Category Archives: Gene Medicine

RenaCARE study expected to be fully enrolled by end of Q2 of 2022

AUSTIN, Texas, April 26, 2022 /PRNewswire/ -- Natera, Inc.(NASDAQ: NTRA), a global leader in cell-free DNA testing, today announced the RenaCARE (Renasight Clinical Application, Review and Evaluation) study - a real world, prospective, multi-center clinical study to assess the clinical utility of the Renasight genetic testing panel. The study has already enrolled 1,600 patients across 25 sites, representing leading academic and private nephrology clinics in the U.S., and will enroll up to 2,000 patients. It is expected to complete enrollment in the second quarter of 2022 with a publication expected to be submitted in late 2022.

The study aims to demonstrate how genetic findings impact the management of patient care and examines diagnostic outcomes of patients tested with the Renasight genetic testing panel. In addition, the study will assess patient satisfaction, health knowledge and genetic literacy. This study follows a 2019 publication1 in The New England Journal of Medicine (NEJM) showing that 89% of patients who tested positive with a multi-gene genetic test had actionable clinical implications.

"Chronic kidney disease affects more than 10% of the global population, and our 2019 NEJM study showed roughly a 10% genetic yield among CKD patients," said Ali Gharavi, M.D., chief of the Division of Nephrology at New York-Presbyterian/Columbia University Irving Medical Center, director of the Center for Precision Medicine and Genomics in the Department of Medicine, interim director of the Institute of Genomic Medicine at Columbia University Vagelos College of Physicians and Surgeons, the study's principal investigator and a close collaborator with Kidney Disease: Improving Global Outcomes foundation (KDIGO) and the National Kidney Foundation (NKF). "We're optimistic that this study will show that next-generation sequencing (NGS) multi-gene assays can be used in a real world setting, to inform and guide disease management and help improve patient outcomes."

"We're confident that the RenaCARE study will confirm the high clinical utility shown in prior studies. This will be an important addition to the growing body of evidence showing the value of genetic testing to clarify an undifferentiated diagnosis, identify a genetic subtype within a diagnosis, reclassify a diagnosis, or provide insights for genetic counseling, family planning and clinical trial access," said Hossein Tabriziani, M.D., senior medical director of organ health for Natera.

All patients undergoing testing using the Renasight panel are offered optional pre- and post-test genetic information sessions with a genetic counselor in addition to their test results. Similarly, providers have access to Natera's genetic counselors for questions about the Renasight testing panel and review of test results.

Natera designed and launched the Renasight genetic testing panel with the feedback of general nephrologists, pediatric nephrologists, and transplant nephrologists. Natera has performed over 10,000 Renasight tests to date.

About Renasight

The Renasight test is a germline genetic test that screens for hereditary causes of kidney disease. It is indicated for patients with diagnosed kidney disease and is run from a patient's blood or saliva sample. Providers can use the Renasight test to identify a genetic predisposition, clarify a clinical diagnosis, or identify the etiology of an unknown kidney disease to help inform medical management. Additionally, genetic counseling and familial testing can be offered based on the test result. The test has been developed and its performance characteristics determined by the CLIA-certified laboratory performing the test. The test has not been cleared or approved by the U.S. Food and Drug Administration (FDA). CAP accredited, ISO 13485 certified, and CLIA certified.

About Natera

Natera is a global leader in cell-free DNA testing, dedicated to oncology, women's health, and organ health. Our aim is to make personalized genetic testing and diagnostics part of the standard of care to protect health and enable earlier and more targeted interventions that help lead to longer, healthier lives. Natera's tests are validated by more than 100 peer-reviewed publications that demonstrate high accuracy. Natera operates ISO 13485-certified and CAP-accredited laboratories certified under the Clinical Laboratory Improvement Amendments (CLIA) in Austin, Texas and San Carlos, California. For more information, visit http://www.natera.com.

Forward-Looking Statements

All statements other than statements of historical facts contained in this press release are forward-looking statements and are not a representation that Natera's plans, estimates, or expectations will be achieved. These forward-looking statements represent Natera's expectations as of the date of this press release, and Natera disclaims any obligation to update the forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual results to differ materially, including with respect to whether the results of clinical or other studies will support the use of our product offerings, our expectations of the reliability, accuracy and performance of our screening tests, or of the benefits of our screening tests and product offerings to patients, providers and payers. Additional risks and uncertainties are discussed in greater detail in "Risk Factors" in Natera's recent filings on Forms 10-K and 10-Q and in other filings Natera makes with the SEC from time to time. These documents are available atwww.natera.com/investorsandwww.sec.gov.

ContactsInvestor Relations:Mike Brophy, CFO, Natera, Inc., 510-826-2350Media:Kate Stabrawa, Communications, Natera, Inc., 720-318-4080pr@natera.com

References

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Natera Announces Definitive Study to Evaluate the Clinical Utility of Renasight in the Diagnosis and Management of Chronic Kidney Disease (CKD) -...

This therapy, using sustained release of nitric oxide, may be a novel, efficient and safe way to prevent and treat multiple metabolic diseases.

Jeonga Kim, Ph.D.A novel therapy developed at the University of Alabama at Birmingham ameliorates obesity and Type 2 diabetes in mice fed a high-fat diet. The therapy acts through sustained release of nitric oxide, a gaseous signaling chemical whose most important function in the body is relaxing the inner muscles of blood vessels.

Because reduced bioavailability of nitric oxide is the hallmark of cardiometabolic syndrome, supplying exogenous nitric oxide at a sustained level may be an efficient way of treating the cardiometabolic syndrome, said Jeonga Kim, Ph.D., leader of the UAB study. The strategy of reducing body weight by the local delivery of nitric oxide may be a novel, efficient and safe way to prevent and treat multiple metabolic diseases.

This study, published in the journal ACS Applied Materials & Interfaces, used an ingenious self-assembling, nanomatrix gel capable of releasing a burst of nitric oxide in the first 24 hours, followed by sustained nitric oxide release for four weeks. The gel was developed by UAB researchers Ho-Wook Jun, Ph.D., and Brigitta Brott, M.D., and it is licensed through the UAB Harbert Institute for Innovation and Entrepreneurship by their UAB spinoff company, Endomimetics LLC.

The gel was injected subcutaneously into 8-week-old mice every two weeks for 12 weeks. Gel-injected mice and control mice were fed a high-fat diet, known to induce obesity and insulin resistance.

At the end of 12 weeks, the nitric oxide-mice had gained 17 percent less body weight, compared to controls, and that weight difference was due mainly to decreased fat, not lean mass or water content. The researchers saw increased phosphorylation of the enzyme hormone-sensitive lipase and a reduction in the size of fat cells in epididymal white adipose tissue, or eWAT. Increased lipolysis may explain the reduced body weight, Kim says.

The nitric oxide-mice also showed improved glucose tolerance, and decreases in fasting serum insulin and leptin levels.

Kim and colleagues found wide-ranging changes in measures of inflammation and metabolism in the nitric-oxide mice, compared to controls. The expression of four inflammatory genes, including a marker for macrophages, was reduced in eWAT.

The nitric oxide gel also appeared to stimulate the browning of adipose tissue, through increased gene expression of uncoupled protein 1 in brown adipose tissue and beige adipose tissue. Nitric oxide is known to increase mitochondrial biogenesis, a mechanism for the conversion of white adipocytes to beige adipocytes. Brown adipose tissue, or brown fat, produces heat to maintain body temperature in cold conditions. The fat cells in brown adipose tissue and in inguinal adipose tissue from the nitric oxide-mice were also smaller than cells from controls.

The nitric oxide gel also protected against non-alcoholic fatty liver disease, as seen by lower liver weight, reduced triglycerides in the liver, and reduced triglycerides and cholesterol in blood serum. The nitric oxide gel also improved insulin sensitivity, as measured by increased expression of five insulin-signaling molecules in skeletal muscle, liver or eWAT.

The mice that received the nitric oxide gel also had improved cerebral blood flow, and they showed significantly improved spatial learning ability, as measured by the Morris water maze test. It is unknown whether those changes were a direct effect of nitric oxide or were mediated through the neuroprotective effects of adipocyte beiging.

Co-authors with Kim, Jun and Brott in the study, Subcutaneous administration of nitric oxide-releasing nanomatrix gel ameliorates obesity and insulin resistance in high fat diet-induced obese mice, are Guang Ren and Sushant Bhatnagar, UAB Department of Medicine, Division of Endocrinology, Diabetes and Metabolism; Patrick Tae Joon Hwang and Reid Millican, Endomimetics, LLC, Birmingham, Alabama; Juhee Shin, UAB Department of Biomedical Engineering; Brigitta C. Brott and Martin E. Young, UAB Department of Medicine, Division of Cardiovascular Disease; and Thomas van Groen and Craig M. Powell, UAB Department of Neurobiology.

At UAB, Kim is an associate professor in the UAB Department of Medicine, Division of Endocrinology, Diabetes and Metabolism, and she is a scientist in the UAB Comprehensive Diabetes Center.

Support came from UAB and from National Institutes of Health grants DK079626, HL128695, HL163802, AG058078, DK95975-03, DK120684-01, DK109789 and NS047466.

The departments of Medicine and Neurobiology are in the Marnix E. Heersink School of Medicine at UAB. Biomedical Engineering is a joint department of the Heersink School of Medicine and the UAB School of Engineering.

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A novel therapy ameliorates obesity and Type 2 diabetes in mice fed a high-fat diet - University of Alabama at Birmingham

MORGANTOWN, W.Va. -- A new data-driven mechanistic approach that predicts cell types within tissue will help to reduce drug costs and treat diseases that were difficult to develop drugs for, according to a West Virginia University scientist.

David Klinke, professor in the Department of Chemical and Biomedical Engineering, developed and tested a mechanistic approach to predict the number and function of different cell types within a particular tissue and how they change when a malignant (cancerous) cell acquires the ability to secrete a protein.

Ultimately, we want to develop drugs that broaden the clinical benefit of immunotherapies, said Klinke, whos also an adjunct assistant professor in the WVU School of Medicine and member of the Cancer Institute.

Mechanistic models have been created by hand by experts, but there are gaps in researchers understanding of biology because 90% of research publications focus on only 20% of genes in humans.

Research from this study, published in Nature Communications, sifts through large datasets to predict how secretion of one gene product by a malignant cell influences other cell types within a tissue directly from the data. This provides a complement to the hand-created models that play important roles in drug development.

Under normal conditions, ones immune system defends against infectious disease, Klinke said. However, most cancers arise through an evolutionary process of mutation and selection. Every cell has the blueprint in its DNA to make every gene product. In that process of mutation and selection, re-expression of some of these gene products may provide malignant cells with the ability to suppress immune response.

Human tissues are made up of specialized cell types that are organized to maintain function in a changing environment. Ultimately, the functional orientation of cell types within a tissue interact to create a heterocellular network -- a network of many different cell types that interact to collectively achieve a goal. A heterocellular network is important to create and maintain tissue equilibrium.

While researchers know that tissue equilibrium is disrupted during oncogenesis, or the development of a tumor, there is no clear understanding of how genetic alterations influence the heterocellular network within human tissues.

Klinke said one of the barriers for broadening clinical benefit is that malignant cells create environments that suppress host immunity.

This new data-driven approach allows researchers to predict how a gene product secreted by a malignant cell changes the prevalence and functional orientation of other cell types within a human tissue.

Klinke said that studying how one event causes another is challenging to do in systems where it's difficult for researchers to see what is happening like within an intact human tissue.

To test their predictions, using digital cytometry and Bayesian network inference, Klinke and his team examined immunocompetent mouse models of cancer. With this approach, Klinke was able to predict how a protein secreted by malignant cells alters the heterocellular network in the context of melanoma and breast cancer.

Digital cytometry, which is the measurement of the number and characteristics of cells, and Bayesian network (a probabilistic graphical model) inference were used because there are datasets available with these models that contain sequenced homogenized (similar) tumor tissue.

We can change the expression of a gene and then see whether the prevalence and functional orientation of different cell types in the tumor changes similarly as predicted by the Bayesian network model.

Klinke said the conventional approach to predict the functional orientation of cell types is to change the expression of a secreted protein and then quantify different cell types using different experimental approaches.

For this study, Klinke used mechanistic modeling to represent the mechanisms that support the biology and predict scenarios using simulation instead of actually testing the scenario in humans.

These models are highly complicated but let me use a simple analogy, Klinke said. Say that we want to hit a target using an artillery shell and we have only one shot. Given our understanding of the laws of physics, we know that we need to know a few things about the projectile and all the forces acting on the projectile. Given this information, we can simulate with a computer that if we fire the projectile in a certain direction or angle, it will land in a certain location.

Similarly, we know a lot about the underlying biology associated with a drug, but there are also some things that we dont know, and we cant test everything in humans. Given common conversations in the media about the high price of drugs, testing new drugs in humans is expensive and the vast majority of new drugs tested dont work.

Klinke said that one of the ways that mechanistic modeling and simulation can help is by providing a way to bring all the different pieces of understanding together in the same context.

If there are key aspects missing, we run simulations to see if targeting some aspect of the biology with a drug makes sense. Mechanistic modeling and simulation have had an impact on a number of other industries, and this is now being applied to drug development.

Klinke hopes that this research can be used in other contexts like cancers or immunologic diseases.

Ultimately, we all care that when we get sick, there are treatments that can improve our health and not bankrupt us in the process. Like many other industries, the pharma industry is turning increasingly to mechanistic modeling and simulation to better prioritize potential targets and reduce the time to clinic. Collectively, this will help reduce drug costs and help treat diseases that were difficult to develop drugs for.

Citation: Data-driven learning how oncogenic gene expression locally alters heterocellular networks

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WVU researcher develops data-driven approach to help reduce drug costs and treat diseases - West Virginia University

In 2011, Ashley Van Zeeland, a researcher with a recent PhD in neuroscience, was working as a fellow at the Scripps Research Translational Institute when she met a young woman with a mysterious illness. The girls parents were frightened and felt isolated: They didnt know if their daughter would surviveand if she did, what kind of life she could expect. Van Zeeland was part of the team that sequenced the familys DNA and, utilizing a new technology in the field of informatics, identified a new gene that was responsible for the girls rare disorder. It was a pivotal moment for the parents, who found peace of mind knowing their daughter could expect a full life and join the small community of young survivors of the same illness.

It was a pivotal moment for Van Zeeland as well. Seeing knowledge and technology come together to change that familys life cemented her desire to further expand the enormous potential of genomics. Her path led her to Illumina, the San Diego-based biotech company with a mission to improve human health by unlocking the power of the genome. Their transformative work has earned Illumina a spot on Fast Companys list of the worlds Most Innovative Companies.

Illumina has been innovating genetic sequencing for more than 20 years. When the company introduced its first sequencer in 2006, the cost to sequence a single genome was $300,000. Today, that cost can be as low as $600.

Illuminas advances in genetic sequencing are reshaping medicine. Genetic sequencing has now allowed cancer patients to find treatment options specific to their tumors, doctors to anticipate adverse drug reactions and personalize medicine, public health agencies around the world to track and detect new COVID variants, and patients with rare diseases to more easily identify and treat their illnesses. The next wave of innovation includes nucleic medicine, gene- and cell-based therapies, and early diagnostic tests.

Our next moonshot is the $100 genome, a huge goal that really rallies everybody together and turns the innovation dials all the way up, says Van Zeeland, who is now Illuminas VP and head of Illumina Open Innovation. Our open innovation and philanthropic efforts will make sure these transformative innovations are actually getting out and changing lives.

Through Illumina Open Innovation, Illumina is investing in new startups and partnering in research efforts to develop solutions in areas such as rapid sequencing and data security. To really unlock the power of the genome is something we cant do alone, Van Zeeland says. Illumina Open Innovation is all about creating opportunities and structures to invite innovation in and work collaboratively to drive even farther on what this technology can do.

At the same time, Illuminas philanthropic arm is working to increase access to the technology. Illumina worked with the African Union, Africa CDC, and the Bill & Melinda Gates Foundation to help establish the Africa Pathogen Genomics Initiative (PGI). In April 2021, Illumina committed $60 million in sequencing capabilities to a global pathogen genomics initiative, which expands the Africa PGI model globally. It is invigorating to be part of this incredible brain trust with so many diverse skillsets working to unlock the potential of genomics, Van Zeeland says. I know that the next five years are going to be twice as impactful as the past 10. And that is exciting.

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How collaboration is driving the next wave of genomics - Fast Company

SOMERVILLE, Mass.--(BUSINESS WIRE)--Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as GENE WRITING technology, announced today that it has raised over $300 million in Series C financing. Investors included a wholly-owned subsidiary of the Abu Dhabi Investment Authority (ADIA); Alaska Permanent Fund Corporation; Altitude Life Science Ventures; ARTIS Ventures; Cormorant Asset Management; Tesseras founder, Flagship Pioneering; Hanwha Impact Partners; Longevity Vision Fund; March Capital; SALT Fund; SoftBank Vision Fund 2; funds and accounts advised by T. Rowe Price Associates, Inc., and others including all of Tesseras existing institutional shareholders.

We are thankful for the support from our new partners and existing investors alike in this latest funding round. It is our belief that genetic medicine will be the most important next epoch in medicineoffering the ability to cure genetic diseases and to someday even prevent disease from occurring, said Geoffrey von Maltzahn, Ph.D., Co-Founder, Chief Executive Officer, and Board Director of Tessera Therapeutics. Todays announcement will help us realize the promise of GENE WRITING technology and our mission of curing disease by writing in the code of life.

While there have been many advancements in the area of genetic medicine over the past decade, Tesseras GENE WRITING platform is charting an entirely new courseone that aims to revolutionize genetic medicine as we know it, said Noubar Afeyan, Ph.D., Co-Founder and Chairman of Tessera Therapeutics and Founder and CEO of Flagship Pioneering. This latest funding round is a testament to the immense potential of this bioplatform to provide new treatments and cures to previously untreatable genetic diseases.

Tesseras GENE WRITING technology is designed to cure disease by writing in the code of life. The GENE WRITING platform can change any base pair to any other, make small insertions or deletions, and write entire genes into the genome with delivery of only RNA. This unlocks the potential to cure nearly any genetic disease, create life-changing medicines for other serious conditions such as cancer, and prevent illnesses with curative, scalable, and easily administered genetic medicines that could become a new modality in human healthcare.

GENE WRITING technology is inspired by natures genome architectsMobile Genetic Elements (MGEs)to overcome the limitations of gene editing and gene therapies. MGEs evolved to write new sequences of DNA into the genome, in contrast to nucleases like CRISPR which evolved to destroy DNA. Therefore, MGEs offer the potential for immense impact in genetic medicine by writing short and long therapeutic sequences of DNA into human cells. Tessera has designed, built, and tested tens of thousands of engineered and synthetic MGEs to create programmable GENE WRITING systems that can write and rewrite the genome with high-efficiency, specificity, and fidelity.

The Tessera team has developed a remarkable GENE WRITING platform that confers unique advantages over alternative genetic medicine technologies, including base editing, CRISPR, and gene therapies, said Vali Barsan, M.D., of SoftBank Investment Advisers. Having worked closely with the company over the past year, its hugely exciting to observe GENE WRITING catalyze a new category of genetic medicine.

For more information on Tessera Therapeutics, including how GENE WRITING technology works, partnership opportunities, and job openings, please visit http://www.tesseratherapeutics.com.

About Tesseras GENE WRITER Technology

Tesseras GENE WRITER tools are based on natures genome architects, Mobile Genetic Elements (MGEs)the most abundant class of genes across the tree of life, representing approximately half of the human genome. Tessera has evaluated tens of thousands of natural and synthetic MGEs to create GENE WRITER candidates with the ability to write therapeutic messages into the human genome. Tesseras research engine further optimizes the discovered GENE WRITER candidates for efficiency, specificity, and fidelityessentially compressing eons of evolution into a few months.

About Tessera Therapeutics

Tessera Therapeutics is pioneering GENE WRITING technology, which consists of multiple technology platforms designed to offer scientists and clinicians the ability to write therapeutic messages into the human genome, thereby curing diseases at their source. The GENE WRITING platform allows the correction of single nucleotides, the deletion or insertion of short sequences of DNA, and the writing of entire genes into the genome, offering the potential for a new category of genetic medicines with broad applications both in vivo and ex vivo. Tessera Therapeutics was founded by Flagship Pioneering in 2018, a life sciences innovation enterprise that conceives, resources, and develops first-in-category companies to transform human health and sustainability. For more information about Tessera, please visit http://www.tesseratherapeutics.com.

About Flagship Pioneering

Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in more than $140 billion in aggregate value. To date, Flagship has deployed over $2.6 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 42 transformative companies, including Axcella Therapeutics (NASDAQ: AXLA), Codiak Biosciences (NASDAQ: CDAK) Denali Therapeutics (NASDAQ: DNLI), Evelo Biosciences (NASDAQ: EVLO), Foghorn Therapeutics (NASDAQ: FHTX), Indigo Ag, Moderna (NASDAQ: MRNA), Omega Therapeutics (NASDAQ: OMGA), Rubius Therapeutics (NASDAQ: RUBY), Sana Biotechnology (NASDAQ: SANA), Seres Therapeutics (NASDAQ: MCRB), and Sigilon Therapeutics (NASDAQ: SGTX).

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Tessera Therapeutics Announces Over $300M Series C Financing to Advance its GENE WRITING Platform - Business Wire

LONDON, April 25, 2022 -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead gene therapy obecabatagene autoleucel (obe-cel), a CD19-directed autologous chimeric antigen receptor (CAR) T therapy that is being investigated in the ongoing FELIX Phase 2 study of adult relapsed / refractory B-Acute Lymphocytic Leukemia (ALL).

The FDA grants RMAT designation to drug candidates in recognition of the therapy's potential to address significant unmet medical needs in patients with serious or life-threatening conditions. Similar to Breakthrough Therapy designation, RMAT designation provides a number of important benefits in the drug development process, including early interactions with the FDA and other actions to expedite development and review.

"We are very proud to have received RMAT designation, which is an important regulatory milestone for obe-cel," said Dr. Christian Itin, Chief Executive Officer of Autolus. "This supports our belief that obe-cel has the potential to address a serious unmet medical need. It will enable us to further optimize our development and regulatory process and desire to bring this potentially curative therapy to patients as quickly as possible."

obe-cel has previously been granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) and Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom.

# # #

About Autolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the Company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information, please visit http://www.autolus.com.

About obe-cel (AUTO1)

Obe-cel is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, obe-cel may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells. In collaboration with Autolus' academic partner, UCL, obe-cel is currently being evaluated in a Phase 1 clinical trialsfor B-NHL. Autolus has progressed obe-cel to the FELIX trial, a potential pivotal trial for adult ALL.

About obe-celFELIX clinical trial

Autolus' FELIX Phase 1b/2 clinical trial of obe-cel is enrolling adult patients with relapsed / refractory B-precursor ALL. The trial had a Phase 1b component prior to proceeding to the single arm, Phase 2 clinical trial. The primary endpoint is overall response rate, and the secondary endpoints include duration of response, MRD negative CR rate and safety. The trial is designed to enroll approximately 140 patients across 34 of the leading academic and non-academic centers in the United States, United Kingdom and Europe. [NCT04404660]

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus' development of the obe-cel program; the future clinical development, efficacy, safety and therapeutic potential of its product candidates, including progress, expectations as to the reporting of data, conduct and timing and potential future clinical activity and milestones; expectations regarding the initiation, design and reporting of data from clinical trials; expectations regarding regulatory approval process for any product candidates; the collaboration between Autolus and Blackstone; the discovery, development and potential commercialization of potential product candidates including obe-cel using Autolus' technology and under the collaboration agreement; the therapeutic potential for Autolus in next generation product developments of obe-cel in B-cell malignancies; the potential and timing to receive milestone payments and pay royalties under the strategic collaboration; and the Company's anticipated cash runway. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in such statements. These risks and uncertainties include, but are not limited to, the risks that Autolus' preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the results of early clinical trials are not always being predictive of future results; the cost, timing and results of clinical trials; that many product candidates do not become approved drugs on a timely or cost effective basis or at all; the ability to enroll patients in clinical trials; possible safety and efficacy concerns; and the impact of the ongoing COVID-19 pandemic on Autolus' business. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Autolus' actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission on March 10, 2022, as well as discussions of potential risks, uncertainties, and other important factors in Autolus' subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Autolus undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Contact:

Olivia Manser

+44 (0) 7780 471568

o.manser@autolus.com

Julia Wilson

+44 (0) 7818 430877

j.wilson@autolus.com

Susan A. Noonan

S.A. Noonan Communications

+1-917-513-5303

susan@sanoonan.com

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Syncona : FDA Grants Regenerative Medicine Advanced Therapy (RMAT) designation to Autolus' CAR T cell therapy, obe-cel, for the treatment of adult...

Rowan Callahan, Elias Spiliotopoulos,and Thuy Ngo, Ph.D, inside their lab at OHSU Knight Cancer Research Building on Friday, April 22, 2022. (OHSU/Christine Torres Hicks)

Oregon Health & Science University scientists have devised an accurate and sensitive way to test blood to see if a pre-cancerous condition is escalating to outright cancer potentially enabling treatment early in tumor development when cancer is more likely to be curable.

The OHSU researchers focused on two common conditions that can precede highly lethal cancers: liver cirrhosis, which boosts the risk of liver cancer, and a precursor to the blood cancer multiple myeloma called MGUS, which stands for monoclonal gammopathy of undetermined significance.

If the findings hold up in larger clinical studies, they set the stage for a simple, affordable method to screen people at risk by taking a small blood sample a few times a year.

Our biomarker may recapitulate the transition to cancer, says Thuy Ngo, Ph.D., who led the research. Ngo is an assistant professor of molecular and medical genetics in the OHSU School of Medicine and a member of CEDAR, the OHSU Knight Cancer Institutes Cancer Early Detection Advanced Research Center.

Ngos team reported the findingstoday in the journal NPJ Precision Oncology.

Many competing groups are developing blood tests for cancer early detection, but most are focused on finding cancers in a general population. Ngo and coauthors say there is an unmet need for a blood test that can identify patients with pre-malignant conditions who require further intervention due to a higher likelihood of cancer incidence. In the case of cirrhosis and MGUS, some will progress to cancer, but many others will not. There currently is no reliable way to predict which cases will progress.

The blood test devised by Ngo and colleagues looks for molecules called messenger RNA, which convey instructions from genes. Each RNA molecule is built of chemical units arranged in a single strand like letters of an alphabet that spell out the message of a gene.

RNA routinely escape from cells and circulate in the blood, but unprotected, the free molecules quickly degrade; this makes it technically challenging to read the sequences of a mass of cell-free RNA collected from blood and make sense of it.

A few years ago, not many people believed cell-free messenger RNA could be reliably detected in the blood because it is prone to degradation, Ngo says. We found a way to handle it, and we are among the first to apply it in cancer and pre-cancer early detection.

In the new research, the team took blood samples from eight people with liver cancer, 10 with multiple myeloma, four with liver cirrhosis, eight with MGUS, and 20 non-cancer donors. They painstakingly sequenced the RNA from the samples to obtain a view of gene activity represented by the messenger molecules recovered from blood.

From this, they were able to identify patterns of gene activity in the different groups of people and build models to distinguish those with cancer from those with pre-malignant conditions, and from those without cancer.

The models were able to distinguish multiple myeloma blood samples from non-cancer samples with 90% accuracy, and multiple myeloma from its pre-malignant condition with 100% accuracy. They were able to distinguish liver cancer samples from non-cancer with 93 to 100% accuracy, and liver cancer from cirrhosis with 100% accuracy.

Experiments showed that the level of the cell-free RNA biomarkers displayed a gradual transition from non-cancerous states through pre-cancerous conditions and cancer. The researchers validated the biomarkers using a set of subjects different from the ones used to develop the models, and they proved highly accurate at distinguishing cancer from non-cancer.

This is promising, but the sample set is small, so we need to be modest here, Ngo cautions. Its still very early stage and further clinical validation will be needed.

The research lays the foundation for developing inexpensive assays that measure levels of cell-free RNA in blood for a small panel of genes that can differentiate cancer from pre-malignant conditions.You dont need any fancy equipment for doing this, Ngo says. Any central lab with standard equipment can do the work. Sophisticated sequencing was only required for the discovery work of identifying the telltale gene signatures.

Ngo and OHSU have filed a patent on their findings, and Ngo says she is talking with a company about a licensing deal to develop the technology. We have several directions to move it forward, she says.

Ngos priority is people at risk for liver cancer. One-quarter to one-third of adults in the U.S. have fatty liver disease, making them vulnerable to cirrhosis and liver cancer. Across the globe, infectious hepatitis puts enormous numbers of people in danger of developing liver cancer. This is why, Ngo says, the world needs a simple, affordable test to identify those at highest risk and monitor their health for the earliest signs of malignant growth.

This research was supported by grants from Oregon Health & Science University (CEDAR3250918), Cancer Research UK/OHSU (C63763/A27122), OCTRI (UL1TR000128), Kuni Foundation, Department of Defense (W81XWH2110853) and Susan G. Komen Foundation (CCR21663959).

Oregon Health & Science University has filed patent applications based on this work. The authors report no other competing interests.

Link:
Blood test could reveal transition to cancer in people at risk - OHSU News

Scientists from the United Kingdom may have identified several more environmental causes for cancer after evaluating the genetic data of over 12,000 cancer patients from National Health Servicefiles.

In arguably thelargest studyof its kind, researchers at Cambridge University Hospitals and the University of Cambridgelooked into whole-genome sequencing data of 12,222 patients to detect possible patterns in the DNA of cancer patients and succeeded. They found 58 new "mutational signatures," or patterns that offer clues on whether a particular patient had been exposed to certain environmental elements which led to cancer. These included cellular malfunctions, smoking and exposure to UV light.

Data were taken from the NHS's 100,000 Genomes Project, Genomics England's massivegenome sequencing projectbased on data from more than 85,000 patients diagnosed with cancer or rare diseases.

The research,publishedinScience, featured a new method the Signature Fit Multi-Step (FitMS) algorithm to enhance discrimination of common mutational processes from the rare, lower-frequency mutagenic processes. FitMS looks at signatures from new samples and compares these with existing findings by monitoring commonalities and identifying additional rare signatures.

The set of reference signatures used was identified after comparing and contrasting independent tissue-specific signatures and clustering mutational signatures from various tissues that may be due to similar processes. Other mutation bases were also linked to past clinical and treatment histories, where applicable.

"Whole genome sequencing gives us a total picture of all the mutations that have contributed to each person's cancer. With thousands of mutations per cancer, we have unprecedented power to look for commonalities and differences across NHS patients, and in doing so we uncovered 58 new mutational signatures and broadened our knowledge of cancer," said Dr. Andrea Degasperi, the study's first author and a research associate at the University of Cambridge, in a statement.

"The reason it is important to identify mutational signatures is because they are like fingerprints at a crime scene - they help to pinpoint cancer culprits. Some mutational signatures have clinical or treatment implications - they can highlight abnormalities that may be targeted with specific drugs or may indicate a potential 'Achilles heel' in individual cancers," added Serena Nik-Zainal, leader of the study and a professor of genomic medicine and bioinformatics at the University of Cambridge.

Cancer is a complex beast and severalstudies on mutationsalso came to light at the American Association for Cancer Research's annual meeting in mid-April 2022. Aadi Biosciencegot the U.S. Food and Drug Administration's green light for Fyarro, a new drug designed to treat PEComa, a very rare type of sarcoma. Fyarro has demonstrated the ability to treat patients with TSC1 or TSC2 mutations.

Ikena Oncologyalso presented data on an ongoing trial for IK-930. This TEAD inhibitor targets the Hippo signaling pathway and binds to TEAD transcription factors with the goal of preventing the transcription of genes that drive the progression of cancer.

Read the rest here:
UK Researchers Uncover 58 New Mutational Signatures of Cancer - BioSpace

A team of US researchers have found a way to diagnose skin cancer using blood tests.

The researchers have shown in a lab-based study that melanoma cells can be detected in blood and plasma. If the test makes it through clinical trials, the researchers hope that it could one day be used to sidestep the invasive biopsies that are currently required to diagnose melanoma.

The test uses melanoma-specific antibodies, and a device designed specifically to react them with blood. The device is called MelanoBean, and it works with microfluidics: manipulating tiny amounts of fluid to do interesting things that they wouldnt do in larger volumes.

The test is described in a paper in Advanced NanoBiomed Research.

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This is the first comprehensive study of circulating tumour cells or CTCs to evaluate the efficacy of surgery using microfluidic systems in melanoma, including changes in the number of CTCs, CTC cluster configuration, and gene expression profiling, says first author Dr Yoon-Tae Kang, a researcher at the University of Michigan, US.

Read more: New cancer detection technique using old oranges

The researchers found that with their test, melanoma cells (CTCs) could be found in the blood of cancer patients at all stages of the disease I through to IV.

It could also identify whether any CTCs were hanging around in the blood of patients whod had skin cancer surgery to get their cells removed.

CTCs have the potential to pinpoint treatment resistance and recurrence, and can be a valuable biomarker to non-invasively monitor for disease progression, says corresponding author Dr Sunitha Nagrath also from the University of Michican.

Theres never been a more important time to explain the facts, cherish evidence-based knowledge and to showcase the latest scientific, technological and engineering breakthroughs. Cosmos is published by The Royal Institution of Australia, a charity dedicated to connecting people with the world of science. Financial contributions, however big or small, help us provide access to trusted science information at a time when the world needs it most. Please support us by making a donation or purchasing a subscription today.

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Detecting skin cancer with a blood test - Cosmos

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HER2 negative Breast Cancer Pipeline Insights | Research Report 2022 by DelveInsight - GlobeNewswire