Category Archives: Genetic Engineering

SAN RAFAEL, Calif. and SHANGHAI, Dec. 16, 2021 /PRNewswire/ -- 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, today announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases.

BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

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.

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"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.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com. Information on such website is not incorporated by reference into this press release.

About Skyline Therapeutics

Skyline Therapeutics is a fully integrated gene and cell therapy company dedicated to the discovery, development and delivery of innovative therapies. Established in 2019, Skyline Therapeutics has built a proprietary AAV-based gene therapy platform that integrates novel capsid engineering and vector design, analytical and process development, and state-of-the-art GMP manufacturing capabilities that support large scale clinical-grade vector production. The Skyline team of world-class experts and leaders in science, technology and business brings industry-leading know-how and is advancing a pipeline of diversified programs that address multiple diseases including ocular, neurological, metabolic and blood disorders. Skyline Therapeutics is also broadening its therapeutic expertise to cover more disease areas with high unmet need such as cardiovascular disorders through strategic partnerships. Headquartered in China, Skyline Therapeutics currently has research, development and manufacturing capabilities in Shanghai and Hangzhou. http://www.skytx.com

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: expectations related to the multi-year global strategic collaboration with Skyline for the discovery, development and commercialization of AAV gene therapies for dilated cardiomyopathy and pre-specified payments to Skyline for R&D, regulatory and commercial milestones, and the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. Additional important factors to be considered in connection with forward-looking statements are detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

References

Givertz MM, Mann DL. Epidemiology and natural history of recovery of left ventricular function in recent onset dilated cardiomyopathies. Curr Heart Fail Rep. 2013;10(4):321-330. doi:10.1007/s11897-013-0157-5

Bozkurt B, Colvin M, Cook J, et al. Current Diagnostic and Treatment Strategies for Specific Dilated Cardiomyopathies: A Scientific Statement From the American Heart Association [published correction appears in Circulation. 2016 Dec 6;134(23 ):e652]. Circulation. 2016;134(23):e579-e646. doi:10.1161/CIR.0000000000000455

Prevalence of rare diseases: Bibliographic data, Orphanet Report Series, Rare Diseases collection, January 2021, Number 1: Diseases listed in alphabetical order. http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_diseases.pdf

Miura K, Nakagawa H, Morikawa Y, et al. Epidemiology of idiopathic cardiomyopathy in Japan: results from a nationwide survey. Heart. 2002;87(2):126-130. doi:10.1136/heart.87.2.126

Huang GY, Gao H, Meng XG. Epidemiology and etiology of dilated cardiomyopathy. Chinese Journal of Prevention and Control of Chronic Diseases. 2009;17(1):42-44.

Jing Zhong, Li-Ping Li, Jian-Feng Zhou and Yong-He Ding (November 5th 2020). Genetic Determinant of Familial Dilated Cardiomyopathy and Genotype-Targeted Therapeutic Strategy, Cardiac Diseases - Novel Aspects of Cardiac Risk, Cardiorenal Pathology and Cardiac Interventions, David C. Gaze and Aleksandar Kibel, IntechOpen, DOI: 10.5772/intechopen.94434. Available from: https://www.intechopen.com/chapters/73939

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

Felisa Feng

Skyline Therapeutics

felisa.feng@skytx.com

Skyline Therapeutics

Cision

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SOURCE BioMarin Pharmaceutical Inc.

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BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases - Yahoo Finance

There is a possibility that the COVID-19 virus was "most likely" leaked from a lab in China,British MPs were informed on Thursday.

Harvard scientist Dr Alina Chan, while testifyingbefore the House of Commons Science and Technology Committee, said that it was reasonable to hypothesise and investigate whether the Covid virus was modified in the lab.

The first cases were linked to a seafood market in Wuhan, which is located acrossthe Yangtze riverwhere a virology lab is located.

When asked by the panel about the probability of a lab leak as the origin of the pandemic, Chan said a lab origin is more likely than natural origin at this point.

We all agree that there was a critical event at the Huanan Seafood Market, that was a superspreader event caused by humans. There is no evidence pointing to a natural animal origin of the virus at that market, she noted.

Speaking on the possibility of thevirus being engineered in the lab before a leak, Chan said: "We have heard from many top virologists that a genetically engineered origin of this virus is reasonable...and this includes virologists who themselves have made modifications to the first SARS virus."

On the question of her level of confidence that the world would eventually be able to establish the true origins of COVID-19, Chan said it was simply a matter of time.

"Right now it's not safe for people who know about the origin of this pandemic to come forward. It might be five years from now, it might be 50 years from now, but we live in an era where there's so much data being collected and stored...we just need a credible, systematic investigation," she said.

Also read | Scientists working on Covid origin notice timeline discrepancies; 1st case was a vendor at Wuhan market

"We know now that this virus has a very unique feature, called the furin cleavage site that makes it the pandemic pathogen it is. So, without this feature, there's no way this would be causing this pandemic."

"Only recently in September did a proposal get leaked showing that scientists from the EcoHealth Alliance were in collaboration with the Wuhan Institute of Virology developing this pipeline for inserting novel furin cleavage sites, these genetic modifications, into SARS-like viruses in the lab," Chan said.

According to Tory lord Lord Ridley, who has co-authored a book on the virus's origins with Dr Chan, the fact that experts have still not located the animal host that would support a natural origin despite two years of investigation, supports the notion that COVID-19 emerged from a lab.Recent discoveries of China's attempted cover-up have compelled British and US intelligence agencies to investigate the lab-leak idea seriously, which was previously regarded as a lunatic conspiracy theory.

Watch | Gravitas: Lab leak "most likely" source of Covid-19, British MPs told

The explosive charges, on the other hand, are expected to put pressure on the UK government to question Beijing's assertions that the virus originated in nature.

They're also expected to raise more questions about Chinese President Xi Jinping's connection with the WHO, which was accused last year of issuing a whitewash report on the outbreak.

(With input from agencies)

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Reasonable to hypothesise Covid virus is genetically engineered: Expert - WION

An international, multidisciplinary team of researchers from theTranslational Synthetic Biology Laboratoryat Pompeu Fabra University (Barcelona, Spain), led by Dr.Marc Gell, has published an article in the scientific journalNature Communicationsshowing the potential of Find Cut-and-Transfer (FiCAT) technology as a state-of-the-art tool forgene writingto develop advanced therapies that are safer and more effective in their future clinical application in patients withgenetic and oncological diseasesthat have few treatment options.

The UPF Translational Synthetic Biology Laboratory has been working on gene editing and synthetic biology applied to gene therapies since 2017. FiCAT technology is an important scientific breakthrough to overcome the current limitations of the technology used today for genome editing and gene therapy.

Human genome engineering has significantly progressed in the last decade with the development of new editing tools, but there was still a technology gap that would allow therapeutic genes to be transferred efficiently with few size limitations, comments Dr. Marc Gell, supervisor of the study.

In this work, the researchers develop an efficient and precise programmable gene writing technology based on the combination of modified proteins CRISPR-cas and piggy Bac transposase (PB), succeeding in inserting small and large fragments. Dr.Maria Pallars, co-first author of the study explains that: CRISPR stands out for its precision when editing small fragments. However, transposases allow us to insert large fragments but in an uncontrolled manner. We have combined the best of each technology.

In this way, FiCAT technology allows us to precisely insert large fragments of DNA into the genome. This means we can develop therapeutic solutions to diseases that currently have no treatment, such as Duchenne muscular dystrophy, or some cases of hereditary blindness, in which the affected gene is large in size, asserts Dr.Avencia Snchez-Mejas, a senior researcher with the group and co-supervisor of the work.

They tested the technology in human and mouse cell lines achieving efficiencies of 522% with minimal off-target insertions and have demonstrated on-target gene transfer in vivo in mouse liver and germline cells in mouse models. Lastly, they performed a directed evolution of FiCAT and further improved efficiency by 25-30%. We have been progressively modifying enzymes so that they acquire the function we were looking for, selecting the ones that displayed a better function, detailsDimitrije Ivani, co-first author of the article. Our work is a clear example that enzyme engineering in the context of genome editing has great potential, he concludes.

UPF has transferred FiCAT technology via the spinoffIntegra Therapeutics, founded in 2020 by the researchers Marc Gell and Avencia Snchez-Mejas, seeking to get this scientific knowledge and technological capacity to reach the biopharmaceutical industry to develop safe and efficient advanced technologies that reach patients. Recently, Integra Tx has obtained 4.5 million euros in funding from Advent France Biotechnology (France), Invivo Capital (Spain) and Takeda Ventures (USA).

The work published in Nature Communications was carried out with funding from the Societal challenges AEI, AGAUR- PRODUCTE, UPGRADE-Horizon 2020 of the European Commission; la Caixa CaixaImpulse Validate and CaixaImpulse Consolidate programmes; the Ramn Areces Foundation; and the Ramn y Cajal programme of the Spanish Ministry of Economy, Industry and Competitiveness.

Reference:Pallars-Masmitj M, Ivani D, Mir-Pedrol J, et al. Find and cut-and-transfer (FiCAT) mammalian genome engineering. Nat Commun. 2021;12(1):7071. doi: 10.1038/s41467-021-27183-x

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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New Gene-Writing Tool Helps To Develop Advanced Gene Therapies - Technology Networks

NEW YORKand DUBAI, United Arab Emirates, Dec. 15, 2021 /PRNewswire/ --Secondcell Bioand Alliance Care Technologies Internationaland announce new strategic partnership using Chromovert Technology to establish a novel, cutting-edge research focused on cell technology and rapid drug discovery to combat rare genetic diseases in the United Arab Emirates.

Chromovert has been extensively validated to streamline a key step of the drug discovery process by a boutique biotechnology company co-founded by its inventor, Dr. Kambiz Shekdar, PhD and the late Nobel-laureate Dr. Gunter Blobel, MD, PhD of The Rockefeller University. The present initiative aims to make revolutionary advancements by scaling-up and applying the demonstrated biotechnology to implement drug discovery at scale, with targets including CNS indications, COVID/Pulmonary Edema (ENaC), orphan and rare diseases and Thalassemia.

In the pharmaceutical drug discovery and development process, Chromovert is a cutting-edge technology that goes beyond CRISPR by enabling the creation of previously out-of-reach designer cells for improved drug discovery. The novel technology was published for the first time in March 2021 by Springer Nature: Cell engineering method using fluorogenic oligonucleotide signaling probes and flow cytometry.Founded by Dr. Shekdar,Secondcell Biobrings optimized materials and methods to operate Chromovert Technology to market.

According to Dr. Shekdar, "Our technology can have a global impact on genetics and cell research, particularly in the area of rare genetic diseases drug discovery. At this stage we want to partner with companies that are positioned to move quickly to advance current research toward finding cures. We are excited at the prospect of joining the healthcare revolution taking place in the UAE and having the opportunity to work with the best researchers from across the globe."

Alliance Care Technologies International CEO Michele Tarnow echoes those sentiments. "We have the privilege to work with the UAE's leading hospitals and research centers. By helping to bring Secondcell Bio and Dr. Shekdar's research to the UAE, we help the Emirates achieve their goal of becoming the leader in global health care by 2030. We are excited to work alongside Dr. Shekdar to bring this cutting-edge research to the UAE."

As part of the expansion into the UAE, a world class Scientific Advisory Board will be appointed to identify and prioritize additional therapeutic indications. Dr. Shekdar notes, "As whole genome sequencing of individuals and analysis by AI increases, additional drug targets will be identified, including for personalized medicine. By strategically locating in the UAE, we enhance data collection in that region and can prioritize drug discovery against new and relevant targets."

Tarnow notes additional advantages to working with Secondcell Bio and Chromovert. "In addition to the promising research advances that can be achieved, the advanced technology used to develop S4, our Syndromic Surveillance solution, has the potential to enhance Secondcell Bio data collection. We are excited to explore ways we can collaborate to improve health care across multiple sectors."

Previously, Chromovert was used to create exceedingly rare laboratory cells that accurately mimic the biology of human pain perception, resulting in the discovery and development of a novel clinical-stage pain blocker fast-tracked by the FDA and presented at The White House as part of Governor Chris Christie's panel on the opioid epidemic. The biotechnology was also the subject of a $100M+ natural flavors discovery engine implemented with The Coca-Cola Company, Kraft Foods and Nestle to create human tastebud-like cells for miniaturized turbo taste testing of natural extracts to discover exquisite natural flavors to cut sodium and sugar in the diet.

Demonstrating the breadth and range of technology, Drs. Shekdar and Blobel also established Research Foundation to Cure AIDS (RFTCA) based upon Chromovert. RFTCA is a U.S. 501(c)3 not-for-profit organization seeking to develop a global cure for AIDS by using Chromovert to increase the efficiency of promising, but stunted, investigational cell therapy strategies to cure HIV infection. Sickle Cell Disease, Thalassemia and certain other diseases also stand to benefit by the addition of Chromovert Technology.

The strategic partnership by ACTI and Secondcell aims to realize the full potential of the technology to discover and develop a pipeline of candidate drugs for diseases with unmet need. Currently, the industry average failure rate for drug discovery programs in pharmaceutical companies is reported to be approximately 98%. One key factor contributing to the high failure rate is the lack of cells that accurately mimic the biology of human disease in the laboratory dish. Chromovert Technology has been demonstrated to permit the rapid creation of laboratory cells that accurately mimic human disease, allowing for the improved discovery of candidate drug molecules with a greater overall likelihood of success.

ACTI and Secondcell Bio invite researchers, entrepreneurs and investors interested in learning more about current research, collaborations or investment opportunities to contact for more information.

About Secondcell Bio and Chromovert Technology

Secondcell Bio is a biotechnology company established to achieve previously inaccessible goals in cell and genetic engineering via dedicated research and commercialization partnerships.

Chromovert Technology enables creation of highly desired but previously out-of-reach cells for improved drug discovery. The technology was invented by Dr. Kambiz Shekdar in the Nobel-Prize winning laboratory of the late Gunter Blobel, MD, PhD, at The Rockefeller University. Materials and methods to operate Chromovert are available for research use only from Secondcell Bio at https://www.secondcellbio.com/.

Link to detailed scientific publication on Chromovert Technology:https://link.springer.com/article/10.1007/s10529-021-03101-5

About Alliance Care Technologies International

Alliance Care Technologies International (ACTI) is driven by a mission to provide global access to quality healthcare. At Alliance Care Technologies International, we believe global health is improved when physicians, patients, researchers and entrepreneurs work together to improve quality and efficiency across the continuum of healthcare.

Alliance Care Technologies International develops and deploys advanced technology and artificial intelligence solutions that solve today's most pressing healthcare challenges. We work with industry leaders to design individualized solutions that enhance the patient experience, streamline administrative processes, manage costs, and ultimately, improve healthcare outcomes.

https://www.alliancecaretech.com/

Link to detailed press on Secondcell Bio: https://www.prnewswire.com/news-releases/secondcell-bio-publishes-biotechnology-for-improved-drug-discovery-originating-from-the-rockefeller-university-301377258.html

Contact InformationSecondcell Bio[emailprotected]

SOURCE Secondcell Bio

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DRUG DISCOVERY PARTNERSHIP ANNOUNCED IN UAE USA - PRNewswire

PHILADELPHIA, Dec. 17, 2021 (GLOBE NEWSWIRE) -- Century Therapeutics (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology, today announced it has been selected for addition to the NASDAQ Biotechnology Index (NASDAQ: NBI), effective as of market open on Monday, December 20, 2021.

The NBI is designed to track the performance of a set of securities listed on the NASDAQ Stock Market® (NASDAQ®), made up of NASDAQ-listed companies classified as Biotechnology & Pharmaceuticals by the Industry Classification Benchmark (ICB), and is widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. To be selected for addition to the NBI, a company must have a minimum market capitalization of $200 million; demonstrate an average daily trading volume of at least 100,000 shares; and must be Nasdaq-listed. The index is modified market capitalization-weighted such that constituents are capped at 8% (for the top 5) and at 4% (for the remaining) at each quarterly index rebalance; the entire index is reviewed and reconstituted annually in December.

For more information about the NASDAQ Biotechnology Index visit http://www.nasdaq.com.

About Century Therapeutics

Century Therapeutics, Inc. (NASDAQ: IPSC) is harnessing the power of adult stem cells to develop curative cell therapy products for cancer that we believe will allow us to overcome the limitations of first-generation cell therapies. Our genetically engineered, iPSC-derived iNK and iT cell product candidates are designed to specifically target hematologic and solid tumor cancers. We are leveraging our expertise in cellular reprogramming, genetic engineering, and manufacturing to develop therapies with the potential to overcome many of the challenges inherent to cell therapy and provide a significant advantage over existing cell therapy technologies. We believe our commitment to developing off-the-shelf cell therapies will expand patient access and provide an unparalleled opportunity to advance the course of cancer care. For more information on Century Therapeutics please visit http://www.centurytx.com.

Century Therapeutics Forward-Looking Statement

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, The Private Securities Litigation Reform Act of 1995. All statements contained in this press release, other than statements of historical facts or statements that relate to present facts or current conditions, including but not limited to, statements regarding our clinical timelines. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as may,” might,” will,” should,” expect,” plan,” aim,” seek,” anticipate,” could,” intend,” target,” project,” contemplate,” believe,” estimate,” predict,” forecast,” potential” or continue” or the negative of these terms or other similar expressions. The forward-looking statements in this presentation are only predictions. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition, and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond our control, including, among others: our ability to successfully advance our current and future product candidates through development activities, preclinical studies, and clinical trials; our reliance on the maintenance of certain key collaborative relationships for the manufacturing and development of our product candidates; the timing, scope and likelihood of regulatory filings and approvals, including final regulatory approval of our product candidates; the impact of the COVID-19 pandemic on our business and operations; the performance of third parties in connection with the development of our product candidates, including third parties conducting our future clinical trials as well as third-party suppliers and manufacturers; our ability to successfully commercialize our product candidates and develop sales and marketing capabilities, if our product candidates are approved; and our ability to maintain and successfully enforce adequate intellectual property protection. These and other risks and uncertainties are described more fully in the Risk Factors” section of our most recent filings with the Securities and Exchange Commission and available at http://www.sec.gov. You should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. Moreover, we operate in a dynamic industry and economy. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that we may face. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

For More Information: Company: Elizabeth Krutoholow investor.relations@centurytx.com Investors: Melissa Forst/Maghan Meyers century@argotpartners.com Media: Joshua R. Mansbach century@argotpartners.com

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Century Therapeutics Added to the NASDAQ Biotechnology Index - Stockhouse

As 2021 draws to a close, many are looking forward to a well-deserved winter break. Its a great time to cozy up next to the fire with a book, so we asked University of Chicago scholars and staff what they would recommend reading.

The list includes books that weave together many different threads of human experiencefamily, history, science, government, nature and moreand one book that explores an intelligent robots experience of the human world.From the possibility of transformative justiceto the prospect of nature rebounding after a crisis, there is something to match every sensibility.

Recommended by: Alex Ji, Assistant Professor of Astronomy and Astrophysics

This science fiction novel tells the story of how an artificial intelligence navigates a human world. Klara is an Artificial Friend, a solar-powered robot built to provide companionship to children. I was quickly transfixed by the style of Klaras first-person narration of the daily patterns of human and robot life, which provides an interesting perspective on both the potential and limitations of artificial intelligence. As the novel progresses, Klara's unique lens is trained on deeper questions regarding childhood illness, genetic engineering, social class, and what it means to love.

Recommended by: M. Todd Henderson, Michael J. Marks Professor of Law

Eels are delicious. Thats about all I knew. This book opened my eyes to one of the worlds most fascinating creatures. I just couldnt wait to tell my wife facts after every reading. (She wasnt as keen on hearing them, especially the third or fourth time.) But you wont be able to help yourself either. Aristotle makes an appearance, as does Freud and countless others who have tried to understand eels. No one has. No one has ever seen one breed and no one really understands them. The author alternates between chapters about eels and memoirs of he and his father fishing for eels in Sweden. The factual chapters are hugely interesting; the personal ones are deeply moving, especially for fathers with sons. Youll like both types of chapters, even if you dont like eels or dont have sons.

(Read the full list of UChicago Law School professor recommendations here.)

Recommended by: Jen Kennedy, Director of Student Centers

We Do This Til We Free Us is a collection of interviews, articles, and a variety of previously released works by Mariame Kaba that leads the reader through an argument for the abolition of the prison-industrial complex. Kaba, an activist and educator with roots in New York City and Chicago, dives deep into the details of transformative justice and the shift in thought this would require as a society. The works are thoughtful, broad ranging, and never shy away from discussion of the gaps in current organizing. Its a slim read but a great primer for those looking to learn more. As Mariame says, Hope is a discipline.

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What UChicago faculty and staff want you to read over winter break - UChicago News

GAITHERSBURG, Md., Dec. 15, 2021 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced the submission of a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for its COVID-19 vaccine by its partner Takeda Pharmaceutical Company Limited (Takeda). Novavax' recombinant nanoparticle COVID-19 vaccine with Matrix-M adjuvant, known as TAK-019 in Japan and NVX-CoV2373 outside Japan, is the first protein-based COVID-19 vaccine to be submitted under an NDA in Japan.

With the support of the MHLW, the companies are working to establish the capability to manufacture TAK-019 at Takeda's facilities in Japan and aim to begin distribution in early 2022, pending regulatory approval.

"Today's submission marks further progress in our quest to ensure broad global access to our protein-based COVID-19 vaccine," said Stanley C. Erck, President and Chief Executive Officer of Novavax. "Our partnership with Takeda reflects our shared commitment to tireless collaboration to deliver a COVID-19 vaccine, built on a well-understood vaccine platform."

The NDA submission includes an interim analysis from Takeda's ongoing Phase 1/2 immunogenicity and safety clinical trial of NVX-CoV2373/TAK-019 in Japan, in which the vaccine demonstrated a robust immune response and was well tolerated with no serious adverse events. Takeda submitted all available chemistry, manufacturing and controls (CMC), non-clinical and clinical data as of December 2021. Additional CMC data will be subsequently submitted to the Japan Pharmaceuticals and Medical Devices Agency (PMDA) during the NDA review period.

The application also includes safety and efficacy data from Novavax' two pivotal Phase 3 trials: PREVENT-19, which included 30,000 participants in the U.S. and Mexico and demonstrated 100% protection against moderate and severe disease, 93.2% efficacy against the predominantly circulating variants of concern and variants of interest, and 90.4% efficacy overall; and a trial of 15,000 participants in the U.K. that demonstrated efficacy of 96.4% against the original virus strain, 86.3% against the Alpha (B.1.1.7) variant and 89.7% efficacy overall. In both trials, NVX-CoV2373 demonstrated a reassuring safety and tolerability profile.

In addition to the submission of the NDA to MHLW in Japan, Novavax and Serum Institute of India Pvt. Ltd. (SII) recently received Emergency Use Authorization (EUA) for the vaccine in Indonesia and the Philippines, and the companies have filed for EUA in India and for Emergency Use Listing (EUL) with the World Health Organization (WHO). Novavax also announced regulatory filings for its vaccine in the United Kingdom, Australia, New Zealand, Canada, Singapore, United Arab Emirates, and the European Union and with the WHO. Additionally, Novavax and SK bioscience announced a Biologics License Application (BLA) submission to MFDS in South Korea. Novavax expects to submit its complete CMC data package to the U.S. FDA by the end of the year.

About NVX-CoV2373NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19.

Novavax' COVID-19 vaccine is packaged as a ready-to-use liquid formulation in a vial containing ten doses. The vaccination regimen calls for two 0.5 ml doses (5 mcg antigen and 50 mcg Matrix-M adjuvant) given intramuscularly 21 days apart. The vaccine is stored at 2- 8 Celsius, enabling the use of existing vaccine supply and cold chain channels.

About Matrix-M AdjuvantNovavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About NovavaxNovavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform harnesses the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. NVX-CoV2373, the company's COVID-19 vaccine, received Emergency Use Authorization in Indonesia and the Philippines and has been submitted for regulatory authorization in multiple markets globally. NanoFlu, the company's quadrivalent influenza nanoparticle vaccine, met all primary objectives in its pivotal Phase 3 clinical trial in older adults. Novavax is currently evaluating a COVID-NanoFlu combination vaccine in a Phase 1/2 clinical trial, which combines the company's NVX-CoV2373 and NanoFlu vaccine candidates. These vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visit http://www.novavax.com and connect with us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking StatementsStatements herein relating to the future of Novavax, its operating plans and prospects, its partnerships, the ongoing development of NVX-CoV2373, the scope, timing and outcome of future regulatory filings and actions, the capability to manufacture TAK-019 at Takeda's facilities in Japan and aim to begin distribution in early 2022, Novavax' quest to ensure broad global access of its protein-based COVID-19 vaccine, Novavax' plans to deliver a COVID-19 vaccine to people around the globe, Novavax' plan to supplement the CMC data submitted to the PMDA with additional CMC data, and Novavax' plans to submit a complete CMC data package to the U.S. FDA by the end of the year are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include challenges satisfying, alone or together with partners, various safety, efficacy, and product characterization requirements, including those related to process qualification and assay validation, necessary to satisfy applicable regulatory authorities; difficulty obtaining scarce raw materials and supplies; resource constraints, including human capital and manufacturing capacity, on the ability of Novavax to pursue planned regulatory pathways; challenges meeting contractual requirements under agreements with multiple commercial, governmental, and other entities; and those other risk factors identified in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Novavax' Annual Report on Form 10-K for the year ended December 31, 2020 and subsequent Quarterly Reports on Form 10-Q, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at http://www.sec.gov and http://www.novavax.com, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Contacts:

InvestorsNovavax, Inc.Erika Schultz | 240-268-2022ir@novavax.com

Solebury TroutAlexandra Roy | 617-221-9197aroy@soleburytrout.com

MediaAlison Chartan | 240-720-7804Laura Keenan Lindsey | 202-709-7521media@novavax.com

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SOURCE Novavax, Inc.

Company Codes: NASDAQ-NMS:NVAX

Link:
Novavax Announces Submission of New Drug Application in Japan for Approval of COVID-19 Vaccine - BioSpace

SAN DIEGO, Dec. 13, 2021 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, today reported interim results from its Phase 1/2 PRIME clinical trial of P-BCMA-101 for the treatment of relapsed/refractory multiple myeloma (R/R MM) at the 2021 American Society of Hematology (ASH) Annual Meeting.

The results show that P-BCMA-101, a non-viral transposon-based autologous CAR-T, was well tolerated and demonstrated strong anti-tumor activity in advanced, late line R/R MM patients. The learnings from P-BCMA-101 informed the development of the Company's first allogeneic program, P-BCMA-ALLO1 which is also being evaluated in R/R MM patients. The Company previously announced that it is winding down the P-BCMA-101 autologous program in favor of the allogeneic program, P-BCMA-ALLO1.

"We are encouraged by the outcomes seen from our clinical trial of P-BCMA-101, results that continue to validate our approach and that have informed P-BCMA-ALLO1, our first fully allogeneic CAR-T program for patients with multiple myeloma, as well as our other programs. Our focus is on creating differentiated product candidates with a high percentage of T stem cell memory (Tscm) cells," said Eric Ostertag, M.D., Ph.D., chief executive officer of Poseida Therapeutics. "Looking ahead, we continue to advance P-BCMA-ALLO1 and P-MUC1C-ALLO1 and look forward to presenting data in 2022 for both of these allogeneic programs."

The PRIME trial is a Phase 1/2, open label 3+3 single dose escalation of P-BCMA-101 CAR-T cells. The primary objective of the study is to determine the safety and maximum tolerated dose of P-BCMA-101 based on dose limiting toxicities (DLT), and the key secondary objective is to assess the anti-myeloma effect of the product. The median patient age was 62, with a median time since diagnosis of approximately 5.8 years. Patients were heavily pre-treated, with a median of 7 prior lines of therapy (2-18). As of the data cut-off date of October 15, 2021, a total of 98 patients have been dosed with P-BCMA-101.

The best observed treatment regimen was a combination with rituximab (n=14), with an overall response rate (ORR) of 78%, a VGPR/sCR rate of 43% and 100% overall survival at the time of the data cutoff. Progression free survival was also improved with rituximab, with median overall survival rates not yet reached in several cohorts including the rituximab combination cohorts. Response rates for other cohorts are consistent with results previously reported.

Across the study, no dose-limiting toxicities were observed. 28% of patients developed cytokine release syndrome (CRS) and 7% of patients developed neurotoxicity. None of the patients developed Grade 3 or higher CRS, and 2% of patients developed Grade 3 neurotoxicity. There were no treatment-related deaths among the patient population and no patients needed ICU admission as a result of CAR-T related toxicities. 28 patients were treated on a fully outpatient basis.

"P-BCMA-101 demonstrated strong anti-tumor activity in advanced multiple myeloma patients, and cohorts to date have shown minimal CRS and neurotoxicity, which allows for safe administration in an outpatient environment and combinations with other therapies," said Caitlin Costello, M.D., Associate Clinical Professor of Medicine and member of the Division of Blood and Marrow Transplantation at the University of California, San Diego. "These data indicate that the piggyBac transposon-based platform is an attractive option for allogeneic CAR-T cells, which has led to a first-in-human Phase 1 study."

The Company's first fully allogeneic CAR-T cell product, P-BCMA-ALLO1 utilizes Poseida's proprietary piggyBac DNA delivery system and Cas-CLOVER site-specific gene editing system to create an allogeneic product that prevents both graft-vs-host and host-vs-graft diseases and also incorporates a next-generation BCMA binder. P-BCMA-ALLO1 manufacturing involves a proprietary "booster" molecule that allows for numerous doses to be produced from a single manufacturing run, while maintaining desirable Tscm cells, which can reach percentages in the 60-80% range.

The Investigational New Drug (IND) application for P-BCMA-ALLO1 was given a safe to proceed designation by the FDA in August 2021. The Phase 1 study is an open label, dose escalation study following a 3+3 design of dose escalation in subjects with R/R MM. The study will assess the safety and maximum tolerated dose of P-BCMA-ALLO1 based on dose limiting toxicities. Key secondary objectives of the study include the anti-myeloma effect and safety of P-BCMA-ALLO1.

About Poseida Therapeutics, Inc.Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary genetic engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics. To learn more, visit http://www.poseida.com and connect with us on Twitter and LinkedIn.

Forward-Looking StatementStatements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential benefits of Poseida's technology platforms and product candidates, Poseida's plans and strategy with respect to developing its technologies and product candidates, and anticipated timelines and milestones with respect to Poseida's development programs and manufacturing activities. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Poseida's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry and the other risks described in Poseida's filings with the Securities and Exchange Commission. All forward-looking statement contained in this press release speak only as of the date on which they were made. Poseida undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

SOURCE Poseida Therapeutics, Inc.

http://www.poseida.com

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Poseida Therapeutics Provides Update on BCMA-Targeted CAR-T Clinical Trials at the 2021 American Society of Hematology (ASH) Annual Meeting -...

Figure 1. Leading researchers in the collaborative project. The full list of the co-authors in the Nature Communication paper: Zhong Guo, Oleh Smutok, Wayne A. Johnston, Patricia Walden, Jacobus P. J. Ungerer, Thomas S. Peat, Janet Newman, Jake Parker, Tom Nebl, Caryn Hepburn, Artem Melman, Richard J. Suderman, Evgeny Katz, Kirill Alexandrov.

The best and most efficient way to perform multi-disciplinary research is by doing it in collaboration. One of such research programs, including synthetic biology, materials science, bioelectrochemistry, bioelectronics, and biosensors, has been performed in a close collaboration between scientists at the Department of Chemistry and Biomolecule Science, Clarkson University, Dr. Oleh Smutok, Dr. Artem Melman (deceased on November 25, 2021), and Dr. Evgeny Katz, with a team of Australian scientists led by Dr. Kirill Alexandrov, Queensland University of Technology (Figure 1). This collaboration being active for several years has been supported with grants from Human Frontiers Science Program (HFSP) and US Department of Defense with the total funding over 1 million dollars. The results from the collaborative efforts have been published in numerous scientific papers and covered by several patents. The most recent and impressive publication was a paper in Nature Communications one of the top scientific journals (Impact Factor 14.92). The paper entitled Design of a methotrexate-controlled chemical dimerization system and its use in bio-electronic devices (Nature Commun. 2021, 12 article No. 7137) reports on a novel artificial enzyme produced by genetic engineering that can be activated with a drug (methotrexate) molecules. The artificial enzyme was immobilized at an electrode surface and used for the drug biosensing with extremely high sensitivity and specificity (Figure 2).

In addition to the fundamental novelty of using the artificial signal-activated enzyme, the study is highly relevant for practical biomedical application. Methotrexate is a toxic drug used in anti-cancer chemotherapy and its overdose has serious, life-threatening side effects. Thus, the methotrexate analysis in biological fluids is important for keeping the drug at the optimal concentration. The study opens the future options for biomedical applications of the developed biosensor and possibilities for other biosensing systems based on the same concept. It should be noted that the success of this project was based on the collaboration of scientists with expertise in different areas, synthetic biology, synthetic organic chemistry, and bioelectrochemistry. This is an exemplary collaboration that serves as a model of performing multi-disciplinary research. While the artificial enzyme preparation was carried out by the Australian team led by Dr. Alexandrov, the bioelectrochemical study of the developed biosensor was performed by Dr. Smutok at Clarkson University. Both US and Australian teams are continuing their successful work combining synthetic biology and bioelectronics and are expecting many more interesting and practically important results. The scientific efforts are combined with the education of graduate and undergraduate students participating in the project.

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Nature Communications paper published by two collaborating teams at Clarkson University (NY, USA) and Queensland University of Technology (Australia)...

The Company's manufacturing subsidiary, Expression Manufacturing LLC, provides end-to-end research and development capabilities and early phase and commercial scale manufacturing of both GMP cell and viral vector products. The facility manufactures lentiviral (LV) and adeno associated viral (AAV) vectors.

"With GMP vector manufacturing backlogs typically exceeding 18 months, we wanted to bring in-house manufacturing capabilities online and provide CDMO services to commercial clients as soon as possible. The opening of the facility allows us to immediately support our internal therapeutic pipeline in hematology and oncology. In fact, we just completed three full-scale engineering runs for our lead cell therapy product ET206, which is intended to treat Neuroblastoma," saidBill Swaney, President of Expression Manufacturing LLC.

Mr. Swaney is an internationally recognized expert in cell and gene therapy manufacturing and has conducted over 70 GMP production runs for academic and commercial clients. He comes to Expression Manufacturing from the Cincinnati Children's Hospital Medical Center where he was the Director of the Vector Production Facility & Viral Vector Core.

"The opening of our manufacturing facility signals our commitment to strengthen southwest Ohio's position as an emerging location for biotechnology while also rapidly advancing our internal therapeutic pipeline through clinical trials and commercialization," said Dr. Mohan Rao, Chief Executive Officer of Expression Therapeutics, Inc.

About Expression Therapeutics, Inc.

Expression Therapeutics, Inc. is a fully integrated clinical stage cell and gene therapy company developing novel therapeutics for deadly childhood and adult genetic diseases.

The Company's scientific breakthrough in transgene bioengineering enables it to optimize the gene therapy for each target indication:bleeding disorders,primary immunodeficiencies, andoncology. The oncology platformis based onproprietary non-signaling chimeric antigen receptor technologythat enhances the targeting capability of gamma delta T cells and alleviates concerns of long-term T cell ablation and tonic signaling to developallogeneic cell therapiesforneuroblastoma,T cell leukemia and lymphoma, andacute myeloid leukemia.

Ashley Walsh Director of Corporate Development Expression Therapeutics [emailprotected]

SOURCE Expression Therapeutics

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Expression Therapeutics Celebrates Opening of Cell and Gene Therapy Manufacturing Facility in Ohio - PRNewswire