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Matthews, Wise to receive ARA’s Gold Medal at 2023 Laureates … – Rice News

At the Association of Rice Alumnis 2023 Laureates Awards ceremony May 4, the group will bestow its highest honor the Gold Medal Award to Kathleen Shive Matthews and Scott W. Wise 71.

The annual ceremony will also honor several prominent alumni with the Distinguished Alumni Award and several members of the Rice community with the Meritorious Service Award.

Kathleen Shive Matthews

For nearly five decades at Rice, Matthews has set the highest standard for impactful research, mentorship and inclusive leadership. The Stewart Memorial Professor Emerita of BioSciences has dedicated her career to scientific discovery and expanding opportunities for women and minorities in STEM.

Matthews, who obtained a Bachelor of Science degree in chemistry from the University of Texas at Austin and a doctorate in biochemistry from the University of California, Berkeley, joined Rice in 1972 as a founding member of the biochemistry department, where she conducted groundbreaking research on protein-DNA interactions underlying gene regulation. Her contribution to the field for more than five decades is noteworthy, as evidenced by her 170-plus publications, her service on numerous scientific committees and boards, and her impressive awards from the National Institutes of Health, the National Science Foundation, the American Society for Biochemistry and Molecular Biology, and the American Association for the Advancement of Science, to name just a few.

A thoughtful and engaging leader, Matthews chaired the biochemistry department from 1987 to 1995 and served as dean of the Wiess School of Natural Sciences from 1998 to 2009. Simultaneously, she helped found the Keck Center for Computational Biology and the Gulf Coast Consortia, which support many successful graduate student and postdoctoral training and research programs in Houston. After her tenure as dean, she continued to instill a sense of wonder in her students and provide guidance to both young and seasoned faculty, including as a founding fellow for the Center for Teaching Excellence. Notably, she helped create the NSF-sponsored ADVANCE program to increase the pipeline, presence and promotion of women in science and was instrumental in securing permanent support with creation of the Office of Faculty Development. Upon her retirement, Matthews became Rices faculty ombuds, a position held by a retired faculty member and reporting directly to the president.

Throughout her career, Matthews has expressed her love for Rice in tireless service to the students, faculty and staff, as well as alumni and retirees. As one nominator noted, Dr. Kathleen S. Matthews is one of Rice Universitys finest treasures.

Scott W. Wise 71

Throughout his decades of service, including as Rices former chief investment officer and as alumni trustee, Wise has stewarded the university through a period of transformative growth and impact.

Wise earned a Bachelor of Arts degree in economics from Rice in 1971 and a Master in Accounting degree from the University of Texas at Austin in 1973. As a pitcher on the Rice baseball team, Wise was a four-year letterman and co-captain and posted the lowest earned run average in the Southwest Conference in 1970. During his 31-year career at Rice, including over 20 years as vice president for investments and treasurer at Rice, he was responsible for the management and oversight of all investment matters. From 2009-2010, he also served as the founding president of the Rice Management Co. By the end of his tenure, Rices endowment assets had grown from approximately $1 billion in 1989 to more than $4 billion. In 2010, Wise was recruited by TIAA to become founding president of Covariance Capital, an investment management company for nonprofit clients. For the last seven years, Wise has served as CEO and CIO for the Cullen Foundation, which supports health care, educational and civic initiatives in the Houston area, including at Rice.

Having previously served on the ARA Board of Directors, Wise became president of the ARA in July 2013 and later was selected as an alumni trustee. He has also served as a university associate of Will Rice College, a lecturer in the Jones School, a member of the Rice Owl Club board and a co-chair of his 50th class reunion. Wise chaired a number of search committees during his Rice tenure and recently served as a member of the presidential search committee that recommended to the Board of Trustees the appointment of Reginald DesRoches as Rices eighth president. Avid supporters of Rice Athletics and the arts, Wise and his wife, Geraldina Interiano Wise 83 85, are present at a myriad of Rice events and are enthusiastic ambassadors for the university.

In addition to Wises remarkable commitment to Rice, he is also active in the Houston community, sitting on the boards for the Houston Grand Opera Endowment, the Houston Symphony Endowment and several other institutions. As one nominator noted, Wise is a model for what graduates of Rice University should aspire to become.

Gilda Barabino 86

Barabino is a globally renowned bioengineering researcher and leader known for producing innovative health solutions and advancing engineering education. She received her Bachelor of Science degree in chemistry from Xavier University of Louisiana before becoming the first African American woman at Rice and just the fifth in the entire country to earn a doctorate in chemical engineering.

Barabino currently serves as the second president of Olin College of Engineering, where she is dedicated to educating the next generation of engineering innovators. Previously, she served as dean of the Grove School of Engineering at the City College of New York, where she developed programs to increase access to engineering education. She also held academic and administrative appointments at Emory University, the Georgia Institute of Technology and Northeastern University.

As an engineer, Barabino is known for integrating engineering innovation and entrepreneurship to solve medical challenges. Her research in the biomechanics of blood flow and thrombosis was foundational in the field, and her seminal research in sickle cell disease has informed current technologies and formed the basis for novel therapies. She is equally admired for promoting diversity and inclusion in STEM, as well as increasing the participation of minority women and men in entrepreneurial activities and interdisciplinary team science. Leading on a global stage, Barabino founded the National Institute for Faculty Equity and is a past president and chair of the board of the American Association for the Advancement of Science.

Barabino serves on multiple boards and advisory boards for universities across the United States, and she has received many of the highest honors in academia, including being elected to the AAAS, the National Academy of Engineering and the National Academy of Medicine.

Sandra K. Johnson 88

A pathbreaking engineer, leader and innovator, Johnson exemplifies the unconventional wisdom and entrepreneurial mindset that are advancing technology and bringing distinction to Rice.

Johnson earned her Bachelor of Science and Master of Science degrees, both in electrical engineering, from Southern University and Stanford University, respectively. In 1988, she became the first Black woman to earn a doctorate in electrical engineering from Rice. Upon graduating from Rice, Johnson began her tenure at IBM, serving for more than 25 years in various capacities, including as a researcher, a business development executive with assignments in the Middle East and Africa, and chief technology officer for IBM Central, East and West Africa. Her research and management responsibilities have ranged from Linux performance and WebSphere database development to Java server performance teams. Notably, she was part of the team that developed the prototype for IBMs Scalable Parallel Processor, the base machine for the world-famous Deep Blue chess-playing machine.

After retiring from IBM in 2014, Johnson became CEO of SKJ Visioneering LLC, a technology consulting company, and Global Mobile Finance Inc., a fintech startup company, both based in North Carolinas Research Triangle Park. A true trailblazer in her field, she has authored or co-authored over 80 publications and is a Master Inventor with over 40 issued and pending patents. Her numerous awards include being named an Electrical and Electronics Engineers Fellow, an Association for Computing Machinery Distinguished Engineer and a member of the IBM Academy of Technology, which consists of the top 1% of IBMs 250,000 technical professionals. Throughout her journey as an engineer, startup executive and author, Johnson has remained committed to mentoring and inspiring young people with her education, career and personal experiences.

Andy Karsner '89

Karsner is a leading conservationist, venture capitalist, innovation strategist, energy entrepreneur and diplomat. With more than three decades of commercial, philanthropic and public service leadership that touches every continent and spans every natural resource, he has helped accelerate and scale the worlds response to energy transition and climate change.

Karsner is a senior strategist at X (formerly Google X), the innovation lab of Alphabet Inc., and chairs Manifest Energy, a clean technology development and investment firm he founded in 2008. He is regularly called upon by world leaders and leading corporations in energy, natural resources, semiconductors and cloud computing for his experience and strategic insight.

From 2006 to 2008, Karsner served as U.S. Assistant Secretary of Energy, responsible for policy, regulation and Americas applied science National Labs and multibillion-dollar research, development and commercialization. More recently, he served as managing partner of Emerson Collective and co- founded Elemental, with Laurene Powell Jobs, as an investment and philanthropic platform dedicated to scalable, equitable market-based solutions for energy, food, water and climate. In 2017, he helped cofound Nia Tero a leading global NGO advocating for indigenous peoples.

Karsner holds a Bachelor of Arts degree with honors from Rice and a Master of Arts degree from Hong Kong University. Through all of his high-profile international accomplishments, including advising and serving in the governance of numerous universities, nonprofits and privately held companies, Karsner remains connected to Rice, advising the university on its energy and climate strategy and its distinct work to advance economics and material science. He was described as a force of nature by one nominator, and another described him as relentless in the pursuit of achieving consequential solutions the world requires to address climate change (and) security, and uplift the human condition.

Eric Nelson 83

Throughout his 30-plus-year career in the United States Foreign Service, Nelson has distinguished himself in service to his country and in his advocacy of democracy, equality and human rights.

Nelson earned a Bachelor of Science degree in chemical engineering from Rice in 1983 and an MBA from the University of Texas at Austin in 1988. After graduating from Rice, he volunteered for the Peace Corps in Liberia in West Africa, kicking off a long and dedicated career in public service that includes serving in Islamabad; Milan; Mexico City; Frankfurt, Germany; Santo Domingo, Dominican Republic; and Washington. He has held several senior management and diplomatic positions, including deputy chief of mission in Costa Rica, consul general in Munich and executive director at the Department of State, supporting three secretaries of state and accompanying them to 70 countries. From 2019 to 2022, he served as U.S. Ambassador to Bosnia and Herzegovina. Nelson is now the U.S. Ambassador in Residence at the George C. Marshall Center for Security Studies in Germany, guiding the institutes strategy to address regional, transnational and global challenges.

Throughout his award-winning career, Nelson has been a champion for peace and equality and social and economic progress. As a founding director of Gays and Lesbians in Foreign Affairs Agencies (GLIFAA), he championed fair treatment for LGBT foreign service employees. He has worked for the empowerment and well-being of others, including through his advocacy for a youth leadership program in Bosnia and Herzegovina and his promotion of innovative technology such as the monitoring of air quality by U.S. embassies abroad.

As one nominator described Nelson, his commitment to making our world a better place reflects well on who he is as a person and brings honor and distinction to the larger Rice community.

Ikhlas Sabouni 81 87

An accomplished architect, devoted teacher and pathbreaking leader in higher education, Sabouni exemplifies excellence in her decadeslong commitment to her profession and her students.

As dean of the School of Architecture at Prairie View A&M University, Sabouni has helped transform a small, unaccredited department of architecture that was within the College of Engineering and Architecture into a well-recognized, autonomous school. Sabouni, who was the first female dean of architecture in Texas among the states eight schools of architecture, has led the school through several reaffirmations of accreditation and increased its enrollment fourfold. She initiated new programs in construction science, community development and digital media arts, in addition to establishing two service and research centers in the School of Architecture.

A champion for diversity, equity and inclusion, Sabouni is devoted to promoting architecture as a career choice to the many students she has taught, supported, interacted with and ultimately inspired. A devoted teacher, she has established several scholarship endowments to support her students' financial needs and worked to ensure that students have job opportunities upon graduating.

Sabounis tremendous service to the architecture community extends to the local, regional and national levels. She is a member of multiple boards and has earned numerous awards. She received a Master of Architecture degree in 1981 and a doctorate of architecture degree in 1987 from Rice, where she won the William Dunlap Darden Award in Architecture for her outstanding doctoral dissertation.

As one nominator summarized, Sabouni has used the education, the cultivation of values, habits and attitudes to teach students, to administer in an increasingly effective and efficient manner, promote the architecture profession and serve the greater community in ways that only a dedicated architect can do.

Donald Bowers 91

Known widely for his wisdom, kindness, equanimity and thoughtful leadership, Bowers has demonstrated an extraordinary passion for service that continues to sustain Rices excellence while inspiring the next generation of Rice leaders.

A native Houstonian, Bowers earned his bachelor's degree in managerial studies from Rice and an MBA with a concentration in banking and finance from Sam Houston State University. He is vice president of the Federal Reserve Bank of Dallas, where he leads its Industry Outreach Program and serves as corporate secretary for the Houston Branch Board of Directors. A dedicated mentor, Bowers has served on the Center for Career Development Alumni Advisory Board and the Initiative for Students Commission, hosted numerous externships and internships, and led student leadership experiences through the Doerr Institute for New Leaders. As a former athlete, Bowers has remained a staunch supporter of the Rice football program, has mentored athletes through the Student Athlete Leadership Development Program and spearheaded efforts to establish the O.J. Brigance Courage Award.

Bowers commitment to Rice extends to the highest levels of alumni leadership, including as the president of the Association of Rice Alumni and in a variety of executive capacities in the Association of Rice University Black Alumni. Elected to the Rice Board of Trustees in 2018, he served the presidentially appointed Task Force on Slavery, Segregation and Racial Injustice, chaired the Building and Grounds Committee and led efforts in the redesign of the Academic Quadrangle and Founders Memorial. He also serves on the Baker Institute Board of Advisors and supports a range of philanthropic causes at Rice spanning scholarships, the Owl Club and the Black Student Association.

John R. Eldridge 75

An engaging alumnus, parent, career mentor and advocate of the humanities and the arts, Eldridges contributions to Rice are deep and span decades.

Eldridge earned his Bachelor of Arts degree in anthropology from Rice in 1975, before doing community service work and then graduating from the University of Texas School of Law in 1980. Over his exemplary 40-year career in environment law, he has won numerous accolades working for top international clients, while also managing his substantial pro bono and public service efforts. Eldridge was recently named a knight in Frances National Order of Merit for his brilliant career and efforts to grow relations between France and the United States.

Utilizing his professional strengths, he has for years chaired Rices Humanities Advisory Board, where he helped advocate for the upcoming Susan and Fayez Sarofim Hall for student arts. He has served diligently to advance the mission of Rices Center for Career Development, serving as chairman of its advisory board since 2016. He meets regularly with Rice students about careers, and his law firm Haynes Boone has hosted externships for many years.

Proud Rice parents of Graham 16 and Will 16, Eldridge and his wife, Annette, have been active leaders in Rices Parent Leadership Council, in addition to serving as community associates at Lovett College for over a decade. They routinely earn the award for best community associates, and Eldridge was vital to making Lovetts 50th anniversary celebration a success. Their considerable generosity extends to financial support spanning the humanities, the Rice Annual Fund, Fondren Library, fellowships and more. Eldridges nominators praised him as insightful and intelligent, but also so progressive and kind and applauded his tireless service and support.

Andrea Galindo

A dedicated staff member, volunteer and founding member of Rices Mariachi Luna Llena, Galindo has made incredible strides to raise cultural awareness not only of mariachi music but of Mexican culture throughout campus and the broader Houston community.

Galindo, who earned her bachelors and masters degrees in biology, is a pre-award specialist in Rices Office of Sponsored Research and Compliance, as well as an active associate at Wiess College, where she won Associate of the Year twice. Since Mariachi Luna Llenas founding in 2013, she has served as a lead singer and guitar player, helping cement the mariachi as a pillar of Rice culture on campus. Over her decade of involvement with the group, Galindo has performed at an array of public events, including at local schools, the Miller Outdoor Theater and the Houston Rodeo. She also sang the national anthem several times at professional sporting events, including Houston Astros and Rockets games.

Throughout the COVID-19 pandemic, Galindo spent considerable energy and effort to keep the mariachi going, organizing Zoom meetings, keeping up with current and past members, and, as one nominator noted, serving as the glue that has held together the mariachi family through the years. Additionally, she serves as an instructor for the Rice Salseros and is a familiar face at the weekly Spanish Tables at Wiess College.

In her extraordinary efforts to build up those around her, Galindo has fulfilled the ideals of Rice in cultivating a diverse and welcoming community of future researchers, artists and leaders.

Frank Jones 58 61

Jones, the Noah Harding Professor of Mathematics, has invested his entire academic and professional life in making Rice a better institution and Rice students better people. One nominator summarized Jones as a legend who has inspired countless generations of students through his decades of mathematics instruction at Rice.

After receiving his Bachelor of Arts degree in chemical engineering at the then-Rice Institute in 1958, Jones completed his Ph.D. in mathematics at Rice in 1961, winning the Sigma Xi Award for proficiency in research at the doctors degree level.

A member of the Rice faculty since 1962, Jones has received several awards for his commitment to excellent teaching. He received his first George R. Brown Award for Superior Teaching in 1968, then won again in 1972, 1973, 1978, 1985 and 1990. In 1980 and 1991, Jones received Rices highest teaching honor, the George R. Brown Prize for Excellence in Teaching. Additionally, he is a two-time winner of the Nicolas Salgo Distinguished Teacher Award, receiving it in 1977 and 1994. Jones was also named the 2007 Texas Professor of the Year by the Carnegie Foundation for the Advancement of Teaching and the Council for Advancement and Support of Education.

Joness extraordinary teaching ability is matched by his dedication to encouraging and mentoring the hundreds of students he has taught over the years. He welcomes the opportunity to interact with his students anytime they reach out to him, spending as much time as needed with each student, in person or virtually, to maximize their learning experience. As one nominator described Jones, he has the rare and wonderful combination of giving superb classes, with apparently effortless, beautiful and clear lectures, and caring deeply about his students as people.

Tamara Siler 87

Siler, who received her Bachelor of Arts degree in history from Rice, has dedicated her professional life to strengthening the Rice undergraduate community by recruiting, admitting and enrolling top scholars from historically excluded backgrounds.

As the deputy director of admission, access and inclusion at Rice, Siler serves as a senior leader in the Office of Admission and plays a vital role in mentoring staff in the fundamentals of the evaluation process at Rice and the importance of diversity and inclusion to the university. Recognized nationally as a thought leader in admissions, she is a sought-after speaker and has served as a former board member of the National Association for College Admission Counseling and a past president of the Texas Association for College Admission Counseling. She currently serves as a College and University Resource Committee member for International Baccalaureate-North America.

A consummate ambassador, Siler has recruited hundreds of faculty, staff and students to support on-campus programming, while engaging numerous Rice alumni in the effort to increase diversity at Rice. She supports many affinity groups, including the Association of Rice University Black Alumni and Rice Pride, where she helps organize the annual Pride Parade, and her devotion to her beloved Brown College is legendary.

Whenever she is not working passionately to move Rice forward, Siler is giving back to the community as a leading performer for local theater. As one nominator summarized, All these talents that she has cultivated over the years communicator, connector, singer and actor have contributed to making Rice an excellent and bold university. Her presence at Rice has been invaluable.

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Matthews, Wise to receive ARA's Gold Medal at 2023 Laureates ... - Rice News

GeneTether Therapeutics Inc. Announces Fiscal Year 2022 … – TheNewswire.ca

Vancouver, BC - TheNewswire - April 27, 2023 - GeneTether Therapeutics Inc., (together with its wholly-owned subsidiary GeneTether, Inc., GeneTether or the Company) (CSE:GTTX), an early-stage genetic medicine company focused on developing its disruptive proprietary platform technology to significantly increase the efficiency of DNA insertion into the genome, announced today the filing of its audited financial statements for the fiscal year ending December 31, 2022 and reported on corporate highlights from 2022. All dollar amounts are presented in the United States dollar, unless otherwise noted. Complete financial statements along with related management and discussion and analysis can be found in the System for Electronic Document Analysis and Retrieval, the electronic filing system for the disclosure documents of issuers across Canada, at http://www.SEDAR.com.

Commenting on the Companys progress, CEO Roland Boivin said, as previously announced, given the persistent weakness in the global capital markets, we have recently decided to significantly reduce the development of our GeneTether platform technology and conduct a review of strategic alternatives focused on maximizing shareholder value. Our disciplined approach to conserving cash and optimizing our cash runway position will serve us well as we embark on this crucial phase. Despite that, we have been able to make important progress that we believe will provide strategic advantages as we continue to explore our options. Finally, I would like to reiterate that our current cash position, which stood at $1,796,000 as of December 31, 2022 and our minimal cash burn rate, allow us the flexibility to explore investments in new technologies or add value to our GeneTether platform to increase the potential for M&A or collaboration opportunities.

R&D and Intellectual Property

Despite the challenging global markets conditions in 2022, GeneTether achieved R&D progress of its proprietary GeneTether platform:

In March 2023, the Company announced the results of a series of experiments conducted by a highly specialized contract research organization that confirmed the efficacy of its GeneTether technology in binding Lacl-Cas9 fusion proteins to template DNA. This validation specifically highlights the high level of template binding efficiency of the GeneTether platform, which is a crucial component of the Companys strategy to enhance gene editing efficiency. Further, cell-based experiments, conducted internally, as well as by third parties, confirmed that a GeneTether LacI-Cas9 fusion protein retains the ability to recognize and cut DNA when compared to unmodified Cas9. These studies were performed using several cell types and gene targets.

In October 2022, the Company announced pivoting RD efforts towards advancing its in-house programs versus outsourcing most activities, which would allow the Company to preserve cash and exert greater control over experimental designs and development protocols. Subsequently in February 2023, the Company announced the decision to scale back development of the GeneTether platform technology and explore strategic alternatives that will optimize shareholder value. This decision has been mainly driven by the current state of the global capital markets, which are experiencing persistent weakness.

On May 24, 2022, the United States Patent and Trademark Office granted patent #11,339,385 entitled Modified Nucleic Acid Editing Systems for Tethering Donor DNA related to its GeneTether platform technology. The claims cover a composition of matter for tethering a donor DNA template to a fusion protein of a nuclease and the lac repressor DNA binding domain, in particular the donor DNA includes the lac operator sequence. The patent term is scheduled to expire on July 14, 2040.

On May 11, 2022, a research paper co-authored by the Companys co-founder, Geoffrey Sargent, Ph.D. and titled Seamless Gene Correction in the Human Cystic Fibrosis Transmembrane Conductance Regulator Locus by Vector Replacement and Vector Insertion Events was published in the peer-reviewed journal Frontiers in Genome Editing. This publication describes novel HDR products and the subsequent derivation of seamless gene correction of the W1282X CFTR mutation in human induced pluripotent stem cells.

Completion of Initial Public Offering and Concurrent Private Placement (the Offering)

On March 29, 2022, the Company announced that it closed its Offering of Units at a price of C$0.60 per Unit for aggregate gross proceeds of C$4,500,000. Each Unit consists of one Common Share, and one Common Share purchase warrant (Warrant). Each Warrant entitles the holder to acquire an additional Common Share at an exercise price of C$0.72 until March 29, 2025. Including the compensation to the agent in connection with the Offering, the total estimated cash share issuance cost in connection with the Offering was approximately $600,000 (C$751,000).

Financial Results

The Companys total assets as at December 31, 2022 were approximately $1,944,000, including approximately $1,796,000 in cash. Net and comprehensive loss for the twelve months ended December 31, 2022 were approximately $1,715,000 and 1,916,000, respectively.

About GeneTether

Founded by EGB Ventures founder and managing partner, William J. Garner, M.D., and veteran gene editing researcher, R. Geoffrey Sargent, Ph.D., GeneTether is focused on developing its disruptive proprietary platform technology to significantly increase the efficiency of DNA insertion into the genome for gene correction and complementation strategies. The Companys wholly-owned platform technology uses a proprietary method to tether donor DNA templates to the genome editing complex, making the template readily available for use during the genome editing repair stage. The Company is leveraging its platform technology to develop curative therapies for the treatment of rare genetic diseases. GeneTethers proof of concept study demonstrated an approximately 7x higher gene editing efficiency as compared to the same gene editing payload without application of GeneTethers technology.

For more information, visitwww.genetether.com.

Contacts:

Roland Boivin, CEO

(833) 294-4363 ext. 1

roland@genetether.com

Jean Jen, CFO and Corporate Secretary

(833) 294-4363 ext. 2

jean@genetether.com

Forward-Looking Disclaimer

This news release contains statements that constitute "forward-looking statements." Such forward looking statements involve known and unknown risks, uncertainties and other factors that may cause GeneTethers actual results, performance or achievements, or developments in the industry to differ materially from the anticipated results, performance or achievements expressed or implied by such forward-looking statements. Forward looking statements are statements that are not historical facts and are generally, but not always, identified by the words "expects," "plans," "anticipates," "believes," "intends," "estimates," "projects," "potential" and similar expressions, or that events or conditions "will," "would," "may," "could" or "should" occur.

Forward-looking statements in this document include the expectation that the Company will significantly advance its research and development plan, the expectation that the Company will develop collaboration opportunities with other genetic medicines companies or develop M&A opportunities, and all other statements that are not statements of historical fact.

Although GeneTether believes the forward-looking information contained in this news release is reasonable based on information available on the date hereof, by their nature forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements, or other future events, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. By their nature, these statements involve a variety of assumptions, known and unknown risks and uncertainties and other factors, which may cause actual results, levels of activity and achievements to differ materially from those expressed or implied by such statements.

Examples of such assumptions, risks and uncertainties include, without limitation, assumptions, risks and uncertainties associated with the global COVID-19 pandemic; general economic conditions; adverse industry events; future legislative and regulatory developments; the Companys ability to access sufficient capital from internal and external sources, and/or inability to access sufficient capital on favorable terms; the ability of GeneTether to implement its business strategies; competition; the ability of GeneTether to obtain and retain all applicable regulatory approvals and other assumptions, risks and uncertainties, including those set forth under the heading Risk Factors in the Companys final prospectus dated March 21, 2022.

THE FORWARD-LOOKING INFORMATION CONTAINED IN THIS NEWS RELEASE REPRESENTS THE EXPECTATIONS OF THE COMPANY AS OF THE DATE OF THIS NEWS RELEASE AND, ACCORDINGLY, IS SUBJECT TO CHANGE AFTER SUCH DATE. READERS SHOULD NOT PLACE UNDUE IMPORTANCE ON FORWARD-LOOKING INFORMATION AND SHOULD NOT RELY UPON THIS INFORMATION AS OF ANY OTHER DATE. WHILE THE COMPANY MAY ELECT TO, IT DOES NOT UNDERTAKE TO UPDATE THIS INFORMATION AT ANY PARTICULAR TIME EXCEPT AS REQUIRED IN ACCORDANCE WITH APPLICABLE LAWS.

The Canadian Securities Exchange nor its Regulation Service has approved nor disapproved the contents of this news release.

NOT INTENDED FOR DISTRIBUTION TO UNITED STATES NEWS WIRE SERVICES OR FOR DISSEMINATION IN THE UNITED STATES

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GeneTether Therapeutics Inc. Announces Fiscal Year 2022 ... - TheNewswire.ca

Cell Isolation Technology Market to Eyewitness Increasing Revenue Growth during the Forecast Period by 2029 – openPR

Cell Isolation Technology

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The research report includes specific segments by region (country), company, Type, and Application. This study provides information about the sales and revenue during the historical and forecasted period. Understanding the segments helps identify the factors that aid the market growth. The Cell Isolation Technology research report provides information about the market area, which is further subdivided into sub-regions and countries/regions. In addition to the market share in each country and sub-region, this report chapter also contains information on profit opportunities.

The Cell Isolation Technology research report is an expert's analysis that mainly includes companies, types, applications, regions, countries, etc. Also, the reports analyse sales, revenue, trade, competition, investment, and forecasts. Industrial Analytics market research covers COVID-19 impacts on the upstream, midstream, and downstream industries. Also, this study offers detailed market estimates by emphasising statistics on several aspects covering market dynamics like drivers, barriers, opportunities, threats, and industry news & trends.

The future of cell isolation technology is an exciting area of development in biotechnology. Cell isolation involves separating specific cells from a complex mixture of cells in order to study them or use them for therapeutic purposes. Advances in cell isolation technology are essential for developing new treatments and therapies for a wide range of diseases.

One key area of development for the future of cell isolation technology is the use of microfluidics. Microfluidics involves using tiny channels and valves to manipulate small volumes of fluids. This technology can enable the isolation of individual cells with high precision and throughput, making it a powerful tool for studying rare cell populations and developing personalized medicine.

Another area of development is the use of magnetic cell isolation. This technology uses magnetic beads coated with antibodies to selectively isolate specific cells from a mixture. Magnetic cell isolation can be highly specific and efficient, enabling the isolation of rare cell populations with high purity.

In conclusion, the future of cell isolation technology looks incredibly promising, with the potential to transform the way we approach biotechnology and healthcare. As technology continues to advance, we can expect to see more innovative uses of cell isolation technology, enabling the development of new treatments and therapies for a wide range of diseases.

Overview of the market:The report presents the overview of the market with the production of the cost, dispatch, application, use volume and arrangement. The Cell Isolation Technology research report offers significant bits of information into the business focus from the early stage including some steady techniques chalked out by perceptible market pioneers to develop a strong foothold and development in the business. Moreover, the important areas of the Cell Isolation Technology market are also assessed on the basis of their performance.

This Cell Isolation Technology research report delivers key insights and gives clients a competitive advantage through a detailed report This report focuses on the key global players, defining, describing, and analyzing the market value, market share, market competition landscape, SWOT analysis, and development plans over the next few years. Also the report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

The report provides an in-depth assessment of the growth and other aspects of key countries (regions), such as the United States, Canada, Mexico, Germany, France, the United Kingdom, Russia, Italy, China, Japan, Korea, India, Southeast Asia, Australia, Brazil, and Saudi Arabia, among others. It also sheds light on the progress of key regional, including those in North America, Europe, Asia-Pacific, South America, and the Middle East and Africa.

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Segmentation Analysis of the market The market is segmented on the basis of the product, type, end users and application. Segmentation is considered to be the most vital part of the report which helps the reader to understand the market in prcised way.

By ManufacturersThermo Fisher Scientific, Inc.

Merck

BD Biosciences

Beckman Coulter, Inc.

Terumo BCT

GE Healthcare

Stemcell Technologies

Bio-Rad Laboratories, Inc.

By TypeCentrifugation

Flow Cytometry

Cell Electrophoresis

By End usersStem cell research

Cancer research

Tissue regeneration

In-vitro diagnostics

Others

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Geographical SegmentationNorth AmericaSouth AmericaAsia and PacificMiddle East and AfricaEurope

Reasons to buy ReportThe report offers in depth analysis of the market by providing the definition, application and classifications.The SWOT analysis and strategies of each vendor in the market in provided in the report.The offers comprehensive insights into current industry trends, trend forecast and growth drivers.The report provides a detailed overview of the vendor landscape, competitive analysis and key market strategies to gain competitive landscape.

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Deep Research Reports is digital database of syndicated market reports for global and China industries. These reports offer competitive intelligence data for companies in varied market segments and for decision makers at multiple levels in these organizations.

This release was published on openPR.

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Cell Isolation Technology Market to Eyewitness Increasing Revenue Growth during the Forecast Period by 2029 - openPR

Aspect Biosystems, Novo Nordisk Partner to Develop Bioprinted … – BioPharm International

Aspect Biosystems and Novo Nordisk A/S have announced a collaboration, development, and license agreement to develop bioprinted tissue therapeutics.

On April 12, 2023, Aspect Biosystems and Novo Nordisk A/S announced a collaboration, development, and license agreement to develop bioprinted tissue therapeutics designed to replace, repair, or supplement biological functions inside the body with the aim of delivering a new class of truly disease-modifying treatments for diabetes and obesity, according to a release.

The agreement states that Novo Nordisk will receive an exclusive, worldwide license to use Aspects bioprinting technology to develop up to four products for the treatment of diabetes and/or obesity. Meanwhile, Aspect will receive initial payments of US$75 million, including an upfront payment, research funding, and an investment in the form of a convertible note. According to the press release, Aspect is also eligible to receive up to US$650 million in future development, regulatory, commercial and sales milestone payments per product, as well as tiered royalties on future product sales.

We are thrilled to partner with Novo Nordisk, a global leader and pioneer in the fight against diabetes and chronic diseases, to create breakthrough therapeutics that could transform the lives of millions of people around the world, said Tamer Mohamed, chief executive officer, Aspect Biosystems, in a press release. This partnership leverages Aspects full-stack tissue therapeutic platform, talented team and bold vision, and reinforces our strategy to create bioprinted tissue therapeutics through partnerships with global industry leaders while also advancing our internal therapeutic pipeline.

A new platform technology from Aspect may allow for the development of a novel class of cell-based medicine designed to be biologically functional, encapsulated to be immune-protective, and suitable for surgical implantation. For Novo Nordisk, their team has developed expertise to differentiate stem cells into a wide array of cells that may be used to replace damaged and lost cells which could lead to a specific disease, like insulin-producing beta cells in type 1 diabetes, or manufacturing capabilities to produce the cells at scale.

The goal of the partnership is to create implantable bioprinted tissues to replace, repair, or supplement biological functions. Further, these tissues will be designed to be allogeneic, which means the cells used are derived from a single source to increase the practicality of future large-scale manufacturing.

Novo Nordisk has built strong capabilities when it comes to producing functional and highly pure therapeutic replacement cells at the highest quality and at scale, said Jacob Sten Petersen, corporate vice president and head of Cell Therapy R&D, Novo Nordisk, in a press release. Collaborating with Aspect Biosystems adds an important component to our strategy to develop comprehensive cell therapy products. We are excited to co-develop solutions for cell therapy delivery that could lead to life-changing treatments for those living with a serious chronic disease.

Source: Aspect Biosystems

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Seres Therapeutics Announces $250 Million Debt Financing with … – BioSpace

Company to receive proceeds of $110 million upon closing of the agreement

Financing to support commercial launch of VOWST and fund pipeline development

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today announced that it has entered into a new $250 million senior secured debt facility (Term Loan Facility) provided by funds managed by Oaktree Capital Management, L.P. (Oaktree). The Company drew the first tranche of $110 million at closing, with three additional tranches available. These additional tranches include $90 million that will be available in two tranches of $45 million each based upon the achievement of certain applicable VOWST sales targets, and an additional $50 million will be available to the Company at Oaktrees discretion to support potential future business development activities.

Of the $110 million advanced by Oaktree at closing, approximately $53 million retires outstanding debt, and after deducting fees and expenses, the net proceeds to the Company are approximately $50 million. The Term Loan Facility carries an interest rate equal to the three-month secured overnight financing rate (SOFR) plus 7.875%, with the interest rate capped at 12.875% per annum. The Term Loan Facility is expected to mature six years from funding and carries an initial interest only period for the first three years, which may be extended to six years based upon the achievement of certain VOWST sales targets.

This flexible debt offering from Oaktree provides Seres with support for the upcoming commercial launch of VOWST, the first orally administered microbiota-based therapeutic to prevent recurrence of C. difficile Infection (CDI) in adults following antibacterial treatment for recurrent CDI (rCDI), as well as for the advancement of Seres additional microbiome therapeutic candidates, including SER-155. Oaktree is one of the top capital providers for innovative biopharma companies, and we look forward to working closely together, said David Arkowitz, Chief Financial Officer at Seres.

With the approval of VOWST, Seres has firmly established itself as a leader in the exciting microbiome therapeutics field. We see a tremendous opportunity for VOWST and Seres multiple innovative pipeline programs to fill significant unmet medical needs across a variety of therapeutic areas. We are delighted to be collaborating with the Company during its next phase of growth and playing a role in bringing an entirely new treatment modality to patients, said Aman Kumar, Co-Portfolio Manager of Life Sciences Lending at Oaktree.

Oaktree is a leading provider of debt and royalty financing for the global life sciences industry. Since 2020, funds managed by Oaktree have committed over $3.0 billion across 31 investments for companies across the healthcare spectrum.

Morgan Stanley & Co. LLC acted as sole structuring agent to the Company. Latham & Watkins LLP served as legal counsel to Seres. Sullivan & Cromwell LLP served as legal counsel to Oaktree.

About Seres Therapeutics Seres Therapeutics, Inc. (Nasdaq: MCRB) is a commercial-stage company developing novel microbiome therapeutics for serious diseases. Seres lead program, VOWST, obtained U.S. FDA approval in April 2023 as the first orally administered microbiota-based therapeutic to prevent recurrence of C. difficile infection (CDI) in adults following antibacterial treatment for recurrent CDI and is being commercialized in collaboration with Nestl Health Science. Seres is evaluating SER-155 in a Phase 1b study in patients receiving allogeneic hematopoietic stem cell transplantation to reduce incidences of gastrointestinal infections, bloodstream infections and graft-versus-host disease as well as additional preclinical stage programs targeting Infection Protection in medically compromised patients. The Company is also conducting research to inform further development of microbiome therapeutics for ulcerative colitis.

For more information, please visit http://www.serestherapeutics.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including the receipt of future funding tranches; future business development activities; the achievement of sales milestones; and other statements which are not historical fact.

These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but 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, including, but not limited to, the following: we have incurred significant losses, are not currently profitable and may never become profitable; our need for additional funding; our limited operating history; the impact of the COVID-19 pandemic; our unproven approach to therapeutic intervention; the lengthy, expensive and uncertain process of clinical drug development; our reliance on third parties and collaborators to conduct our clinical trials, manufacture our product candidates and develop and commercialize our product candidates, if approved; and our ability to retain key personnel and to manage our growth. These and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), on March 7, 2023, and our other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230427005329/en/

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Initiation of scutellum-derived callus is regulated by an embryo-like … – Nature.com

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Grnenthal and King’s College London collaborate to develop human induced pluripotent stem cell-based microfluidic cultures for pain research – Yahoo…

Dr Ramin Raouf from King's College London and Grnenthal strive to develop reliable microfluidic culture models relevant for pain research based on human induced pluripotent stem cell-derived neurons

Grnenthal has expertise in developing human induced pluripotent stem cells towards sensory neurones and will support the lab of Dr Raouf with a total consideration of more than 350.000.

AACHEN, Germany and LONDON, April 27, 2023 /PRNewswire/ -- Grnenthal and King's College London announced a 24 months collaboration to develop microfluidic culture (MFC) models based on human induced pluripotent stem cells (iPSCs) and tailored to pain research. The collaboration aims to build on Dr Ramin Raouf's pioneering work on MFCs by establishing models using human iPSC-derived neurons that closely mimic the functionality of human nociceptive neurones. Grnenthal will support the lab of Dr Raouf with its competencies in characterising human iPSCs and a total consideration of more than 350.000.

The collaboration aims to address a significant need for better transational models in pain research. Traditional rodent behavioural models have frequently failed to translate into the clinical setting due to fundamental differences in molecular, cellular and genetic mechanisms of pain across species. As a result, there is a high interest in establishing pre-clinical models that can more accurately represent the conditions in the human body. Chronic pain is a considerable burden that impacts up to one in five people worldwide and is the most common reason for seeking medical help. It stresses healthcare systems and economies, while patients frequently experience limited efficacy from available medicines.

"Compared to traditional cell culture techniques, microfluidic cultures replicate more accurately the anatomy and physiology of the nervous system. Therefore, they can provide significant advantages in pre-clinical pain research", says Dr Ramin Raouf, Lecturer in Molecular Neuroscience at King's College London. "I believe adapting them with human iPSCs will create a transformative platform for generating translatable insights into the mechanisms of pain which will eventually contribute to reducing the attrition rate in clinical development."

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"We are delighted to join forces with Dr Ramin Raouf, a leading expert in microfluidic culture models. Taking this method to the next level may significantly enhance our understanding of how investigational medicines modulate pain", says Jan Adams, M.D., Chief Scientific Officer Grnenthal. "As a leading company in pain research, our ambition is to play a crucial role in developing such pioneering methodologies. We aim to anchor these competencies in our organisation and to include such models in our pre-clinical repertoire."

Grnenthal and Dr Ramin Raouf share a common research interest in neuroscience and the investigation of mechanisms of pathological pain. Dr Ramin Raouf is a world-leading researcher in the field of microfluidic cultures who pioneered the use of microfluidic culture models to study nociceptive neurons and established sophisticated rodent models. Grnenthal is a global leader in pain research and management and has delivered six essential treatment options for pain patients in the last decades. Today, the company is dedicated to creating the next generation of innovative non-opioid pain treatments. For R&D, Grnenthal executes a distinctive therapeutic area strategy focusing on four key pain indications: peripheral neuropathic pain, chronic post-surgical pain, chronic low back pain, and osteoarthritis.

About induced pluripotent stem cells

Induced pluripotent stem cells (iPSCs) are derived from a somatic cell that has been reprogrammed back into a pluripotent state by either introducing specific genes coding for transcription factors or adding small molecules that regulate cell identity. Those iPSCs can be differentiated into different cell types with unique characters, including peripheral sensory neurons.

About microfluidic cultures

Microfluidic devices are compartmentalised chips consisting of different chambers, sometimes called lab on a chip or 'tissue chips', allowing cell-to-cell contact via a series of connecting channels.Microfluidic cultures are used in this present collaboration to investigate the effects of analgesic compounds on different cellular compartments of the pain-sensing neuronal network, as well as the communication between neurons involved in pathological pain signalling.

About Grnenthal

Grnenthal is a global leader in pain management and related diseases. As a science-based, privately-owned pharmaceutical company, we have a long track record of bringing innovative treatments and state-of-the-art technologies to patients worldwide. Our purpose is to change lives for the better, and innovation is our passion. We are focusing all our activities and efforts on working towards our vision of a world free of pain.

Grnenthal is headquartered in Aachen, Germany, and has affiliates in 28 countries across Europe, Latin America, and the U.S. Our products are available in approx. 100 countries. In 2022, Grnenthal employed around 4,400 people and achieved revenues of 1.7 bn.

More information: http://www.grunenthal.com

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LinkedIn: Grunenthal Group

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About King's College London and the Institute of Psychiatry, Psychology & Neuroscience

King's College London is one of the top 35 universities in the world and one of the top 10 in Europe (QS World University Rankings, 2021/22) and among the oldest in England. King's has more than 33,000 students (including more than 12,800 postgraduates) from over 150 countries worldwide, and 8,500 staff. King's has an outstanding reputation for world-class teaching and cutting-edge research.

The Institute of Psychiatry, Psychology & Neuroscience (IoPPN) at King's is a leading centre for mental health and neuroscience research in Europe. It produces more highly cited outputs (top 1% citations) onpsychiatry andmental health than any other centre(SciVal2021),and on this metric has risen from 16th (2014) to 4th (2021) in the world for highly cited neuroscience outputs.In the 2021 Research Excellence Framework (REF),90% of research at the IoPPN was deemed 'world leading' or 'internationally excellent' (3* and 4*). World-leading research from the IoPPN has made, and continues to make, an impact on how we understand, prevent and treat mental illness, neurological conditions, and other conditions that affect the brain.

http://www.kcl.ac.uk/ioppn | Follow @KingsIoPPNon Twitter, Instagram, Facebook and LinkedIn

For further information please contact

Grnenthal

King's College London

Christopher Jansen

Communication Business Partner

Grnenthal GmbH

52099 Aachen

Phone: +49 241 569-1428

E-mail: Christopher.Jansen@grunenthal.com

Patrick O'Brien

Senior Media Officer

Insitute of Psychiatry, Psychology & Neuroscience King's College London

Phone: +44 07813 706 151

Email: Patrick.1.obrien@kcl.ac.uk

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Grnenthal and King's College London collaborate to develop human induced pluripotent stem cell-based microfluidic cultures for pain research - Yahoo...

Researchers reveal an ancient mechanism for wound repair – Science Daily

It's a dangerous world out there. From bacteria and viruses to accidents and injuries, threats surround us all the time. And nothing protects us more steadfastly than our skin. The barrier between inside and out, the body's largest organ is also its most seamless defense.

And yet the skin is not invincible. It suffers daily the slings and arrows of outrageous fortune, and it tries to keep us safe by sensing and responding to these harms. A primary method is the detection of a pathogen, which kicks the immune system into action. But new research from the lab of Rockefeller's Elaine Fuchs, published in Cell, reveals an alternative protective mechanism that responds to injury signals in wounded tissue -- including low oxygen levels from blood vessel disruption and scab formation -- and it doesn't need an infection to get into gear.

The study is the first to identify a damage response pathway that is distinct from but parallel to the classical pathway triggered by pathogens.

At the helm of the response is interleukin-24 (IL24), whose gene is induced in skin epithelial stem cells at the wound edge. Once unleashed, this secreted protein begins to marshal a variety of different cells to begin the complex process of healing.

"IL24 is predominately made by the wound-edge epidermal stem cells, but many cells of the skin -- the epithelial cells, the fibroblasts, and the endothelial cells -- express the IL24 receptor and respond to the signal. IL24 becomes an orchestrator that coordinates tissue repair," says Fuchs, head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Development.

Hints from pathogen-induced signaling

Scientists have long understood how the host responses protect our body from pathogen-induced threats: somatic cells recognize invading bacteria or viruses as foreign entities and induce a number of defense mechanisms with the help of signaling proteins such as type 1 interferons.

But how does the body respond to an injury that may or may not involve foreign invader? If we cut a finger while slicing a cucumber, for example, we know it instantly -- there's blood and pain. And yet how the detection of injury leads to healing is poorly understood on a molecular basis.

While type 1 interferons rely on the signaling factors STAT1 and STAT2 to regulate the defense against pathogens, previous research by the Fuchs lab had shown that a similar transcription factor known as STAT3 makes its appearance during wound repair. Siqi Liu, co-first author in both studies, wanted to trace STAT3's pathway back to its origin.

IL24 stood out as a major upstream cytokine that induces STAT3 activation in the wounds.

Microbe-independent action

In collaboration with Daniel Mucida's lab at Rockefeller, the researchers worked with mice under germ-free conditions and found that the wound-induced IL24 signaling cascade is independent of germs.

But what injury signals induced the cascade? Wounds often extend into the skin dermis, where capillaries and blood vessels are located.

"We learned that the epidermal stem cells sense the hypoxic environment of the wound," says Yun Ha Hur, a research fellow in the lab and a co-first author on the paper.

When the blood vessels are severed and a scab forms, epidermal stem cells at the edge of the wound are starved of oxygen. This state of hypoxia is an alarm bell for cell health, and induced a positive feedback loop involving transcription factors HIF1a and STAT3 to amplify IL24 production at the wound edge. The result was a coordinated effort by a variety of cell types expressing the IL24 receptor to repair the wound by replacing damaged epithelial cells, healing broken capillaries, and generating fibroblasts for new skin cells.

Collaborating with Craig Thompson's group at Memorial Sloan Kettering Cancer Center, the researchers showed that they could regulate Il24 gene expression by changing oxygen levels.

Once the researchers pinpointed the origin of the tissue-repair pathway in epidermal stem cells, they studied the wound repair process in mice that had been genetically modified to lack IL24 functionality. Without this key protein, the healing process was sluggish and delayed, taking days longer than in normal mice to completely restore the skin.

They speculate that IL24 might be involved in the injury response in other body organs featuring epithelial layers, which act as a protective sheath. In recent studies, elevated IL24 activity has been spotted in epithelial lung tissue of patients with severe COVID-19 and in colonic tissue in patients with ulcerative colitis, a chronic inflammatory bowel disease.

"IL24 could be working as a cue to signal the need for injury repair in many organs," Hur says.

Linked by function and evolution

"Our findings provide insights into an important tissue damage sensing and repair signaling pathway that is independent of infections," explains Fuchs.

An analysis with evolutionary biologist Qian Cong at UT Southwestern Medical Center revealed that IL24 and its receptors share close sequence and structure homology with the interferon family. Though they may not always be working in coordination at every moment, IL24 and interferons are evolutionarily related and bind to receptors sitting near each other on the surface of cells. The researchers suspect that these signaling molecules derive from a common molecular pathway dating far back in our past.

"We think that hundreds of millions of years ago, this ancestor might have diverged into two pathways -- one being pathogen defense and the other being tissue injury," Liu says.

Perhaps the split occurred to cope with an explosion of pathogens and injuries that caused a sea of troubles for life on Earth.

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Researchers reveal an ancient mechanism for wound repair - Science Daily

The gene-therapy revolution risks stalling if we don’t talk about drug … – Nature.com

A gene-editing therapy to correct deformed red blood cells in sickle-cell disease is in the works but at what cost?Credit: Eric Grave/SPL

We wish to suggest a structure for the salt of deoxyribose nucleic acid (D.N.A.), wrote James Watson and Francis Crick in this journal in 1953 (J. D. Watson and F. H. C. Crick Nature 171, 737738; 1953). This structure has novel features which are of considerable biological interest.

In the 70 years since those famous words were published, researchers have poured huge effort into unravelling those features and harnessing them for medicine. The result is a flourishing understanding of the genetic causes of diseases and a host of therapies designed to treat them.

Seventy years from now, the world might look back on 2023 as a landmark, as well. This year could see the first authorization of a therapy based on CRISPRCas9 gene editing, that involves tweaking the DNA in the bodys non-reproductive (somatic) cells. Gene editing allows scientists and could soon permit clinicians to make changes to targeted regions in the genome, potentially correcting genes that cause disease. Regulators in the United States, the European Union and the United Kingdom are evaluating a therapy that uses this approach to treat sickle-cell disease, and a decision could be made in the next few months.

CRISPR gene therapy shows promise against blood diseases

But even as such advances accrue, researchers are worrying about the future role of gene editing as well as other, more established forms of gene therapy in treating disease. Gene therapies currently carry eye-watering price tags, putting them out of the reach of many who need them. High prices could diminish the willingness of government funders to pay for gene-therapy research. And that, in turn, would make it harder for research institutions to continue to attract top talent to the field. Researchers, especially health economists, must work urgently with industry and governments to find a more affordable funding model.

CRISPRCas9s speedy path to the clinic was paved by years of steady advances in forms of gene therapy that use a virus to shuttle genes into cells. Over the past decade, regulators have approved several such gene therapies, for example CAR-T-cell therapies, which engineer immune cells to treat cancer. Hundreds more are in clinical trials.

These therapies typically cost something like US$1 million for a single treatment, and more once the costs of administering them, such as hospital stays and procedures required to isolate and manipulate cells, are factored in. Last year, the US Food and Drug Administration approved the first gene therapy to treat haemophilia B, a genetic disease that impairs blood clotting. The price is $3.5 million per treatment, making the therapy, called Hemgenix, the most expensive drug in the world.

Gene therapies are more costly to develop and produce than are more well-established treatments based on small-molecule drugs. But gene therapies can also carry the hope of a cure, freeing recipients from both long-term reliance on expensive medicines and the risk of hospitalizations. Some have argued that this justifies the high cost: if a therapy can save millions in downstream treatments, the initial outlay would still save money overall. Over time, after all, the costs of more-conventional treatments add up: one study, for example, found that in the United States, the cost of treating a person with sickle-cell anaemia until the age of 64 is $1.7 million (K. M. Johnson et al. Blood Adv. 7, 365374; 2023).

Researchers welcome $3.5-million haemophilia gene therapy but questions remain

Even in wealthy countries, health-care systems are ill-equipped to shoulder the high initial costs associated with gene therapies. In 2021, therapeutics developer Bluebird Bio in Somerville, Massachusetts, withdrew plans to market a gene therapy for -thalassaemia another blood disorder in Europe, after failing to reach an agreement with European authorities over the price. It said it would focus its sales efforts on the United States, where there has been comparatively little regulation of drug costs.

But even in the United States, costs matter. US health insurance is often subsidized by employers, and some are already saying that they will probably restrict their coverage of gene therapies in the next year, says Steven Pearson, president of the Institute for Clinical and Economic Review, a health-economics think tank in Boston, Massachusetts.

Low- and middle-income countries, meanwhile, are left entirely in the lurch. This is especially painful given that some of the diseases under consideration, such as -thalassaemia and sickle-cell disease, are more common in poorer parts of the world than in wealthy nations. In some sub-Saharan regions, for example, it is estimated that about 2% of children are born with sickle-cell disease. This is likely to be an underestimate, given how little screening is taking place.

It is too soon to know how much the CRISPRCas9 treatment for sickle-cell disease would cost; neither of its developers, Vertex Pharmaceuticals in Boston, Massachusetts, or CRISPR Therapeutics in Cambridge, Massachusetts, have disclosed what they will charge. But researchers are bracing themselves for the price tag to come.

At the Third International Summit on Human Genome Editing, held in London in March, much of the discussion centred on making gene-editing therapies accessible, particularly to low- and middle-income countries. The focus was on technological approaches to streamline the production and testing of such treatments. The sickle-cell treatment, for example, requires clinicians to isolate and edit blood-forming stem cells, destroy those that remain in the body, and then reinfuse the edited cells. Converting this to a genome-editing procedure that could be performed directly in the body rather than in isolated cells could make the treatment cheaper and more accessible.

Expensive treatments for genetic disorders are arriving. But who should foot the bill?

Another appealing approach is to develop gene-therapy platforms that have already been confirmed to be safe and effective. Gene-therapy developers could then just swap in a gene that targets the chosen disease, without the gamut of tests of safety and efficacy that are required when starting from scratch.

But technological solutions such as these will go only so far. US drug pricing has little to do with how much it costs to produce a therapy, says Pearson, because companies can charge as much as the market will bear. How much that price will drop in other countries could be limited by intellectual property rights and hindered by the complexities of making generic copies of biological drugs such as gene therapies. Some academic centres are trying to develop and deploy gene therapies without relying on pharmaceutical companies, but it is unclear how far such efforts can stretch without the financial resources and regulatory expertise found in industry.

In addition to pricing, gene-therapy technologies are mired in debates around regulation and intellectual property. How each of these plays out will determine how far researchers can go in capitalizing on Watson and Cricks initial discovery. Its important that scientists have an active role in these debates, and that they push such discussions to the fore sooner rather than later.

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The gene-therapy revolution risks stalling if we don't talk about drug ... - Nature.com

Research Identifies New Target Which May Prevent Blood Cancer – Cannon Courier

An international coalition of biomedical researchers co-led by Alexander Bick, MD, PhD, at Vanderbilt University Medical Center has determined a new way to measure the growth rate of precancerous clones of blood stem cells that one day could help doctors lower their patients' risk of blood cancer.

The technique, called PACER, led to the identification of a gene that, when activated, drives clonal expansion. The findings, published in the journal Nature, suggest that drugs targeting this gene, TCL1A, may be able to suppress clonal growth and associated cancers.

"We think that TCL1A is a new important drug target for preventing blood cancer," said Bick, the study's co-corresponding author with Stanford University's Siddhartha Jaiswal, MD, PhD.

More than 10% of older adults develop somatic (non-inherited) mutations in blood stem cells that can trigger explosive, clonal expansions of abnormal cells, increasing the risk for blood cancer and cardiovascular disease.

Since arriving at VUMC in 2020, Bick, assistant professor of Medicine in the Division of Genetic Medicine and director of the Vanderbilt Genomics and Therapeutics Clinic, has contributed to more than 30 scientific papers that are revealing the mysteries of clonal growth (hematopoiesis).

With age, dividing cells in the body acquire mutations. Most of these mutations are innocuous "passenger" mutations. But sometimes, a mutation occurs that drives the development of a clone and ultimately causes cancer.

Prior to this study, scientists would measure clonal growth rate by comparing blood samples taken decades apart. Bick and his colleagues figured out a way to determine the growth rate from a single timepoint, by counting the number of passenger mutations.

"You can think of passenger mutations like rings on a tree," Bick said. "The more rings a tree has, the older it is. If we know how old the clone is (how long ago it was born) and how big it is (what percentage of blood it takes up), we can estimate the growth rate."

The PACER technique for determining the "passenger-approximated clonal expansion rate" was applied to more than 5,000 individuals who had acquired specific, cancer-associated driver mutations in their blood stem cells, called "clonal hematopoiesis of indeterminate potential" or CHIP, but who did not have blood cancer.

Using a genome-wide association study, the investigators then looked for genetic variations that were associated with different clonal growth rates. To their surprise, they discovered that TCL1A, a gene which had not previously been implicated in blood stem cell biology, was a major driver of clonal expansion when activated.

The researchers also found that a commonly inherited variant of the TCL1A promoter, the DNA region which normally initiates transcription (and thus activation) of the gene, was associated with a slower clonal expansion rate and a markedly reduced prevalence of several driver mutations in CHIP, the second step in the development of blood cancer.

Experimental studies demonstrated that the variant suppresses gene activation.

"Some people have a mutation that prevents TCL1A from being turned on, which protects them from both faster clone growth and from blood cancer," Bick said. That's what makes the gene so interesting as a potential drug target.

The research is continuing with the hope of identifying additional important pathways relevant to precancerous growth in other tissues as well as blood, he added.

Researchers from more than 50 institutions across the United States, as well as Germany, Sweden, and the Netherlands participated in the study. Other VUMC co-authors were Taralyn Mack, Benjamin Shoemaker, MD, MSCI, and Dan Roden, MD.

The research at VUMC is supported by National Institutes of Health grant OD029586, a Burroughs Wellcome Fund Career Award for Medical Scientists, the E.P. Evans Foundation & RUNX1 Research Program, a Pew-Stewart Scholar for Cancer Research Award, the VUMC Brock Family Endowment, and a Young Ambassador Award from the Vanderbilt-Ingram Cancer Center.

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Research Identifies New Target Which May Prevent Blood Cancer - Cannon Courier