CELL AND GENE THERAPY

MUSC and Helix launch In Our DNA SC, first-of-its-kind population genomics program to drive preventive, precision health care for South Carolinians

Helix | September 21, 2021

The Medical University of South Carolina (MUSC) and Helix have announced a strategic collaboration to develop a first-of-its-kind population genomics initiative in South Carolina called In Our DNA SC. The large-scale program is designed to improve health care outcomes by integrating genetic insights into clinical care and research. The statewide initiative will enroll 100,000 patients in genetic testing over the next four years at no cost to the patient.

The program will enable the use of genomic insights with an initial focus on actionable information regarding a patient's risk for certain forms of cancer and cardiovascular disease. The genetic reports will allow patients and their health providers to develop precision health care plans to proactively mitigate the conditions and take a more preventive approach to patient care. Patient enrollment in In Our DNA SC is expected to begin in the fall.

Medical University of South Carolina and Helix to develop a first-of-its-kind genomics initiative called In Our DNA SC.

In addition, MUSC and Helix will be developing a robust clinico-genomic dataset from consenting participants that will help researchers learn what can cause certain diseases, how we may be able to treat them more effectively and, possibly, improve the standard care for everyone. This is expected to lay the groundwork for a broader collaboration with other organizations across the health care value chain.

As South Carolina's only comprehensive academic health sciences center, delivering the highest quality care throughout the state is our top priority. Precision medicine is an emerging field that is going to transform the future delivery of health care. Being a leader and helping to define this path is core to our mission. We are excited to have the opportunity to partner with Helix to deploy this first-of-its-kind population genomic program for our patients. This collaboration will help drive preventive, precision health care for South Carolinians.

- David J. Cole, M.D., FACS, MUSC president

The strategic relationship with Helix allows MUSC to leverage Helix's unique Sequence Once, Query Often™ model and its end-to-end integration platform to enable immediate application and continual on-demand use of genetic insights throughout a patient's life. By working with South Carolina's only integrated academic health sciences center in the state, Helix gains access to thousands of providers and research staff dedicated to understanding how to deliver the highest quality patient care available to serve the people of South Carolina and beyond.

Enrollment in the program will initially be available to patients who sign up at select MUSC clinics and locations, later expanding to participants throughout the community and state in collaboration with MUSC's clinical affiliates and partners. Additionally, participants who consent to securely contribute their genetic data will help MUSC develop one of the largest clinico-genomic datasets in the country. Analyses from this platform will be used to pioneer and further advance genomics research.

About the Medical University of South Carolina
Founded in 1824 in Charleston, MUSC is home to the oldest medical school in the South as well as the state's only integrated academic health sciences center, with a unique charge to serve the state through education, research and patient care. Each year, MUSC educates and trains more than 3,000 students and nearly 800 residents in six colleges: Dental Medicine, Graduate Studies, Health Professions, Medicine, Nursing and Pharmacy. MUSC brought in more than $271 million in biomedical research funds in fiscal year 2020, continuing to lead the state in obtaining National Institutes of Health funding, with more than $129.9 million. As the clinical health system of the Medical University of South Carolina, MUSC Health is dedicated to delivering the highest quality and safe patient care while training generations of compassionate, competent health care providers to serve the people of South Carolina and beyond. Close to 25,000 care team members provide care for patients at 14 hospitals with approximately 2,500 beds and 5 additional hospital locations in development, more than 300 telehealth sites and nearly 750 care locations situated in the Lowcountry, Midlands, Pee Dee and Upstate regions of South Carolina. In 2021, for the seventh consecutive year, U.S. News & World Report named MUSC Health the No. 1 hospital in South Carolina. MUSC and its affiliates have collective annual budgets of $4.4 billion. The more than 25,000 MUSC team members include world-class faculty, physicians, specialty providers and scientists who deliver groundbreaking education, research, technology and patient care.

About Helix
Helix is the leading population genomics company operating at the intersection of clinical care, research, and genomics. Its end-to-end platform enables health systems, life sciences companies, and payers to advance genomic research and accelerate the integration of genomic data into clinical care. Powered by one of the world's largest CLIA / CAP next-generation sequencing labs and the first and only FDA authorized whole exome sequencing platform, Helix supports all aspects of population genomics including recruitment and engagement, clinically actionable disease screening, return of results, and basic and translational research. In response to the COVID-19 public health crisis, Helix has launched a sensitive and scalable end-to-end COVID-19 test system to meet the needs of health systems, employees, governments, and other organizations across the country.

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CELL AND GENE THERAPY

SOPHiA GENETICS Launches New Deep Learning Capabilities to Support the Detection of Homologous Recombination Deficiencies

SOPHiA GENETICS | March 08, 2022

SOPHiA GENETICS (Nasdaq: SOPH), a leader in Data-Driven Medicine, announced today the launch of their SOPHiA DDM Homologous Recombination Deficiency (HRD) Solution, opening new doors for cancer research. The application provides a unique approach for molecular cancer testing by combining the identification of HRD-causing mutations with the analysis of HRD-induced genomic instability across the whole genome of tumor samples. HRD is caused by a cell's impaired ability to repair DNA double-stranded breaks through the homologous recombination repair (HRR) pathway and is linked with the development of certain cancers. It is also an important predictor of tumor response to treatment with PARP inhibitors. Molecular testing of tumor samples using SOPHiA DDM enables simultaneous detection of both HRR mutations that can cause HRD, including those in the BRCA1 and BRCA2 genes, and the accumulated damage to genomic integrity that can result from them, using shallow whole-genome Next Generation Sequencing. SOPHiA GENETICS is collaborating with AstraZeneca, aiming to expand access to in-house HRD testing across European laboratories and institutions. Providing laboratories with innovative solutions to enable locally-delivered HRD testing is critically important, particularly in settings such as advanced ovarian cancer, where approximately half of all newly diagnosed patients have HRD-positive tumors, We are pleased to work with SOPHiA GENETICS in their mission to empower their laboratory customers and increase the range of options available for HRD detection." Kristina Rodnikova, SVP Global Oncology Diagnostics at AstraZeneca. To combat current challenges with HRD detection offerings, the SOPHiA DDM HRD Solution provides laboratories and institutions with a decentralized approach to HRD testing for tumor samples. The SOPHiA DDM HRD Solution allows the user to retain full ownership of their data and save time and expenses, while also offering comprehensive genomic insights powered by deep learning algorithms. We at SOPHiA GENETICS have always been on a mission to improve health outcomes by advancing data-driven medicine, which is why I am extremely excited to announce the launch of our new HRD research solution, Our SOPHiA DDM HRD Solution will provide researchers and other healthcare professionals with a decentralized approach to fully integrated HRD testing, enabling the HRD status of more advanced ovarian cancer cases to be determined without reliance on send-out testing solutions." Dr. Jurgi Camblong, Co-founder and CEO of SOPHiA GENETICS. About SOPHiA GENETICS SOPHiA GENETICS (Nasdaq: SOPH) is a healthcare technology company dedicated to establishing the practice of data-driven medicine as the standard of care and for life sciences research. It is the creator of the SOPHiA DDM™ Platform, a cloud-based SaaS platform capable of analyzing data and generating insights from complex multimodal data sets and different diagnostic modalities. The SOPHiA DDM™ Platform and related solutions, products and services are currently used by more than 790 hospital, laboratory, and biopharma institutions globally.

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INDUSTRIAL IMPACT

Cellarity Expands Leadership Team to Continue Evolution of a Breakthrough Platform to Encode Biology and Purposefully Create New Medicines

Cellarity | February 11, 2022

Cellarity, a life sciences company founded by Flagship Pioneering to encode and simulate human biology to reimagine the way we create medicines, announced today the expansion of its executive leadership team with three key appointments. As Chief Scientific Officer, Laurens Kruidenier, Ph.D., will lead the continued evolution of Cellarity’s platform and pipeline and oversee cross functional integration across all aspects of computation, drug creation, and optimization. As Chief People Officer, Anna O’Driscoll will lead the development of human resources and talent development strategies to enable the fast growth of Cellarity and make the company a top place to work for talent. As Senior Vice President and Head of Data and Software Engineering, Parul Doshi will spearhead the development of a leading and scalable data infrastructure to support the entire drug creation and development process. “We are building a transformative company and bringing together a strong team of people with a track record of making things happen. Laurens is not only an exceptional translational scientist, with experience and success advancing primary research into clinical-stage programs, he is also a team and company builder, helping integrate and mature drug discovery platforms.” Fabrice Chouraqui, Ph.D., CEO of Cellarity and a CEO-Partner at Flagship Pioneering Dr. Chouraqui continued, “Anna brings deep experience in talent and leadership development in the biotech industry, and she is known for her passion to positively impact the employee experience. Parul not only brings broad expertise in data infrastructure across the entire pharma value chain, from preclinical and clinical to tech operations and commercialization but also hands-on expertise in software engineering. I am excited that these top leaders have joined us at this pivotal time as Cellarity grows our team, advances key programs, and scales a pipeline of important new therapeutics.” Cellarity has developed unique capabilities to encode biology and model computationally the changes that occur between health and disease to design breakthrough medicines. Cellarity's transformative approach to drug creation looks beyond an individual molecular target to understand the complex biological network of cellular changes that underlie the start and progression of a disease. Built at the confluence of systems biology, single cell data, and machine learning, the Cellarity platform leverages a more complete view of cell biology to purposefully create drugs. Since all diseases stem from a disorder at the cellular level, Cellarity’s approach can be applied to virtually every disease and uncovers new biology and treatments that are unlikely to be found through target-based discovery. “Despite the tremendous progress the industry has made in advancing medical treatments, most diseases do not have curative therapies, and there are thousands of conditions that lack treatments,” said Dr. Kruidenier. “I’m impressed with Cellarity’s drug creation engine, which looks beyond the single target and embraces the full complexity of human disease. I look forward to working with the team to realize the truly transformative nature of the Cellarity platform and advance into the clinic novel medicines in diverse therapeutic areas to meet a wide range of patient needs.” Laurens Kruidenier, Ph.D., Chief Scientific Officer Dr. Kruidenier has more than 25 years of experience in discovery research, both in academic settings as well as in the life sciences industry and a track-record of success in drug discovery and development, driving research programs into clinical study and integrating teams and technologies for growth and scale. Prior to joining Cellarity, Dr. Kruidenier was Chief Scientific Officer (CSO) at Prometheus Biosciences, a precision medicine company in the immune-mediated disease space. At Prometheus, he helped mature the company from an early, seed-stage Cedars-Sinai-spinout into a Nasdaq-listed, platform-driven portfolio company with three active Phase 2 clinical trials. Previously, he held the roles of Vice President of Discovery at Second Genome, where he was responsible for a portfolio of microbiome programs and Head of GI Immunology Research at Takeda Pharmaceuticals, where he was responsible for developing the global discovery research and business development strategy into inflammatory diseases of the bowel. Earlier in his career, Dr. Kruidenier held various positions at different discovery units at GlaxoSmithKline, where he led preclinical teams and projects in immuno-inflammation, epigenetic, and protein degradation drug discovery. He completed his post-doctoral training at the University of London and holds an M.Sc. in medical biology from Utrecht University and a Ph.D. in mucosal immunology from Leiden University. His research has resulted in multiple publications in top tier scientific journals, including Nature. Anna O’Driscoll, Chief People Officer Ms. O’Driscoll has more than a decade of experience in human resources and was most recently Vice President of Human Resources at Alnylam Pharmaceuticals, where she worked in roles spanning R&D, Commercial, Corporate Functions, Learning and Development, and Employee Relations as Alnylam evolved from a research organization to a leading biotech company. Prior to Alnylam, she was Head of Talent Strategy and Process at Shire and in the Human Resources Leadership Development Program at Johnson & Johnson. Ms. O’Driscoll graduated with an MBA from the Institute of Chartered Financial Analysts and a bachelor’s degree in marketing and languages from Dublin City University. Parul Doshi, Senior Vice President, Head of Data and Software Engineering Ms. Doshi has more than twenty years of experience in information technology (IT) and software engineering and was most recently Head of IT & Digital for Takeda’s Vaccine division. She spent the past 16 years at Takeda, in roles of increasing seniority across several divisions of the company. She notably led the IT programs to launch oncology products NINLARO® and ALUNBRIG® as well as the consolidation of the Vaccines Business Unit to Cambridge, Massachusetts and integration of ARIAD Pharmaceuticals post acquisition. Prior to Takeda, Ms. Doshi worked as a developer at Patni Computer Systems, servicing major companies such as Fidelity Investments and LEGO®. Ms. Doshi graduated with an MBA in information technology and finance from Savitribai Phule Pune University, and a bachelor’s in economics and applied statistics from the University of Rajasthan. About Cellarity Cellarity's mission is to fundamentally transform the way medicines are created through the development of the first full stack digital cell. Founded by Flagship Pioneering in 2017, Cellarity has developed unique capabilities combining high-resolution data, single cell technologies, and machine learning to encode biology, model interventions, and purposefully design breakthrough medicines. By focusing on the cellular changes that underlie disease instead of a single target, Cellarity’s approach uncovers new biology and treatments and is applicable to a vast array of disease areas. The company currently has programs underway in metabolic disease, hematology, immuno-oncology, and respiratory disease. The company has raised $123 million as part of a Series B funding round with contributions from funds and accounts managed by Blackrock, The Baupost Group, Banque Pictet on behalf of their clients, and eight other investors alongside Flagship Pioneering. About Flagship Pioneering Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in more than $140 billion in aggregate value. To date, Flagship has deployed over $2.6 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 42 transformative companies, including Axcella Health (Nasdaq: AXLA), Codiak BioSciences (Nasdaq: CDAK) Denali Therapeutics (Nasdaq: DNLI), Evelo Biosciences (Nasdaq: EVLO), Foghorn Therapeutics Indigo Ag, Kaleido Biosciences (Nasdaq: KLDO), Moderna (Nasdaq: MRNA), Omega Therapeutics (Nasdaq: OMGA), Rubius Therapeutics (Nasdaq: RUBY), Sana Biotechnology (Nasdaq: SANA), Seres Therapeutics (Nasdaq: MCRB), and Sigilon Therapeutics.

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MEDICAL

Botanical Solution Inc. (BSI) Secures $6.1 Million in Series A First Closing

Botanical Solution Inc. | January 18, 2022

Botanical Solution Inc. innovator of sustainable, consistent and cost-effective Advanced Botanical Materials for agricultural and pharmaceutical applications, has successfully closed $6.1 million in a Series A round led by Palo Alto based VC Firm, Otter Capital. This round of funding will enhance BSI’s value proposition by facilitating the geographic expansion of its current botanical-based products and development of a new product pipeline derived from its proprietary biotech platform. For agricultural markets, this Series A round of funding will support the production and sales growth of BSI’s first commercially successful product marketed as BotriStop® and its recently announced global brand Quillibrium®, which are based on a tissue cultured tree native to Chile, the Quillaja saponaria. The product has been used successfully on a wide variety of fruits and vegetables throughout Chile. Quillibrium® was launched in Peru last year with great success and will soon reach growers across Mexico through BSI’s main distribution partner Syngenta. Quillibrium® continues regulatory progress in the US and will soon initiate registration in additional countries to expand its global footprint. This new funding will also accelerate market readiness of new botanical biopesticide products currently in later development stages and enable the expansion of BSI’s R&D pipeline. For pharmaceutical markets, BSI’s new funding will vastly increase the quantity of Quillaja saponaria plants that can be grown at BSI’s labs and the sustainable production of billions of doses of gold standard adjuvant QS-21, traditionally obtained from old native trees that are increasingly affected by climate change and strict deforestation laws in Chile. QS-21 is currently used in Covid-19 and malaria vaccines, as well as a blockbuster shingles vaccine. With this funding BSI will become a major supplier of pharmaceutical grade QS-21. BSI CEO Gastón Salinas declared, “The BSI team is really excited to welcome Otter Capital. We are happy to reconfirm the strong commitment from our current investors, as well. BSI is building strong foundations to deliver the world’s most advanced botanical biopesticides and ensure the supply of the gold standard vaccine adjuvant QS-21.” According to John Pasquesi, Managing Member at Otter Capital, “I am excited to partner with BSI. BSI has established a strong market presence via distribution agreements with Syngenta and effective, profitable products. BSI’s product development platform is efficient and scalable. Botanical products have excellent consistency and BSI’s approach is truly sustainable. These products have applications in two very large markets – agriculture and pharmaceuticals. I am very impressed by the team at BSI and the progress they have made with limited resources under Gastón’s leadership.” Two longtime investors joining Otter Capital in this First Closing of the Series A are Inversiones el Coigue and Inversiones Eurocel, both based in Santiago, Chile. About BSI BSI, a Delaware Corporation has a proprietary R&D platform for truly sustainable and improved production of consistent and high- quality Advanced Botanical Materials (ABM). ABM-01 is the first ABM produced by the company, based on a tissue cultured plant called Quillaja saponaria. ABM-01 is the active ingredient used in two gold standard products, BSI’s biopesticide Quillibrium® and the adjuvant QS-21, used in modern vaccine development. About Otter Capital Otter Capital was founded by John Pasquesi to make a variety of private equity investments. Otter Capital has a strong track record investing in leading edge agriculture companies. Otter was an early and consistent investor in AgraQuest, a leading biopesticide company where Mr. Pasquesi served as chairman of the board of directors. In 2012, Bayer CropScience acquired AgraQuest for $425 million. More recently, Otter Capital invested in the agricultural biotechnology companies BioConsortia, Inc. and NewLeaf Symbiotics, Inc. Mr. Pasquesi serves on the board of directors of BioConsortia and as chairman of the board of NewLeaf. Otter Capital is an investor in Inguran LLC dba Sexing Technologies, the world’s leading supplier of sexed semen to the dairy industry and a leading provider of dairy and beef genetics. Mr. Pasquesi serves on the board of managers of Inguran.

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INDUSTRY OUTLOOK

Curocell, Breaks Ground on 'CAR-T Manufacturing GMP Facility'

Curocell, Inc. | December 24, 2021

Curocell, currently on PhaseⅠCAR-T Therapy clinical trial with CRC01 has recently broken ground for the new CAR-T Center in Dungok Residential & Industrial Area in Daejeon International Science & Business Belt. The GMP Facility for commercial manufacturing CAR-T Cell therapy and R&D Center for further pipeline development are constituting the 17,325 square-meter site of CAR-T Center. By the first half of 2023, construction is expected to be completed and in 2024, when the CAR-T Center is fully operational, supply of advanced cure should be possible for patients in pain. Also, Curocell is expecting to expand their business to global market by in-housing all processes of CAR-T therapy development and production. About CRC01 (anbalcabtagene autoleucel) CRC01, recognized CD19 and is based on OVIS™, a first-in-class CAR-T platform. OVIS™ technology downregulates PD1 and TIGIT expression on CAR-T cells. Through overcoming the immune suppression by PD-1 and TIGIT ligands, OVIS™ CAR-T has superior cytotoxicity to tumor cells in the tumor microenvironment. Phase II clinical trial of CRC01 will start in South Korea in the first half of 2022. About Curocell Curocell, based in Daejeon, South Korea, is clinical-stage biotech innovating CAR-T therapies. Curocell is developing OVIS™ technology with the goal of improving clinical efficacy of CAR-T therapies.

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