ReNAgade Therapeutics Continues Commitment to GanNA Bio and Glycan Biology




ReNAgade Therapeutics Continues Commitment to GanNA Bio and Glycan Biology

GanNA founders Carolyn Bertozzi, Ph.D., and Richard D. Cummings, Ph.D., to serve as advisors to ReNAgade

Additional investment strengthens ReNAgade’s delivery platform with unique conjugation platform based on novel glycobiology

CAMBRIDGE, Mass.–(BUSINESS WIRE)–ReNAgade Therapeutics, a company unlocking the limitless potential for RNA medicines, today announced its ongoing commitment to exploring the therapeutic opportunity of glycobiology through support for ongoing research at GanNA Bio, and the integration of key individuals of GanNA Bio into the ReNAgade ecosystem. GanNA founders Carolyn Bertozzi, Ph.D., and Richard D. Cummings, Ph.D, will now serve as advisors to ReNAgade.

“ReNAgade will continue to benefit tremendously from GanNA’s promising work in developing extra-hepatic delivery technology that helps expand the breadth and potential of our own RNA medicines,” said Brian Goodman, Co-founder of ReNAgade. “Delivery is foundational to ReNAgade’s mission to overcome the current limitations of RNA medicines. We believe the technology developed within GanNA will help further solidify ReNAgade’s leadership position as we continue advancing our unique, integrated therapeutic platform.”

Formed in 2021 and invested in by ReNAgade, GanNA Bio harnesses novel glycobiology to enable extra-hepatic RNA delivery based on pioneering research licensed from Stanford University, Boston Children’s Hospital, and Beth Israel Deaconess Medical Center. This research was developed in the labs of Carolyn Bertozzi, Ph.D., winner of the 2022 Nobel Prize in Chemistry and Professor of Chemistry in the Stanford School of Humanities and Sciences; Ryan A. Flynn, M.D., Ph.D., Assistant Professor at Boston Children’s Hospital; and Richard D. Cummings, Ph.D., S. Daniel Abraham Professor of Surgery at Beth Israel Deaconess Medical Center, and led by Namita Bisaria, Ph.D, M.B.A., a serial entrepreneur in the RNA therapeutics field. Its platform, based on new advances in glycobiology and synthesis, uses cell-specific glycans to develop next-generation conjugates and unlock the delivery of short and long stranded RNA medicines.

“The combination of GanNA’s innovative glycobiology-based targeting strategy with ReNAgade’s experience in the development of RNA medicines will be a powerful combination for the benefit of patients,” said Dr. Bertozzi. “The progress in glycoRNA science from our seminal publication in the journal Cell is moving fast, and integration with ReNAgade’s pipeline will strengthen our position to develop a broad portfolio of cell-specific delivery systems, leveraging the latest developments in glycobiology in order to expand the array of addressable diseases for RNA medicines. Glycobiology-based RNA medicines represent a new frontier, and it is rewarding to see the possibilities of ReNAgade developing these for clinical use.”

About ReNAgade Therapeutics

ReNAgade exists to unlock the potential for RNA medicines to treat disease anywhere in the body. We combine our novel RNA delivery platforms with a comprehensive RNA platform allowing for an all-RNA system for coding, editing, and gene insertion to develop new medicines.

To accelerate the future of medicine, we bring together a team with deep RNA and delivery expertise to develop paradigm-shifting RNA medicines.

ReNAgade Therapeutics—RNA Without Limits

For more information about the company, its technologies, and its leadership, visit www.renagadetx.com

About GanNA Bio

GanNA was created by Drs. Flynn and Bisaria, and invested by ReNAgade in 2021 following a sponsored research agreement in the lab of Dr. Flynn. The mission is to explore the potential of novel glycobiology therapies following the pioneering work at Stanford University by Drs. Flynn and Bertozzi on glycoRNA. The GanNA Bio Scientific Advisory Board included Drs. Bertozzi, Cummings, Flynn, and ReNAgade CSO, Pete Smith.

University Conflict of Interest Disclosure

Drs. Bertozzi, Cummings and Flynn consult for ReNAgade Therapeutics.

Contacts

Investor Relations:


Emily Brabbit, Argot Partners

(212) 600-1902

renagade@argotpartners.com

Media Relations:


Sarah Sutton, Argot Partners

(212) 600-1902

renagade@argotpartners.com

Lower Lights Health and AndHealth Achieve Pharmacy Accreditation With ACHC




Lower Lights Health and AndHealth Achieve Pharmacy Accreditation With ACHC

COLUMBUS, Ohio–(BUSINESS WIRE)–Lower Lights Health and AndHealth proudly announce they have achieved accreditation through Accreditation Commission for Health Care (ACHC) for Lower Lights Pharmacy’s specialty pharmacy services.

Accreditation is a process through which healthcare organizations demonstrate compliance with national standards. Accreditation by ACHC reflects an organization’s dedication and commitment to meeting standards that facilitate a higher level of performance and patient care.

ACHC is a nonprofit organization that has stood as a symbol of quality and excellence since 1986. ACHC is ISO 9001:2015 certified and has CMS deeming authority for Home Health, Hospice and DMEPOS.

Through a partnership with AndHealth, Lower Lights Health now offers specialty pharmacy services in-house and patients can fill their specialty prescriptions directly through Lower Lights Health Pharmacy.

“Creating access to specialty medications is critical to the wellbeing of our communities. We are honored to have received ACHC accreditation, which is a great testament to the attention to detail and care our patients can expect from our pharmacy team,” said Tracy Cloud, CEO of Lower Lights Health. She added, “Providing pharmacy services in-house ensures there are no gaps in care between a patient’s provider and their pharmacist. Patients achieve better health outcomes when they are surrounded by a team that communicates and collaborates on their care.”

About Lower Lights Health

Lower Lights Health is a Christian based, Federally Qualified Health Center and 501 (c) (3) non-profit providing the highest quality health care to all – regardless of the ability to pay. We connect all aspects of health and wellness to provide patents with total care. Lower Lights Health serves over 15,000 patients from 7 sites in central Ohio. We welcome and serve all members of the community with dignity and respect.

Lower Lights Health… We are Beyond Health Care.

To learn more, visit llchc.org.

About AndHealth

AndHealth helps Community Health Centers (CHCs) radically improve access and outcomes for patients in specialty care and specialty pharmacy, while becoming an even larger and more clinically integrated part of our healthcare system. This Whole-Person Community Care Model provides everything CHCs need to deliver in-house specialty care and specialty pharmacy, built for the unique needs of our medically underserved populations.

The model provides CHCs with more patients, more clinical capabilities, alignment with health systems, and sustainable reimbursement and funding that restores the integrity of the patient’s medical home and their critical reimbursement model—both having shifted away from CHCs through external specialty referrals and contract pharmacies. In restoring this integrity, CHCs can tear down barriers to care and stretch scarce federal resources to reach more patients with comprehensive services that weren’t possible before and that are desperately needed by patients.

Headquartered in Columbus, Ohio, AndHealth is led by former CoverMyMeds co-founder and CEO Matt Scantland and the team that built Ohio’s first healthcare technology unicorn, who have dedicated their lives to transformative innovation that improves access for patients. AndHealth is supported by key investors including the American Medical Association’s innovation subsidiary, Francisco Partners, and the state of Ohio’s economic development organization.

To learn more, visit AndHealth.com.

Contacts

Julia Phillips

VP, Marketing + Enablement

AndHealth

press@andhealth.com

Repare Therapeutics Appoints Steven H. Stein, M.D. to its Board of Directors




Repare Therapeutics Appoints Steven H. Stein, M.D. to its Board of Directors

CAMBRIDGE, Mass. & MONTREAL–(BUSINESS WIRE)–Repare Therapeutics Inc. (“Repare” or the “Company”) (Nasdaq: RPTX), a leading clinical-stage precision oncology company, today announced the appointment of Steven H. Stein, M.D., to its Board of Directors, effective as of the date of the Company’s upcoming annual meeting of shareholders in June 2024. The Company also announces that Todd Foley has decided not to stand for re-election as a director of the Company following the end of his current term as a Class I director on June 17, 2024, after serving more than seven years on the Board.

“On behalf of the Company and our Board of Directors, we thank Todd for his dedication and partnership, which have played a crucial role in shaping Repare into the leading precision oncology company that it is today. Todd has made tremendous contributions during the past seven years, and I am grateful to him and his team at MPM for their continued support,” said Lloyd M. Segal, President and Chief Executive Officer of Repare. “We are also honored to welcome Dr. Stein to our Board. His deep experience, proven track record and accomplishments in oncology clinical drug development will provide valuable guidance as Repare’s pipeline advances to later stages of development.”

“It has been a pleasure collaborating with the Repare management team and Board throughout its evolution into a multi-pronged clinical development company,” said Todd Foley, Managing Director at MPM BioImpact. “I look forward to the ongoing advancement of Repare’s pipeline and its continued progress as a leading precision oncology company.”

Dr. Stein currently serves as the Chief Medical Officer of Incyte Corporation, a position he has held since March 2015. At Incyte, he leads all medical and scientific activities involving clinical development, clinical operations, pharmacovigilance, clinical pharmacology, scientific communications and medical affairs. Prior to joining Incyte, from May 2011 to February 2015, Dr. Stein served as the Senior Vice President and Head of U.S. Clinical Development and Medical Affairs at Novartis Oncology. Prior to then, he was employed by GlaxoSmithKline from February 2004 to April 2011, serving first as its Head of Medicines Development for Hematology and Supportive Care and then as its Vice President, Global Oncology, Clinical Development. Dr. Stein also previously served on the board of directors of Kura Oncology, Inc. from 2017 until May 2023. Dr. Stein earned his MBBCH from the University of Witwatersrand in Johannesburg, South Africa. He has authored more than 100 scientific papers and presentations and is an Adjunct Assistant Professor in the School of Medicine, University of Pennsylvania. Dr. Stein is also an active member of several professional and scientific societies and academic committees.

“I am honored to join the Board of Directors at Repare at such a critical time in the Company’s growth and evolution, and am looking forward to working with its leadership team to deliver on the Company’s mission to develop synthetic lethal medicines that meaningfully improve the lives of cancer patients,” said Dr. Stein.

About Repare Therapeutics, Inc.

Repare Therapeutics is a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics. The Company utilizes its genome-wide, CRISPR-enabled SNIPRx® platform to systematically discover and develop highly targeted cancer therapies focused on genomic instability, including DNA damage repair. The Company’s pipeline includes lunresertib (also known as RP-6306), a PKMYT1 inhibitor currently in Phase 1/2 clinical development; camonsertib (also known as RP-3500), a potential leading ATR inhibitor currently in Phase 1/2 clinical development; RP-1664, a Phase 1 PLK4 inhibitor; RP-3467, a preclinical Polθ ATPase inhibitor program; as well as additional, undisclosed preclinical programs. For more information, please visit reparerx.com.

SNIPRx® is a registered trademark of Repare Therapeutics Inc.

Contacts

Investor Relations & Media Contact:

Robin Garner

Vice President and Head of Investor Relations

Repare Therapeutics Inc.

investor@reparerx.com

bluebird bio Announces Receipt of Expected Notice from Nasdaq




bluebird bio Announces Receipt of Expected Notice from Nasdaq

SOMERVILLE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE) today announced that it received a notice (the “notice”) on April 24, 2024 from The Nasdaq Stock Market LLC (“Nasdaq”) stating that because the Company has not yet filed its Annual Report on Form 10-K for the year ended December 31, 2023 (the “2023 Form 10-K”), it is no longer in compliance with Nasdaq Listing Rule 5250(c)(1) (the “Rule”), which requires listed companies to timely file all required periodic financial reports with the Securities and Exchange Commission (the “SEC”).

The notice has no immediate effect on the listing or trading of the Company’s common stock on the Nasdaq. Under Nasdaq rules, the Company has 60 calendar days from receipt of the notice, or until June 24, 2024, to submit a plan to regain compliance with the Rule.

On April 16, 2024, bluebird previously announced that it would be delayed in filing its 2023 Form 10-K due to the need to restate the Company’s consolidated financial statements as of and for the year ended December 31, 2022 and unaudited financial information for each of the first three quarters of 2023 and 2022 in its 2023 Form 10-K. The notice from Nasdaq is standard practice in the event of a delayed 10-K filing and was anticipated.

The Company is continuing to work expeditiously to complete the filing of the 2023 Form 10-K as promptly as possible. As previously reported, the restatements relate to the identification of embedded leases and the treatment of non-lease components contained in lease agreements. The Company does not expect the restatements to result in any impact on its cash position or revenue.

About bluebird bio, Inc.

bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.

Founded in 2010, bluebird has been setting the standard for gene therapy for more than a decade—first as a scientific pioneer and now as a commercial leader. bluebird has an unrivaled track record in bringing the promise of gene therapy out of clinical studies and into the real-world setting, having secured FDA approvals for three therapies in under two years. Today, we are proving and scaling the commercial model for gene therapy and delivering innovative solutions for access to patients, providers, and payers.

With a dedicated focus on severe genetic diseases, bluebird has the largest and deepest ex-vivo gene therapy data set in the field, with industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.

bluebird continues to forge new paths as a standalone commercial gene therapy company, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, such as statements regarding the Company’s restatement of certain historical financial statements and impact on cash position and revenue, the filing of its 2023 Form 10-K and submission of a plan to regain compliance with the Rule. Such forward-looking statements are based on historical performance and current expectations and projections about bluebird’s future goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond bluebird’s control and could cause bluebird’s future goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K for the year ended December 31, 2022, as updated by its subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

Contacts

Investors:
Courtney O’Leary, 978-621-7347

coleary@bluebirdbio.com

Media:
Jess Rowlands, 857-299-6103

jess.rowlands@bluebirdbio.com

Vagus nerve activation of the spleen shows promise to treat infections, Feinstein Institutes research




Vagus nerve activation of the spleen shows promise to treat infections, Feinstein Institutes research

MANHASSET, N.Y.–(BUSINESS WIRE)–One of the main roles of the spleen is to help the body’s immune system fight infections. The spleen does this through producing and regulating antibodies – antibody production is negatively affected in various conditions, including sepsis and autoimmune diseases like lupus. New research published in Science Advances by The Feinstein Institutes for Medical Research scientists shows that activation of the vagus nerve triggers the spleen, thereby regulating the production of antibodies. This research highlights how the nervous system regulates immunity and suggests potential for non-pharmalogical, vagus nerve modulation to treat a variety of diseases, like lupus and sepsis.

This research –led by Betty Diamond, MD, director of the Institute of Molecular Medicine, in collaboration with Stavros Zanos, MD, PhD, associate professor in the Institute of Bioelectronic Medicine and Barbara Sherry, PhD, professor at the Feinstein Institutes – found that chronic stimulation of the vagus nerve led to a decrease in the production of specific antibodies by splenic B cells that attack and destroy foreign invaders and are able to prevent disease in the future by remembering what those substances look like. This decrease was associated with changes in the way B cells matured and survived in the body, as well as alterations in the functional organization of other immune cells.

“Although we have a good understanding of the role of the vagus nerve in the regulation of the inflammatory response and the innate immune system, this study provides new insights in how the vagus nerve regulates adaptive immunity and the functions of B cells,” said. Dr. Diamond. “Better understanding these mechanisms will elucidate how altered function of the vagus nerve in conditions like sepsis and autoimmune disease may impact immune function and could lead to new therapeutic approaches for these conditions.”

In the study, the Feinstein Institutes team discovered that acetylcholine released in response to vagus nerve stimulation (VNS) directly affects B cells by interacting with specific receptors on their surface, thereby altering their ability to produce signaling molecules and mature.

“Consistent stimulation of the vagus nerve in mice allows us to explore the therapeutic possibilities of bioelectronic medicine in new diseases,” said Dr. Zanos. “These exciting findings warrant further investigation and eventually studies in humans to explore the extent to which vagus nerve stimulation, and other bioelectronic medicine approaches, could become treatment options for diseases involving adaptive immunity.”

For more than four decades, Dr. Diamond has dedicated her career to the study of DNA-reactive B cells, autoantibodies and their origin and effect on the body. In May 2022, in recognition of her breakthrough achievements in molecular medicine and original research, The National Academy of Sciences elected Dr. Diamond as one of its newest members.

Dr. Zanos’s research focus is on understanding the anatomy and physiology of the vagus nerve and the effects vagus nerve stimulation has in inflammation and cardiovascular diseases. This study builds on a 2021 paper by Dr. Zanos’ group in which they described the development and characterization of the first chronic VNS implant in mice. Recently, Dr. Zanos was awarded $6.7 million from the National Institutes of Health to lead a cross-insititonal research team to develop a detailed map of the anatomy of the human vagus nerve and all its more than 100,000 fibers.

“While we understand the spleen is an essential part of the immune system, finding that stimulation of the vagus nerve effects the regulation of adaptive immunity by this critical organ is novel and important,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “The research by Drs. Diamond and Zanos highlights the need to continue producing knowledge about bioelectronic medicine’s potential to treat or prevent autoimmune disease.”

The Feinstein Institutes for Medical Research is the global scientific home of bioelectronic medicine, which combines molecular medicine, neuroscience, and biomedical engineering. At the Feinstein Institutes, medical researchers use modern technology to develop new device-based therapies to treat disease and injury.

Building on years of research in molecular disease mechanisms and the link between the nervous and immune systems, our researchers discover neural targets that can be activated or inhibited with neuromodulation devices, like vagus nerve implants, to control the body’s immune response and inflammation. If inflammation is successfully controlled, diseases – such as arthritis, pulmonary hypertension, Crohn’s disease, inflammatory bowel diseases, diabetes, cancer and autoimmune diseases – can be treated more effectively.

Beyond inflammation, using novel brain-computer interfaces, Feinstein Institutes’ researchers developed techniques to bypass injuries of the nervous system so that people living with paralysis can regain sensation and use their limbs. By producing bioelectronic medicine knowledge, disease and injury could one day be treated with our own nerves without costly and potentially harmful pharmaceuticals.

About the Feinstein Institutes

The Feinstein Institutes for Medical Research is the home of the research institutes of Northwell Health, the largest health care provider and private employer in New York State. Encompassing 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health system science, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine – a new field of science that has the potential to revolutionize medicine. For more information about how we produce knowledge to cure disease, visit http://feinstein.northwell.edu and follow us on LinkedIn.

Contacts

Julianne Mosher Allen

516-880-4824

jmosherallen@northwell.edu

YC-Backed Yoneda Labs Raises $4 Million Seed Led by Khosla Ventures to Build Foundation Model for Chemists




YC-Backed Yoneda Labs Raises $4 Million Seed Led by Khosla Ventures to Build Foundation Model for Chemists

SAN FRANCISCO–(BUSINESS WIRE)–Yoneda Labs, the Y Combinator startup building a foundation model for chemists working in drug discovery, today announced it has raised $4 million in seed capital from Khosla Ventures, 500 Emerging Europe, 468 Capital, Fellows Fund, and Y Combinator.

More than 400,000 chemists are working on chemical manufacturing around the world. While scientists have begun to leverage AI in the discovery and design of drugs, the chemists whose job it is to synthesize the drugs are forced to run trials in wet labs without much, if any, automation, simulation tools, or other computational support. As a result, the pharmaceutical and chemical manufacturing industries waste billions of dollars on failed chemical experiments each year.

“When a chemist wants to couple two molecules together, they are left to old-school literature search and trial-and-error methods in a lab,” said Michal Mgeladze-Arciuch, founder and CEO of Yoneda Labs. “At Yoneda, our vision is to build a foundation AI model that analyzes and predicts what will happen before a chemist has to run their experiment. This would increase their productivity by an order of magnitude and potentially enable the creation of new drugs that isn’t currently possible to do cost-effectively at scale.”

“Machine learning and generative AI models have already begun to accelerate physics-oriented fields like aerospace engineering,” said Jon Chu, partner at Khosla Ventures. “Chemistry will be no different and the team at Yoneda Labs has a novel approach to creating a foundation model for chemistry that could change the way chemicals are manufactured and improve the drug discovery process.”

Founding Team

Yoneda Labs was founded by a group from the University of Cambridge with backgrounds in chemistry, robotics, and machine learning. The idea for Yoneda Labs was born when one of the founders, Jan Oboril, was working on drug development at a major pharmaceutical company. He felt he wasted hundreds of hours running trials in the lab that could be replaced with machine learning algorithms that could run the same experiments in silico.

After joining forces with co-founders Michal Mgeladze-Arciuch, who worked on machine learning algorithms at Jane Street and researched large AI models at UC Berkeley, and Daniel Vlasits, who won an international robotics competition, the team built an initial prototype that improved Jan’s process in the lab and was accepted to Y Combinator.

During their time at YC, the team identified 20,000 chemical reactions to generate proprietary data for their foundation model. In a small-scale trial, the models suggested good conditions in 95% of cases, a stark improvement to the industry standard in which the majority of the experiments fail.

With this funding, they plan to build out a robotics lab capable of running experiments on the remaining 20,000 reactions to continue training the first-ever generalizable AI model for chemists. Later this year, they plan to be able to run and analyze 200 experiments a day, the equivalent output of roughly 20 full-time chemists.

About Yoneda Labs

Yoneda Labs is building the world’s first foundation AI model for chemical manufacturing. The company was founded by Michal Mgeladze-Arciuch, Jan Oboril, and Daniel Vlasits, who met at the University of Cambridge and identified an opportunity to bring machine learning to outdated pharmaceutical practices.

Contacts

Michal Mgeladze-Arciuch

michal@yonedalabs.com
+1 415 527 0498

United States Industrial Valve Market Insights Report 2024-2029 Featuring Key Vendors – Bray International, Crane, Emerson Electric, Flowserve, SLB, Baker Hughes, Danfoss, KITZ, SMC, and Velan – ResearchAndMarkets.com




United States Industrial Valve Market Insights Report 2024-2029 Featuring Key Vendors – Bray International, Crane, Emerson Electric, Flowserve, SLB, Baker Hughes, Danfoss, KITZ, SMC, and Velan – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “U.S. Industrial Valve Market – Focused Insights 2024-2029” report has been added to ResearchAndMarkets.com’s offering.

The U.S. industrial valve market is expected to reach a value of $11.02 billion by 2029 from $8.57 billion in 2023, growing at a CAGR of 4.28%

The U.S. industrial valve market report contains exclusive data on 40 vendors. The competitive scenario in the U.S. industrial valve market is intensifying, with global and domestic players offering diverse products. Regarding market share, a few major players are currently dominating the market. Some companies currently dominating the market are Bray International, Crane, Emerson Electric, Flowserve, SLB, Baker Hughes, Danfoss, KITZ, SMC Corporation, and Velan.

Emerson’s launch of ASCOTM Series 262 and 263 Biodiesel Valves (FER. 8, 2023): Emerson’s introduction of the ASCOTM Series 262 and 263 Biodiesel Valves marks a significant development in the U.S. industrial valve market. These valves, certified for use with biodiesel blended fuel in boilers, enable the transition to a more sustainable energy source, aligning with evolving regulations and reducing greenhouse gas emissions. Market players must adapt by incorporating eco-friendly solutions, capitalizing on the growing demand for sustainable technologies, and ensuring compliance with environmental standards.

Growing Demand for Valves In Healthcare & Pharmaceutical Industries: The growing demand for valves in the healthcare and pharmaceutical industries is a significant driver for the market. As these sectors prioritize precision, sterility, and compliance with stringent regulations, specialized valves play a crucial role. This driver creates opportunities for market players to develop valves tailored to the unique needs of healthcare applications, emphasizing materials that meet hygiene standards and designs that enable precise control. Capitalizing on this demand requires manufacturers to focus on innovation, compliance with industry standards, and customization to address the specific challenges and requirements of the healthcare and pharmaceutical sectors.

Establishment of Smart Cities: Establishing smart cities in the U.S. is a powerful driver influencing the industrial valve market. Smart cities deploy advanced technologies for efficient infrastructure management, including smart water distribution systems, Heating, ventilation, and air conditioning (HVAC) systems, and automated processes that rely on industrial valves. Market players can align their strategies with this driver by offering valves equipped with smart technologies, collaborating on city infrastructure projects, and participating in developing solutions that contribute to the evolution of smart cities. This driver expands market opportunities and positions industrial valve manufacturers as key contributors to developing sustainable and technologically advanced urban environments.

U.S. INDUSTRIAL VALVE MARKET INSIGHTS

The gate product segment holds the largest market share of over 26% in the U.S. industrial valve market. Gate valves remain integral to the U.S. industrial valves market, particularly in applications where full and unobstructed flow are essential. In the oil and gas sector, gate valves are fundamental for controlling the flow of crude oil and natural gas in pipelines. Their robust construction makes gate valves a trusted choice in demand for the oil and gas industry, particularly in refining processes where reliability is crucial for uninterrupted operations, thus driving the segment.

The plastic material type is growing significantly, with the fastest-growing CAGR in the U.S. industrial valves market. Plastic valves are gaining acceptance in the U.S. industrial valves market, driven by their lightweight nature, corrosion resistance, and suitability for specific applications. Plastic valves provide cost-effective solutions, making them a choice in applications where budget considerations play a significant role. This affordability does not compromise performance, making them ideal for various industrial sectors.

Based on operations, the auto-responsive valve segment showcases the highest growth, growing at a CAGR of 4.97% in the U.S. industrial valve market. Auto-responsive valves contribute to energy efficiency by optimizing operations based on real-time data, reducing energy consumption during periods of low demand. This energy-conscious approach aligns with sustainability goals in various industrial sectors and helps grow this segment.

The on/off valve function segment maintains a significant presence and the largest U.S. industrial valves market share. The segmental growth is driven by an on/off valve that serves as the essential component for isolating and controlling the flow of fluids. Widely adopted in industries such as oil and gas, where rapid shutoff is crucial for safety and operational control, on/off valves are in demand for their reliability in critical applications.

Based on size, the 1-6″ segment holds the largest share in the U.S. industrial valves market. The segmental growth is primarily due to their acceptance in a wide range of industries due to their balanced size, making them suitable for both small- and moderately sized fluid control systems. They balance precision and flow capacities, making them ideal for applications with moderate flow rates and controlled adjustments. Commonly used in industrial processes, commercial plumbing, and HVAC systems, these valves cater to a broad spectrum of applications where flexibility in flow control is essential.

KEY QUESTIONS ANSWERED

• How big is the U.S. industrial valve market?
• What is the growth rate of the U.S. industrial valve market?
• What are the trends in the U.S. industrial valve market?
• Who are the major players in the U.S. industrial valve market?

Key Attributes:

VENDOR LANDSCAPE

Key Vendors

• Bray International
• Crane
• Emerson Electric
• Flowserve
• SLB
• Baker Hughes
• Danfoss
• KITZ
• SMC Corporation
• Velan

Other Prominent Vendors

• Curtiss-Wright
• Mueller
• The Weir Group
• Parker Hannifin
• Honeywell International
• Watts
• Powell Valves
• Richards Industrials
• Forbes Marshall
• Tylok
• A-T Controls
• White Knight Fluid Handling
• KLINGER
• Bohmer
• Simtech
• Anything Flows
• Davis Valve
• AVK Group
• Walworth
• LESER
• Microfinish
• Newmans Valve
• Bonney Forge
• Global Valve & Controls
• Gemini Valve
• Superlok
• Gemco Valve
• DEFINOX
• Valcor Engineering
• Valves Only

SEGMENTATION & FORECAST

Product

• Ball
• Butterfly
• Globe
• Gate
• Check
• Others

Material

• Stainless Steel
• Bronze/Glass
• Plastic
• Cast Iron
• Others

Operation

• Manual
• Automatic
• Auto-Responsive

Function

• On/Off
• Control

Size

• Below 1″
• 1-6″
• 7-24″
• Above 24″

Motion

• Linear
• Rotary
• Others

End-User

• Oil & Gas and Energy
• Water & Wastewater Treatment
• Chemicals
• Food & Beverage
• Healthcare
• Others

For more information about this report visit https://www.researchandmarkets.com/r/n7agtn

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Vertex Announces European Commission Approval for KALYDECO® to Treat Infants With Cystic Fibrosis Ages 1 Month and Older




Vertex Announces European Commission Approval for KALYDECO® to Treat Infants With Cystic Fibrosis Ages 1 Month and Older

– KALYDECO ^® is the first and only medicine approved in the EU in this age group to treat the underlying cause of cystic fibrosis for specific mutations in the CFTR gene –

LONDON–(BUSINESS WIRE)–Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KALYDECO^® (ivacaftor) for the treatment of infants down to 1 month of age with cystic fibrosis (CF) who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

“Today’s approval is an important milestone for the cystic fibrosis community. Treating CF early in life can potentially slow the progression of the disease, which is why it is so important to start treatment from a very young age,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.

As a result of existing access agreements in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands, eligible patients will have access to the expanded indication of KALYDECO^® (ivacaftor) shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement authorities across the European Union to ensure access for all other eligible patients. In the U.K., following MHRA approval at the end of 2023, and as a result of the existing reimbursement agreement between Vertex and the National Health Service, eligible infants ages 1 month and older in the U.K. have access to this expanded indication for KALYDECO^® (ivacaftor).

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.

About KALYDECO^® (ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. KALYDECO^® (ivacaftor) was the first medicine to treat the underlying cause of cystic fibrosis in people with specific mutations in the CFTR gene.

KALYDECO^® (ivacaftor) is a prescription medicine for the treatment of people with CF aged at least 1 month and weighing at least 3 kg who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 14 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on LinkedIn, YouTube and Twitter/X.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic, M.D., in this press release, statements regarding the eligible patient population for KALYDECO, expectations for access to KALYDECO for eligible patients, including Vertex’s plans to continue to work with reimbursement authorities across the European Union to ensure access for eligible patients, and statements regarding the potential benefits of KALYDECO. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include risks listed under the heading “Risk Factors” in Vertex’s annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company’s website at www.vrtx.com and www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Contacts

Vertex Pharmaceuticals Incorporated

Investors:
InvestorInfo@vrtx.com

Media:
mediainfo@vrtx.com
or

International: +44 20 3204 5275

Variantyx Secures $36M Investment As Testing Demand And Market Share Continues To Increase




Variantyx Secures $36M Investment As Testing Demand And Market Share Continues To Increase

FRAMINGHAM, Mass.–(BUSINESS WIRE)–Variantyx, a leader in molecular diagnostics, announced today that it has secured an additional $36 million in funding from its portfolio of investors which includes Peregrine Ventures, Pitango HealthTech, New Era Capital Partners and Bosch Ventures. The company, a technology-driven provider of advanced genomic testing for genetic disorders, reproductive health and precision oncology markets has seen a rapid increase in test volumes in the first quarter of 2024, further expanding its market share. The additional investment, which brings the total amount invested in Variantyx to over $125 million, will fund additional technological and commercial expansion, enabling the company to capitalize on the shifting market, seizing the opportunity to provide cutting-edge, genome-based care to a larger number of patients while maintaining strong unit economics and building significant value.

“It’s clear that the current shift away from panel and exome-based testing to whole genome testing benefits patients, providers and payers alike,” said Christine Stanley, Variantyx’s Chief Director of Clinical Genomics. “As a pioneer in clinically-accredited, genome-based testing with tens of thousands of genomes sequenced to date, Variantyx is well-positioned to maintain its leadership in genomic diagnostics with on-going innovation of new genome analysis technologies, providing real improvements in patient outcomes to an increasingly larger subset of the population.”

“The funding demonstrates continued trust and confidence in our ability to deliver on the long-term goals of the company,” said Haim Neerman, CEO of Variantyx. “The additional commercial expansion enabled by this newest investment will help further accelerate our growth to meet the market demand.”

About Variantyx

Variantyx is an award-winning, technology-driven molecular diagnostics company providing disruptive solutions for the genetic disorders, reproductive health, and precision oncology markets. The proprietary whole genome analysis platforms developed by Variantyx allow clinicians to better understand a person’s genetic makeup, leading to unmatched diagnostic capabilities and improved personalized treatment recommendations. For more information, please visit www.variantyx.com.

Contacts

Haim Neerman

Haim.neerman@variantyx.com
(617) 209-2090

C2N Diagnostics Expands Into Japan Through Mediford Corporation Partnership With Precivity™ Blood Testing for Alzheimer’s Disease and Brain Research Market




C2N Diagnostics Expands Into Japan Through Mediford Corporation Partnership With Precivity™ Blood Testing for Alzheimer’s Disease and Brain Research Market

Collaboration Highlights C₂N’s Growing Global Footprint for Offering Blood Biomarker Research Testing That’s Transforming Clinical Research and Early Diagnosis

ST. LOUIS–(BUSINESS WIRE)–#Alzheimers—C₂N Diagnostics, LLC (“C₂N”), a specialty diagnostics company with a vision to bring Clarity Through Innovation®, today announced a partnership agreement with Mediford Corporation, a PHC Holdings Corporation (TSE 6523) Group Company and premier provider of clinical research to the biopharma industry throughout Japan. The agreement greatly increases access to C₂N Biopharma Clinical Research Services, which offers highly sensitive mass spectrometry-based identification, quantification and monitoring of proteins, protein fragments (peptides) and other biomolecules implicated in human neurological diseases and overall brain health.

The Mediford Corporation alliance means more researchers in Japan can access C₂N’s Precivity™ line of high-performance blood tests that aid in the diagnosis, prognosis, staging, and treatment monitoring of Alzheimer’s disease (AD), including Aβ42/40 and %p-tau217, and other proprietary biomarkers such as those that measure the microtubule-binding domain of tau protein (e.g., MTBR-tau). The company’s latest innovation in the MTBR-tau biomarker intends to uniquely detect the presence and burden of neurofibrillary “tau” tangles (NFTs) comprised of aggregated tau proteins in the brain and as a potential alternative to invasive, costly and often inaccessible tau PET tracers.

C₂N’s expertise in treatment monitoring is highlighted by researchers using its Precivity-Aβ™ blood biomarker in the Clarity AD clinical trial for lecanemab and its Precivity-p-tau217™ blood biomarker in the TRAILBLAZER-ALZ 2 clinical trial for donanemab. As a screening tool to improve the time, efficiency and costs of clinical trial enrollment, C₂N’s blood biomarkers are also being used in the landmark global AD prevention study of AHEAD 3-45, involving evaluation of lecanemab safety and efficacy among individuals at high-risk for future development of clinical AD.

In recognizing wide global interest in its services, C₂N has appointed Maki Hoshiko, Ph.D., as the lead for its Japan strategy and scientific collaborations. Hoshiko, who’s based in Kyoto, Japan, has a well-established background as a neuroscientist focused on the commercialization aspects of the field. She previously served in a similar role with Biogen Inc.

Research Indicates Japanese at High Risk for Alzheimer’s Disease

C₂N’s Japan expansion is occurring as the high-density population country accounted for the second-highest diagnosed prevalent cases of Alzheimer’s disease (at 26%) compared to Germany, France, Italy, Spain, the United Kingdom and the United States, according to the “Alzheimer’s disease (AD) – Epidemiology Forecast – 2032.” The report states that the country has nearly 4 million diagnosed prevalent cases of Alzheimer’s disease. Other research has found that Japan has the highest proportion of people with dementia of any country.

C₂N CEO Dr. Joel Braunstein says, “Japan is a vitally important country in the Alzheimer’s research sector, as both a leading technology innovation hub and also a region of the world where society places a high premium on healthy aging of its citizens. We believe our partnership with Mediford Corporation will unlock many opportunities and possibilities. Mediford Corporation is known for providing clinical and non-clinical analysis that supports each stage of drug research and development, including advanced treatment options. We believe this collaboration provides an excellent pathway to reaching clinical trial researchers and drugmakers searching for the newest treatment solutions and products.”

Mediford Corporation President Mr. Kei Shimizu says, “We’ve closely examined the options for offering blood tests to assess for Alzheimer’s pathology and concluded that C₂N’s blood biomarker testing for clinical trial and treatment monitoring delivers one of the highest standard. C₂N’s assays have been used in over 150 Alzheimer’s disease and other research studies throughout the United States and the world; this includes globally impactful treatment and prevention trials involving disease-modifying therapies that are changing the outlook of Alzheimer’s disease. C₂N’s excellence in quality stands out for its success in developing and commercializing novel diagnostics for Alzheimer’s disease and related forms of neurodegeneration to improve treatment decisions and patients’ lives.”

For researchers in Japan interested in learning more, please contact General Affairs and Human Resource Department of Mediford Corporation at medf-dds-gas@gg.mediford.com.

About C₂N Diagnostics, LLC

C₂N is a specialty diagnostics company with a vision to bring Clarity Through Innovation®. C₂N strives to provide exceptional clinical laboratory services and advanced diagnostic solutions in the field of brain health. C₂N’s high-resolution mass spectrometry-based biomarker services and products are used for: clinical decision-making to improve patient care, including diagnosis and treatment monitoring; maximizing the quality and efficiency of clinical trials that test novel treatments for neurodegeneration; and providing innovative tools to help healthcare researchers better understand novel mechanisms of disease, identify new treatment targets, and conduct important epidemiologic studies to improve global public health. C₂N assays have been used in over 150 Alzheimer’s disease and other research studies throughout the U.S. and the world. This includes landmark treatment and prevention trials involving disease-modifying therapies (DMTs) that are changing the trajectory of Alzheimer’s disease. C₂N has ongoing collaborations with multi-national pharmaceutical and biotech companies, leading academic institutions, National Institute on Aging, Alzheimer’s Association, and other non-profits and consortiums. Over 30,000 Precivity™-related biomarker measures have been reported through peer-reviewed publications, with many more manuscripts currently under review.

The company acknowledges generous support from National Institute on Aging, GHR Foundation, Alzheimer’s Drug Discovery Foundation, BrightFocus Foundation and Alzheimer’s Association. For more information visit www.C2N.com.

About Mediford Corporation

Mediford Corporation is a Japanese subsidiary of PHC Holdings Corporation (TSE 6523). It started drug development support services as the basis business in November 2023 as a member of PHC Group. Mediford Corporation provides comprehensive services for drug development from an exploratory phase of R&D to a clinical development phase. Mediford Corporation strives to enhance its services for pharmaceutical companies and analytical laboratories globally through analytical technologies required in both non-clinical and clinical studies to realize emerging varieties of treatment modality, through collaboration with pharmaceutical/biotech companies and academia in advanced scientific fields. For more information visit www.mediford.com.

Contacts

For C₂N Diagnostics

COMPANY CONTACT:

Joni Henderson

JHenderson@c2n.com
314-464-0009

MEDIA CONTACT:

Adam Shapiro

Adam.Shapiro@ASPR.bz
202-427-3603

For Mediford Corporation

[For product and service inquiries]

General Affairs and Human Resource Department

Mediford Corporation

Phone: +81-3-6905-5860

E-mail: medf-dds-gas@gg.mediford.com

[For IR and media inquiries]

Investor Relations & Corporate Communications Department

PHC Holdings Corporation

Phone: +81-3-6778-5311

E-mail: phc-pr@gg.phchd.com