On December 16, 2021 Neogene Therapeutics, Inc., a preclinical stage biotechnology company pioneering a new class of fully individualized T cell receptor (TCR) therapies to treat cancer, reported the appointment of Raphaël Rousseau, M.D., Ph.D. as Chief Medical Officer (Press release, Neogene Therapeutics, DEC 16, 2021, View Source [SID1234597330]). Dr. Rousseau brings more than 20 years of extensive experience in oncology drug development, including engineered T cell therapies, in both academia and the biotechnology industry. He joins Neogene’s executive team and will be based in its U.S. headquarters in Santa Monica.

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"We’re thrilled to welcome Raphaël at this crucial and exciting time in our company’s growth. He is a strong leader, who has led teams across all phases of development and has successfully built and executed multiple global clinical programs," said Carsten Linnemann, Ph.D., President, Chief Executive Officer and co-founder of Neogene. "Raphael’s broad experience with cell therapy and neo-antigen targeting drug modalities will be critical in helping us to diversify our pipeline and advance our programs into the clinic with the goal of bringing T cell therapies to patients across a broad spectrum of solid tumors."

Dr. Rousseau most recently served as Executive Vice President, Head of Product Development, and Chief Medical Officer at Gritstone bio, where he was responsible for the company’s global clinical development strategies and expansion of its neoantigen-based immunotherapies. He led the buildout of the clinical development organization and execution of Gritstone’s clinical strategy including conducting several phase 1 and phase 2 clinical trials. Prior to Gritstone bio, Dr. Rousseau served as the Global Franchise Head of Pediatrics at Genentech and Roche, where he successfully led a global development and study management team and served as the Chair of the Development Review Committee for early and late-stage pediatric asset reviews. While at Genentech, he oversaw the clinical development of the company’s oncology portfolio from first-in-human to registrational trials for several pediatric hematology and oncology indications. Prior to joining industry, Dr. Rousseau was a Professor of Medical and Pediatric Oncology at the Université Claude Bernard in Lyon, France, and the head of the pediatric translational research program at Léon Bérard Cancer Center. There, he created and led the first European academic consortium for the conduct of a phase 1 study using CD19-targeting CAR T cells for the treatment of pediatric high-risk leukemias. Earlier in his career, Dr. Rousseau was a fellow at Baylor College of Medicine in Houston, where he worked with Dr. Malcolm Brenner at the Center for Cell and Gene Therapy. Dr. Rousseau received a Ph.D. in Therapeutic Biotechnologies from the Université Denis Diderot and an M.D. from the Université René Descartes in Paris. He is board certified in pediatrics and has a sub-specialty certification in pediatric hematology-oncology.

"I’m proud to be joining the Neogene team, which has been making great strides in the field of neoantigen-directed T cell therapy, and excited about helping advance our pioneering research into the clinic to potentially help patients who currently have limited treatment options," said Dr. Rousseau. "This is a field that I am extremely passionate about, and I am excited to apply my expertise in successfully advancing these promising therapies into clinical trials as we work to change the solid cancer treatment paradigm."

On December 16, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, reported a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases (Press release, BioMarin, DEC 16, 2021, View Source [SID1234597351]).

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The partnership will leverage Skyline Therapeutics’ integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options.

Under the agreement, BioMarin and Skyline Therapeutics will collaborate on discovery and research through to an Investigational New Drug Application (IND). BioMarin brings experience in gene therapy development, cardiovascular biology and insights into genetic basis of diseases, and Skyline contributes its expertise in developing gene therapy products including vector engineering and design technology and manufacturing capabilities to this collaboration. Each company will advance the programs through clinical development in their pre-defined territories.

In support of its R&D efforts for the collaborative projects, Skyline Therapeutics will receive an undisclosed payment associated with signing, comprising an upfront payment and an equity investment from BioMarin, and is eligible to receive pre-specified payments for R&D, regulatory and commercial milestones.

BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region. In addition, Skyline Therapeutics will be eligible to receive royalty payments on future sales from BioMarin in its territories.

"We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline’s innovative approach to AAV vector engineering and design and our team’s proven expertise in creating and developing gene therapies," said Kevin Eggan, Group Vice President, Head of Research and Early Development, from BioMarin.

"We are excited to partner with Skyline Therapeutics to tackle these genetic forms of dilated cardiomyopathy. This collaboration strengthens our leadership in cardiac gene therapy and extends our R&D collaboration to Asia, where a large number of patients suffer from these devastating diseases," said Brinda Balakrishnan, Group Vice President, Corporate and Business Development at BioMarin. "We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide."

"Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. Mutations in many genes are associated with the development of DCM, among other etiologies for the disease," said Jay Hou, Chief Scientific Officer at Skyline Therapeutics. "Together with BioMarin’s team we have identified a number of critical genes associated with DCM. We are delighted to work closely with BioMarin and apply our AAV vector technology to interrogate these new targets and develop novel treatments for DCM patients."

"The collaboration with BioMarin leverages both companies’ capabilities in the development of gene therapies. With the BioMarin team, we share the goal of working in concert to develop therapies for genetic cardiovascular disease that address high unmet medical needs," said Amber Cai, CEO of Skyline Therapeutics. "Together, we will utilize gene therapy to tackle cardiovascular diseases with a disease modifying trailblazing approach that could change the treatment paradigm in these conditions."

About Dilated Cardiomyopathy (DCM)

DCM is a common cause of heart failure and end-stage DCM, which often leads to heart transplantation. Despite improvements in pharmacotherapy and care, the five-year survival rate of DCM is only about 50%. Hundreds of thousands of patients suffer from the genetic forms of DCM in U.S., EU, China, and Japan. More than 50 genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology. Our aim is to correct the pathways altered by these genetic contributors to DCM through AAV based gene therapy, in each case addressing the root cause of the disease.

On December 16, 2021 Perimeter Medical Imaging AI, Inc. (TSX-V:PINK)(OTC:PYNKF) (FSE:4PC) ("Perimeter" or the "Company"), a medical technology company driven to transform cancer surgery with ultra-high-resolution, real-time, advanced imaging tools to address high unmet medical needs, reported the initiation of a multi-center, randomized, two-arm pivotal clinical trial to evaluate its Perimeter B-Series OCT with ImgAssist AI for use during breast conservation surgery (Press release, Perimeter Medical Imaging AI, DEC 16, 2021, View Source [SID1234597331]). The first patient procedures were performed by Richard E. Fine, MD and Michael Berry, MD, leading breast surgeons based out of the Margaret West Comprehensive Breast Center in Germantown, Tennessee.

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Dr. Richard E. Fine, Director of Education & Research, Margaret West Comprehensive Breast Center, West Cancer Center & Research Institute, stated, "We are excited to be the first site to enroll a patient in the Perimeter pivotal trial that is evaluating the use of Perimeter B-Series OCT imaging technology combined with artificial intelligence. There is a strong unmet medical need to provide breast cancer surgeons with specimen imaging tools to aid their decisions ‘real time’ in the operating room. Through this pivotal study, we can assess if the Perimeter B-Series with artificial intelligence demonstrates an improvement over the current standard of care – potentially setting a new standard for specimen imaging technology during breast conservation surgery."

Dr. Michael Berry, Director of Margaret West Comprehensive Breast Center, commented, "Approximately 15% to 20% of women who undergo breast conservation surgery require reoperation if their surgeon fails to get clear margins. By combining optical coherence tomography with deep learning algorithms, this innovative technology could assist us, as surgeons, to better identify regions of interest on scanned samples, enabling ‘real-time’ decisions on margin status in the OR."

Jeremy Sobotta, Perimeter’s Chief Executive Officer stated, "The initiation of this pivotal trial marks another milestone in our ATLAS AI project and an important step in the clinical development of our breakthrough-device-designated Perimeter B-Series OCT with AI assisted software, which represents the next generation of our commercially available flagship Perimeter S-Series OCT. Our hope is that clinical data generated from this study will provide supporting evidence that our technology can help breast cancer physicians improve outcomes for patients and potentially reduce the burden of additional costs within the healthcare system. We expect that this study will be completed by the end of 2022."

On December 16 2021 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL inhibitors for severe unmet medical needs, reported the licensing of certain intellectual property from the UT Southwestern Medical Center (Press release, BerGenBio, DEC 16, 2021, View Source [SID1234597352]). The exclusive license to intellectual property generated by researchers at UT Southwestern Medical Center supports the Company’s existing IP position underlying use of AXL inhibitors, including its lead product candidate bemcentinib, for the treatment of patients harboring STK11 mutations in NSCLC. No financial terms have been disclosed.

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The STK11 mutation is observed in up to 20% of NSCLC patients. STK11 is an important tumor suppressor gene associated with a poor prognosis in NSCLC patients and which has been reported in some studies to confer resistance to immune checkpoint inhibitors.

Researchers at the UT Southwestern Medical Center and BerGenBio recently presented data at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting exploring why patients harboring a mutation to the STK11 gene may not respond to anti-PD1 therapy, a commonly used treatment in first line NSCLC patients. Preclinical research further indicated that the addition of the selective AXL inhibitor bemcentinib restored the activity of anti-PD1 treatment in relevant lung cancer models

Martin Olin, Chief Executive Officer at BerGenBio, commented: "The license agreement with UT Southwestern Medical Center complements our existing intellectual property filings in this important patient population which appears to poorly respond to anti-PD1 therapy. We will continue to explore how this target group might be addressed by the combination of bemcentinib and an immune checkpoint inhibitor."

On December 16, 2021 NEC Corporation (NEC; TSE: 6701) reported the development of an AI technology with Ono Pharmaceutical Co., Ltd. that detects particles in glass bottles (Press release, NEC, DEC 16, 2021, View Source [SID1234597515]). This technology uses AI to learn the difference in movements between bubbles and particles in liquid for injections within glass bottles. The technology is even able to detect micro-particles that have been difficult to identify until now with high accuracy. Both companies aim to commercialize the technology in the 2022 fiscal year.

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In the process of inspecting liquids for injections that are contained in glass bottles, such as anti-cancer drugs and vaccines, it is common for bottles suspected of containing particles to be automatically removed by inspection equipment, and for a secondary visual inspection to take place by inspectors. Initially, an image-based inspection is performed on all glass bottles. However, normal bottles are often recognized as defective and eliminated. This is due to bubbles being recognized as particles. In addition, advanced skill and know-how are required for inspectors to perform secondary visual inspections, inspection quality often varies, and securing inspectors at the time of production is a challenge.

NEC has partnered with Ono Pharmaceutical to reduce the number of visual inspections by applying this new AI technology to automated inspections, aiming to realize rapid processes that feature uniform inspection quality and help to support the expansion of production.

Image of technology utilization
Technology Features
1. Detect particles with high accuracy based on inspection procedures by inspectors
Visual inspection by inspectors involves tilting glass bottles and stirring liquid in order to identify the differences in the movement between particles and bubbles. This technology uses a high-speed camera to capture the inside state of the bottle. Then, it is analyzed as slow-motion video in order to accurately understand the movement of particles and bubbles, and to recognize the slight differences. This makes it possible to detect particles with high accuracy.

2. Various types of micro-particles can be detected by focusing on motion
In recent years, AI that is capable of recognizing objects within images by using machine learning technologies, such as deep learning, is attracting attention. However, even if a system learns to see particles in an image, it is not possible for the system to work for non-learned particles. Also, it is impractical to enlarge the image so that small particles and bubbles can be distinguished. This new technology distinguishes them by focusing on the movement in which the specific gravity and shape features of particles appear. This allows the technology to work for various kinds of tiny particles.

Going forward, NEC will continue to collaborate with Ono Pharmaceutical and to proceed with verification activities, aiming to bring inspection equipment using this technology into practical use.