CELL AND GENE THERAPY

BeiGene Initiates First-in-Human Phase 1 Clinical Trial of Investigational TYK2 Inhibitor BGB-23339

BeiGene | November 23, 2021

BeiGene a global, science-driven biotechnology company focused on developing innovative and affordable medicines, announced that the first patient has been dosed in a Phase 1 clinical trial of BGB-23339, a potent, allosteric investigational tyrosine kinase 2 (TYK2) inhibitor internally developed by BeiGene scientists.

TYK2 is a member of the JAK family and functions as a critical mediator in cytokine signaling pathways implicated in multiple immune-mediated disorders, such as psoriasis and inflammatory bowel disease. BGB-23339 is a potent, highly selective, investigational TYK2 inhibitor targeting the regulatory pseudokinase (JH2) domain.

Discovered and developed by BeiGene, BGB-23339 is a highly selective, potent, allosteric TYK2 inhibitor that has shown promising activity in preclinical evaluation. Building on our proven track record in oncology, BeiGene is expanding its clinical focus to discover new modalities and platforms in areas of high unmet need, including inflammation and immunology, to bring innovative, impactful medicines to patients.”

Lai Wang, Ph.D., Global Head of R&D at BeiGene

The first-in-human Phase 1 trial (NCT05093270) is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary activity of BGB-23339. The trial is expected to enroll up to 115 healthy volunteers in Australia and/or China.

In addition to its broad portfolio focused on hematological malignancies and solid tumors, BeiGene is applying its research excellence and clinical expertise to address inflammation and immunology, an area of high unmet medical need. BeiGene’s internally developed, highly selective next-generation BTK inhibitor BRUKINSA® (zanubrutinib) is currently being evaluated in a Phase 2 trial in patients with active proliferative lupus nephritis.

About BGB-23339
BGB-23339 is a potent, highly selective, allosteric, investigational tyrosine kinase 2 (TYK2) inhibitor discovered and being developed by BeiGene. TYK2 is a member of the JAK family and functions as a critical mediator in cytokine signaling pathways implicated in multiple immune-mediated disorders. Designed to target the regulatory pseudokinase (JH2) domain on TYK2, BGB-23339 has demonstrated strong selectivity in preclinical studies with potent inhibition of interleukin (IL)-12, IL-23, and Type 1 interferons (IFNs)—pro-inflammatory cytokines that play a determinant role in the induction of inflammation. BGB-23339 is currently being evaluated in a Phase 1 clinical study.

About BeiGene
BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 7,700 colleagues across five continents. 

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding plans for the Phase 1 trial and development of BGB-23339, the potential for BGB-23339 to address unmet medical needs, BeiGene’s plan to expand its clinical focus to discover new modalities and platforms in areas of high unmet need, including inflammation and immunology, and BeiGene’s plans, commitments, aspirations and goals under “About BeiGene”. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products and its ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates and achieve and maintain profitability; the impact of the COVID-19 pandemic on the BeiGene’s clinical development, regulatory, commercial, and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent quarterly report on Form 10-Q as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

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MEDICAL

Texas Children's Pavilion for Women Acquires Austin Perinatal Associates Expanding Hospital's Maternal-Fetal Medicine Footprint to Central Tex

Texas Children's Hospital | May 09, 2022

Texas Children's Physician Group announced today that it has completed the acquisition of Austin Perinatal Associates, a well-known private maternal-fetal medicine practice based in Austin. Located at 6500 North Mopac Expressway, the facility is the ninth community maternal-fetal medicine clinic operated by the Texas Children's Pavilion for Women and the first to offer women's services in Central Texas – bringing the hospital's well-known, top tier women's health care closer to patients in this growing region of the state.  The announcement comes a decade after the expansion of Texas Children's Hospital into obstetrics and gynecological care, with a special focus on high-risk pregnancies, fetal surgery, and multiple births. Additional growth in the area includes a new freestanding, top tier children and women's hospital in North Austin that will top off in mid-May. Texas Children's Hospital Austin is set to open Q1 2024. The clinic, founded by Maternal-Fetal Medicine Specialist Dr. David L. Berry, has served as a convenient location for patients to receive a full array of care, including ultrasounds, genetics services, antenatal testing, and diabetes and hypertension management since 1997. Patients will now have access to clinical support with specialty programs at Texas Children's, including the Texas Children's Hospital Fetal Center, The Women's Place, and nutritional counseling Texas Children's continues to expand from Houston into the Austin area to bring Central Texans the same personalized, family-centered, high-quality medical care and services they've provided since 1954, With more than 60 years of newborn care experience, over 6,000 births a year, and maternal-fetal medicine offices throughout the Houston area, I'm excited to be a part of their expansion efforts into Austin." Berry. Berry is a fourth generation physician who returned to his hometown following his residency and post-graduate fellowship at Baylor College of Medicine to launch Austin Perinatal Associates in order to meet the medical needs of women experiencing high-risk pregnancies. His expertise includes prenatal diagnosis, invasive fetal diagnostics and therapeutics, infectious disease, cancer in pregnancy, and critical care obstetrics. We are proud to announce the growth of our world-class, specialized care for mothers and babies in the Austin community and excited to welcome Dr. Berry to our team, Our maternal-fetal medicine physicians at Texas Children's Fetal Center are known worldwide for their expert care for high-risk pregnancies, and this clinic allows for a seamless connection to our Fetal Center – one of the nation's leaders in the diagnosis and treatment of abnormalities in unborn and newborn infants." Dr. Michael Belfort, OB/GYN-in-Chief at Texas Children's Pavilion for Women and Professor and Chair of the Department of Obstetrics and Gynecology at Baylor College of Medicine. About Texas Children's Hospital Texas Children's Hospital, a not-for-profit health care organization, is committed to creating a healthier future for children and women throughout the global community by leading in patient care, education and research. Consistently ranked as the best children's hospital in Texas, and among the top in the nation, Texas Children's has garnered widespread recognition for its expertise and breakthroughs in pediatric and women's health. The hospital includes the Jan and Dan Duncan Neurological Research Institute; the Feigin Tower for pediatric research; Texas Children's Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; Texas Children's Hospital West Campus, a community hospital in suburban West Houston; and Texas Children's Hospital The Woodlands, the first hospital devoted to children's care for communities north of Houston. The organization also created Texas Children's Health Plan, the nation's first HMO for children; has the largest pediatric primary care network in the country, Texas Children's Pediatrics; Texas Children's Urgent Care clinics that specialize in after-hours care tailored specifically for children; and a global health program that's channeling care to children and women all over the world. Texas Children's Hospital is affiliated with Baylor College of Medicine.

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CELL AND GENE THERAPY

Dyadic Announces a NIIMBL Coronavirus Grant Under the White House’s American Rescue Plan

Dyadic International, Inc. | December 23, 2021

Dyadic International, Inc. a global biotechnology company focused on further improving, applying and deploying its proprietary C1-cell protein production platform to accelerate development, lower production costs and improve access to biologic vaccines and drugs at flexible commercial scales, announced it is the recipient of one of thirty-two project grants awarded by the National Institute for Innovation in Manufacturing Biopharmaceuticals funded through the White House’s American Rescue Plan. Under the NIIMBL grant, Dyadic will receive up to $690,000 in funding to engineer the Company’s proprietary and patented C1 thermophilic fungal protein production platform to produce two different antibodies. “We are honored to have been selected by NIIMBL to receive this grant and to further leverage Dyadic’s C1-cell protein production platform to help the nation prevent, prepare for, and respond to coronavirus and other public health threats. The grant provides us with the opportunity, among other attributes, to further demonstrate to academia, industry, and governmental agencies the rapid speed at which we can develop stable C1 expressing cell lines to produce antibodies as compared to traditional cell lines, such as Chinese Hamster Ovary cells. We look forward to moving this project forward in the new year,” Mark Emalfarb, Dyadic’s President and Chief Executive Officer NIIMBL members include large and small companies, academic institutions, non-profits, and federal agency partners who are well-equipped to significantly strengthen the nation’s preparedness and response to public health crises. Through the ARP funding, NIIMBL has selected projects that will Provide testbeds for process development, for next generation manufacturing approaches, and for drug product manufacturing approaches that provide access to industrially relevant equipment and facilities for pre-competitive manufacturing innovation Support the development of significantly improved methods for vaccine production, quality, and distribution, including mRNA and other vaccine platforms Demonstrate rapid scaling for production of coronavirus antigens and medical countermeasures for variants of concern Train a diverse biopharmaceutical manufacturing workforce Demonstrate the concept of mobile Good Manufacturing Practices capabilities to support mRNA vaccine and gene therapy production Dyadic’s project grant is intended to benchmark the speed that the C1 manufacturing platform will have when compared to current state of the art methods, which could lead to a rapid ability to produce medical countermeasures and vaccines in response to future pandemics. About NIIMBL The National Institute for Innovation in Manufacturing Biopharmaceuticals is a public-private partnership whose mission is to accelerate biopharmaceutical innovation, support the development of standards that enable more efficient and rapid manufacturing capabilities, and educate and train a world-leading biopharmaceutical manufacturing workforce, fundamentally advancing U.S. competitiveness in this industry. NIIMBL is part of Manufacturing USA®, a diverse network of federally-sponsored manufacturing innovation institutes, and is funded through a cooperative agreement with the National Institute of Standards and Technology in the U.S. Department of Commerce with significant additional support from its members. Visit NIIMBL.org to view a summary of these projects and to learn more about NIIMBL. About Dyadic International, Inc. Dyadic International, Inc. is a global biotechnology company that is developing what it believes will be a potentially significant biopharmaceutical gene expression platform based on the fungus Thermothelomyces heterothallica, named C1. The C1 microorganism, which enables the development and large-scale manufacture of low-cost proteins, has the potential to be further developed into a safe and efficient expression system that may help speed up the development, lower production costs and improve the performance of biologic vaccines and drugs at flexible commercial scales.

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Illumina Introduces New Pan-Cancer Companion Diagnostic to Match Patients with Rare Genetic Mutations to Targeted Therapy

Illumina | May 27, 2022

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Paula Dowdy, Senior Vice President and General Manager of Illumina for Europe, the Middle East, and Africa. Across most solid cancer tumor types, NTRK gene fusions can have a rare prevalence of 0.1-3% and can be challenging to detect as these genes fuse with many different partners, many of them previously unknown. Most CDx tests are specific to one type of cancer, but the NTRK claim is pan-cancer, enabling TSO Comprehensive (EU) to target multiple solid tumor types and a broad range of known and novel gene fusion partners across all three NTRK genes. This helps maximize the chances of finding actionable information from each patient's biopsy, to inform treatment with larotrectinib. Since it launched four years ago, VITRAKVI has demonstrated high response rates and highly durable responses in adults and children with TRK fusion cancer, The CE marking of Illumina's TSO Comprehensive (EU) assay inclusive of NTRK gene fusion detection for VITRAKVI is an important advance in enabling precision oncology for patients in Europe. Assays that enable precision oncology through comprehensive genomic testing are crucial for informing optimal treatment plans and help to ensure the best possible outcomes for cancer patients. We look forward to continuing to collaborate with Illumina to ensure that more patients are evaluated comprehensively inclusive of NTRK gene fusions." Christine Roth, Member of the Executive Committee of Bayer's Pharmaceuticals Division and Head of Bayer's Oncology Strategic Business Unit. The performance of TSO Comprehensive (EU) with NTRK fu­sions was assessed by analytical and clinical validation studies. 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Professor Richard Kennedy, Global VP of Biomarker Development & Medical Director, Almac Diagnostic Services. Illumina has a growing pipeline of CDx claims under development through partnerships with pharmaceutical companies, which will be added to TSO Comprehensive (EU) following appropriate regulatory approvals. These CDx claims will help unlock groundbreaking targeted therapies and immunotherapies to make a difference in the lives of cancer patients. As Illumina continues to expand its broad portfolio of oncology partnerships with industry leaders, the company aims to advance cancer diagnostics and precision medicine. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2021, the Group employed around 100,000 people and had sales of 44.1 billion euros. R&D expenses before special items amounted to 5.3 billion euros. About Illumina Illumina is improving human health by unlocking the power of the genome. Our focus on innovation has established us as a global leader in DNA sequencing and array-based technologies, serving customers in the research, clinical and applied markets. Our products are used for applications in the life sciences, oncology, reproductive health, agriculture and other emerging segments.

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RESEARCH

BigHat Biosciences Completes First Stage of Research Collaboration with Amgen

BigHat Biosciences, Inc. | January 12, 2022

BigHat Biosciences, Inc., a biotechnology company with an artificial intelligence/machine learning-guided antibody discovery and development platform, announced the successful completion of the first stage of a previously undisclosed research collaboration and licensing agreement with Amgen applying BigHat’s platform for multi-objective optimization of a next-generation antibody. BigHat’s antibody design platform integrates a high-speed characterization with AI/ML technologies to engineer antibodies with more complex functions and better biophysical properties. This approach reduces the difficulty of designing antibodies and other therapeutic proteins to tackle conditions ranging from chronic illness to life-threatening disease. BigHat’s experimental platform massively speeds up candidate discovery and validation. “This is an important milestone for BigHat, and the AI/ML biologics drug discovery field more broadly, as it demonstrates the ability of their platform to quickly and significantly optimize next-generation antibodies,” said Steve Doberstein, BigHat Independent Board Member and former Chief R&D Officer at Nektar Therapeutics, Inc. Achievement of this first milestone shows that BigHat’s platform has the potential to design high-quality therapeutic antibodies effectively and efficiently. Its platform can synthesize, express, purify, and characterize antibodies in a fraction of the time compared to traditional labs to guide the search for better molecules. “BigHat's platform for data-driven antibody design generated several antibodies significantly better than the starter molecules found using traditional technologies. Now that Amgen has validated the capabilities of BigHat's unique approach to antibody development, we're excited to continue working with them towards a lead antibody for their discovery research.” Vineeta Agarwala, MD, PhD, General Partner at Andreessen Horowitz and BigHat Board Director This successful milestone triggers the initiation of work to create a lead panel of VHH antibodies for patients in need. “We are excited to show the power of our platform to rapidly improve biophysical characteristics and function by directing and learning from each cycle of our AI/ML-enabled experimental platform. We are looking forward to continuing our productive collaboration,” said Peyton Greenside, BigHat’s CSO and Co-Founder. About BigHat Biosciences, Inc. BigHat Biosciences is reimagining antibody discovery and engineering with an AI-first experimental platform that integrates a high-speed wet lab with machine learning to create better antibodies faster and undertake novel designs far beyond what’s possible today. BigHat applies these design capabilities to develop new generations of safer and more effective treatments for patients suffering from today’s most challenging diseases. BigHat is a Series A stage biotechnology company based in the San Francisco Bay Area with a team-oriented, inclusive, and family-friendly culture. BigHat is backed by a16z, 8VC, AME Cloud Ventures, and Innovation Endeavors and counts among its employees and advisors a Nobel Laureate, a serial life-science entrepreneur, and leaders in fields from antibody development to machine learning.

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