPCR discoveries and by founding investor Third Rock Ventures.

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PHARMA TECH

Nucleai Announces a Partnership with Sirona Dx for AI-Driven Discovery of Novel Spatial Biomarkers in Solid Tumors

Nucleai and Sirona Dx | July 14, 2022

Nucleai, a leader in AI-powered spatial biology transforming precision medicine by unlocking the power of pathology data, and Sirona Dx, a leading provider of multiomic single-cell analytical services, today announced a collaboration to advance the discovery of novel spatial biomarkers in solid tumors. The partnership combines Nucleai’s powerful AI spatial analytics platform with Sirona Dx’s leading multiplex assay development expertise to identify novel spatial signatures in high-plex assays that predict response to therapy and inform treatment decisions. Through this strategic collaboration, Sirona Dx will provide Nucleai and its customers with a CRO partner that will enable the deployment of AI-based diagnostic algorithms to support biopharma clinical trials. Nucleai’s robust computational AI-powered spatial biology algorithm supports various image modalities and cancer indications, offering a highly customizable, deployable solution across the drug development ecosystem. These capabilities earned Nucleai the position of one of the top 10 spatial biology companies in 2022. “Multiplex imaging technologies have improved rapidly over the years; however, identifying actionable insights from all the data collected remains immensely challenging. This powerful synergy between Sirona Dx and Nucleai is very exciting as it will allow us to identify actionable spatial signatures that can predict response to immunotherapy.” Avi Veidman, Co-Founder & Chief Executive Officer of Nucleai Sirona Dx is recognized as the original pioneers of spatial biology, having launched the first spatial-omics CRO service to biopharma in 2018. Today, their technology-agnostic, spatial biology suite includes the leading multiplexed imaging platforms and combines deep technical expertise required to develop high-performance multiplexed assays as large as 50 markers. Sophisticated multiplexed tissue imaging capabilities contributed to their selection as a Top 10 CRO of 2021 by Medhealth Outlook. “We are delighted to announce a partnership with Nucleai,” said Andrew Brown, Ph.D. Chief Commercial Officer at Sirona Dx. “Nucleai’s AI-driven analytical capabilities are exceptional. Our combined expertise in multiplexed tissue assay development, data generation and state-of-the-art analysis will unlock new spatial insights and accelerate the spatial biology revolution in precision medicine.” About Nucleai Nucleai is an AI-powered spatial biology company with a mission to transform drug development and clinical treatment decisions by unlocking the power of pathology data. Nucleai provides pharmaceutical companies, contract research organizations, and diagnostics laboratories with a state-of-the-art AI platform to improve clinical trials and clinical decision-making. About Sirona Dx Sirona Dx is a technical, CLIA accredited CRO, founded in 2016 to accelerate the pace of immunotherapy and targeted therapy development. With a leading-edge menu of specialized high complexity, single-cell proteomics and genomics services, Sirona Dx supports exploratory biomarker programs and advanced precision medicine initiatives to deliver safer and more effective treatment solutions.

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PHARMA TECH

BlackRock and QLS Advisors Partner Through BlackRock Systematic to Invest in Biomedical Innovation

BlackRock and QLS Advisors | July 19, 2022

BlackRock and QLS Advisors LLC, a life sciences technology and advisory firm, have announced a partnership through BlackRock Systematic to develop data-driven investment solutions to help accelerate biomedical innovation. The partnership will leverage the firms’ combined Research and Development and investment experience, directing funding toward public and private companies that have the potential to create transformative therapies while seeking to earn attractive investment returns for clients. Biomedicine is at an inflection point: breakthrough therapies—with the potential to extend longevity and improve quality of life—are emerging at an ever-increasing pace. Funding these therapeutic development programs has the potential to deliver attractive investment returns, but as biomedicine has grown more complex, so have the risks to investors. QLS has developed proprietary technology for estimating the probability of success of clinical trials, a key driver of financial performance, and systematically incorporating these forecasts into fundamental valuation models and portfolio construction algorithms. Their approach applies machine learning techniques to a multitude of predictive factors including therapeutic characteristics, clinical trial design features, disease-specific characteristics, and drug company track records. This methodology, coupled with BlackRock Systematic’s global platform, proprietary analytics, and more than 35 years1 of data-driven investment experience, presents a differentiated approach to investing in life sciences companies. “BlackRock’s partnership with QLS applies sophisticated quantitative investment technologies to the intersection of finance, academia, and life sciences. QLS brings an industry-leading approach to investing in life sciences that complements the work that BlackRock Systematic is doing in our actively managed portfolios. We expect this will create compelling, differentiated solutions that can deliver attractive returns for our clients while providing funding for life-changing therapies.” Rich Kushel, Head of the Portfolio Management Group BlackRock Andrew Lo, QLS Co-founder and Professor of Finance at the MIT Sloan School of Management commented: “We founded QLS to improve the way financial resources are allocated to emerging therapies, with the goal of lowering the cost of capital in this important sector. With BlackRock, we gain a partner who is a recognized leader in data-driven investing and shares our passion for delivering investment returns with positive outcomes for society. We’re thrilled and honored to join forces with a firm that has such a strong and trusted global brand, broad network, and access to world-class investment resources.” BlackRock and QLS have also assembled a Scientific Advisory Council comprising renowned academics, medical doctors, and inventors. The Council will support sourcing, due diligence, and value creation through the members’ vast networks and decades of experience as industry leaders in their respective fields. The Council is expected to include Dr. Robert Langer, Institute Professor, MIT Department of Chemical Engineering, and founder of over 40 biotechnology companies including Moderna. Ms. Lita Nelsen, retired director of the MIT Technology Licensing Office; now consultant in technology licensing. Dr. Larry Norton, Senior Vice President, Norna S. Sarofim Chair in Clinical Oncology and Medical Director of the Evelyn H. Lauder Breast Center at Memorial Sloan Kettering Cancer Center, Past President of the American Society of Clinical Oncology, ASCO David Karnofsky Awardee, FASCO, FAACR. Dr. Richard H. Scheller, Chairman of Research and Development BridgeBio. Board of Directors BridgeBio, Alector, 23andMe, DICE and others. Former Executive Vice President Research and Early Development Genentech and former HHMI Investigator and Professor Stanford University Medical School. Dr. Phillip A. Sharp, Institute Professor, MIT Department of Biology, geneticist and molecular biologist who co-discovered RNA splicing, co-founded Biogen and Alnylam, and the recipient of the 1993 Nobel Prize in Physiology or Medicine. About BlackRock Systematic BlackRock’s systematic investment platform brings together BlackRock’s community of systematic investors across equity, fixed income, and factor-based strategies. Systematic portfolios combine cutting-edge technology, scientific research, and human insight in the relentless pursuit of investment performance. With over 35 years of experience and nearly 200 professionals across the globe managing over US$200 bn in client assets as of March 31, 2022, BlackRock Systematic partners with clients to deliver quantitative alpha-seeking and factor-based investment solutions for the whole portfolio. About QLS Advisors LLC QLS Advisors LLC is a life sciences technology and advisory company based in Cambridge, MA, dedicated to fostering biomedical innovation. QLS employs a unique blend of fundamental and quantitative tools to help clients manage risk, assess reward, and develop investment and financing strategies for portfolios of healthcare-related assets.

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