Researchers Create Embryoids Using Stem Cells

BioSpace | July 02, 2019

Much of what happens as a human embryo develops is unknown. Its been defined in animal models, but not in humans. Much of the reason for that is related to ethical limitations on what type of research can be conducted on human embryos. There is a ban, which is called the 14-day rule, on conducting experiments on real human embryos after 14 days of growth. Researchers at Rockefeller University appear to have found a way to work around that ethical limitation, although it brings its own set of concerns with it. The scientists, led by molecular biologist Ali Brivanlou, used human embryonic stem cells to develop living models of human embryos that could be studied in the laboratory. The three-dimensional model of a human epiblast had the size, cell orientation and gene expression roughly equivalent to a day 10 human epiblast. We came up with a model of human embryos that are developed outside of the womb and is not the product of the sperm and the eggs but is the product of human embryonic stem cells that self-organize into complicated structures, Brivanlou told NPR.

Spotlight

President and CEO Howard Minigh details the benefits plant biotechnology has brought globally over past 20 years.

Spotlight

President and CEO Howard Minigh details the benefits plant biotechnology has brought globally over past 20 years.

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CELL AND GENE THERAPY

IndyGeneUS AI Secures Pathogen Sequencing for COVID-19 Genomic Surveillance Initiatives

IndyGeneUS AI | May 20, 2021

Through collaborations, IndyGeneUS AI has acquired genomic analysis and testing capabilities. This pathogen sequencing capacity has emerged at a critical moment in efforts to improve genomic surveillance and monitor COVID-19 variant strains across the continent. IndyGeneUS founder and CEO Yusuf Henriques emphasized the importance of this work. "COVID-19 and its variants pose a danger to the entire African continent, so we must act quickly. Our partners understand the urgency of this mission and are united with their cooperation." Next-generation sequencing (NGS)-based genotyping, targeted sequencing, and comprehensive bioinformatics analysis are among the capabilities. Comparative genomic analysis, disease genetics, epigenomics, and genome mapping are some of the applications of our capabilities. Bradford Wilson, Ph.D., Chief Scientific Officer at IndyGeneUS AI, said, "These capabilities are pivotal for the Continent and the future of precision medicine." "We are currently in talks with several large pharmaceutical firms that see the value of what we are developing to combat infectious and non-communicable diseases alike," he said. In less than a year, IndyGeneUS AI has partnered with Kenyan organizations such as the KAVI Institute of Clinical Research at the University of Nairobi and Afya Rekod. We've also teamed up with Encrypgen, which has created a blockchain-encrypted marketplace for commercializing insights gleaned from our repository and compensating participants who contribute data. IndyGeneUS has certainly understood the increasing need for infrastructure to sustain genomic surveillance activities by developing sequencing capabilities. Following the introduction of COVID-19 variant strains, the World Health Organization (WHO) and the African Centres for Disease Control (Africa CDC) established a network of laboratories last year to improve the continent's capacity to identify emerging variants of concern. More funding and infrastructure are needed for these initiatives. IndyGeneUS intends to accelerate sequencing capabilities across Africa by using public and private partnerships. About IndyGeneUS AI IndyGeneUS AI is a genomics company based in Nairobi is also developing the world's largest block-chain encrypted repository of African clinical and multi-omics data for disease prevention and diagnosis, drug discovery and development, clinical disease management, and the promotion of precision health equity. Mr. Yusuf Henriques, COO Dr. Wilmot Allen, and CSO Dr. Bradford Wilson formed IndyGeneUS. The company, which is owned by African Americans and Africans, has offices in Washington, DC, and a presence in South Africa through the Founder Institute. The Founder Institute is the world's largest pre-seed startup accelerator.

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INDUSTRIAL IMPACT

Legend Biotech Announces Extension of PDUFA Date for Cilta-Cel

Cilta-cel | November 01, 2021

Legend Biotech Corporation (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, today announced that the U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) target date for ciltacabtagene autoleucel (cilta-cel) to February 28, 2022. Cilta-cel is a BCMA-directed chimeric antigen receptor T cell (CAR-T) therapy being investigated for the treatment of adults with relapsed and/or refractory multiple myeloma. The Biologics License Application (BLA) was submitted by Legend Biotech’s collaboration partner Janssen Biotech, Inc. “We are working closely with Janssen and the FDA to facilitate an efficient and thorough review of the BLA for cilta-cel,Financial Officer at Legend Biotech.We remain confident that cilta-cel has shown great promise in patients with relapsed and refractory multiple myeloma, and we are focused on making this therapy available to them in the US as soon as possible.” Ying Huang, Ph.D., Chief Executive Officer and Chief The FDA notified Janssen on October 28, 2021 of the extension of the PDUFA date to allow sufficient time to review information recently submitted pertaining to an updated analytical method following an FDA information request. Legend and Janssen met with the FDA on November 1. No additional clinical data have been requested. About Cilta-cel Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the U.S. and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel. In December 2020, Legend announced initiation of rolling submission of Biologics License Application to the FDA seeking approval of cilta-cel for the treatment of relapsed and/or refractory multiple Myeloma, which was accepted under Priority Review in May 2021. Cilta-cel was previously granted Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, and Orphan Drug Designation in February 2019. About Multiple Myeloma Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.1 Although treatment may result in remission, unfortunately, patients will most likely relapse.2 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.3 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.4,5 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.6 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.7 About Legend Biotech Legend Biotech is a global, clinical-stage biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogenic chimeric antigen receptor T-cell, T-cell receptor (TCR-T), and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of safe, efficacious and cutting-edge therapeutics for patients worldwide. We are currently engaged in a strategic collaboration to develop and commercialize our lead product candidate, ciltacabtagene autoleucel, an investigational BCMA-targeted CAR-T cell therapy for patients living with multiple myeloma. Applications seeking approval of cilta-cel for the treatment of patients with RRMM are currently under regulatory review by several health authorities around the world, including the U.S. Food and Drug Administration and the European Medicines Agency. Cautionary Note Regarding Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the timing and outcome of regulatory reviews relating to cilta-cel, including the BLA being reviewed by the FDA. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Legend Biotech’s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products; unexpected clinical trial results, including as a result of additional analysis of existing clinical data or unexpected new clinical data; unexpected regulatory actions or delays, including requests for additional safety and/or efficacy data or analysis of data, or government regulation generally; as well as the other factors discussed in the “Risk Factors” section of Legend Biotech’s Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 2, 2021. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in this presentation as anticipated, believed, estimated or expected. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

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MEDICAL

Palatin Announces Initiation of Pivotal Phase 3 PL9643 MELODY-1 Clinical Trial in Patients with Dry Eye Disease

Palatin Technologies, Inc | December 29, 2021

Palatin Technologies, Inc. a specialized biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin peptide receptor system, announced the initiation of the pivotal Phase 3 MELODY-1 clinical study of PL9643 in patients with dry eye disease. The study is designed to enroll up to 400 patients at multiple U.S. sites, and top-line results are currently expected second half calendar 2022. "We are excited to initiate the MELODY-1 study of PL9643 in patients suffering from dry eye disease. This is the second melanocortin peptide Palatin has advanced into Phase 3, demonstrating Palatin's expertise and efficiency in melanocortin peptide development for indications with unmet medical need." Carl Spana, Ph.D., CEO and President of Palatin The pivotal Phase 3 MELODY-1 clinical study is a multi-center, randomized, double–masked and vehicle–controlled study evaluating the safety and efficacy of the melanocortin agonist, PL9643 ophthalmic solution, compared to vehicle in subjects with dry eye disease . The study design is based on positive Phase 2 results of PL9643 for the treatment of dry eye disease, and an end-of-phase 2 meeting with the with the U.S. Food and Drug Administration, reaching agreement on all key elements of a pivotal Phase 3 clinical program, including study design, endpoints, interim assessment, and patient population. In addition, to support a New Drug Application filing, a second phase 3 study and long-term safety study will be required. If the program progresses as planned, top-line results from MELODY-1 would be available second half calendar 2022, MELODY-2 data read out second half calendar 2023, with a potential NDA submission first half calendar 2024. "Progressing PL9643 into Phase 3 is a significant milestone for the Company and patients with DED," said Michael Raizman, MD, Chief Medical Officer of Palatin. "PL9643, with its differentiating factors, could provide a more tolerable and effective treatment option for patients suffering from dry eye disease." About Dry Eye Disease Dry eye disease is a common inflammatory disease that, left untreated, can become extremely painful and lead to permanent damage to the cornea and vision. Dry eye disease affects the cornea and conjunctiva of the eye resulting in irritation, redness, pain, and blurred vision. It is estimated to affect over 20 million people in the United States. The disease is characterized by insufficient moisture and lubrication in the anterior surface of the eye, leading to dryness, inflammation, pain, discomfort, irritation, diminished quality of life, and in severe cases, permanent vision impairment. Existing therapy for dry eye disease is generally regarded as inadequate by many physicians and patients, and often requires week or months to demonstrate activity. About Melanocortin Receptor Agonists and Inflammation The melanocortin receptor system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. There are five melanocortin receptors, MC1r through MC5r. Modulation of these receptors, through use of receptor-specific agonists, which activate receptor function, or receptor-specific antagonists, which block receptor function, can have medically significant pharmacological effects. Many tissues and immune cells located in the eye express melanocortin receptors, empowering our opportunity to directly activate natural pathways to resolve disease inflammation. About Palatin Palatin is a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin and natriuretic peptide receptor systems, with targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. Palatin's strategy is to develop products and then form marketing collaborations with industry leaders to maximize their commercial potential.

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