The life sciences industry is constantly evolving, influenced by new technologies, regulatory changes, and market dynamics. To help professionals stay informed, the MCCC Life Science Program is hosting MCCC Trends in Life Science on Wednesday, March 12, 2025, at MCCC, 51 Monroe Street, Suite 1800, Rockville, MD. This must-attend event will provide valuable insights into the forces shaping the future of life sciences.

Why Attend?

✔️ Stay ahead with expert insights on emerging industry trends
✔️ Learn how to navigate complex regulatory and tax landscapes
✔️ Build connections with key stakeholders in the life sciences ecosystem

GERMANTOWN, Md. & VENLO, Netherlands–(BUSINESS WIRE)– QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced that the U.S. Food and Drug Administration (FDA) has cleared the QIAstat-Dx Gastrointestinal Panel 2 Mini B for clinical use, further strengthening its syndromic testing portfolio in the United States.

This marks QIAGEN’s second FDA clearance of a QIAstat-Dx panel in 2025, and builds on the authorization of five panels for use on the QIAstat-Dx system within the last 10 months.

QIAGEN has now received regulatory clearances for three mini panels for detection of respiratory and gastrointestinal conditions, with these panels designed for outpatient use to aid fast and informed treatment decisions.

ROCKVILLE, Md. and SUZHOU, China, March 05, 2025 (GLOBE NEWSWIRE) — Ascentage Pharma (NASDAQ: AAPG; HKEX: 6855), a global biopharmaceutical company engaged in discovering, developing, and commercializing therapies to address global unmet medical needs primarily for hematological malignancies, today announced that its drug, olverembatinib, has been granted a Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA), for combination with low-intensity chemotherapy for the first-line treatment of newly-diagnosed patients with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).

On April 1, 2025, Georgetown University’s Office of Technology Commercialization, in partnership with the Georgetown Entrepreneurship Initiative, will host the first-ever Georgetown Research and Innovation Showcase. This half-day event will bring together researchers, entrepreneurs, investors, and industry leaders to highlight groundbreaking discoveries and explore commercialization opportunities.

Attendees can expect engaging research presentations, an industry panel discussion, and a fast-paced Venture Fair, where selected faculty, student, and alumni-led startups will pitch their disruptive technologies to an audience of venture capitalists, angel investors, and corporate innovation teams.

The event will culminate in a networking reception, offering a unique opportunity for collaboration and funding discussions between the Georgetown community and key industry players. Whether you’re an innovator, investor, or looking to connect with cutting-edge ventures, this is an event you won’t want to miss.

? Date: April 1, 2025
? Time: 1:30 PM – 6:30 PM ET
? Location: Georgetown University

Don’t miss your chance to be part of this exciting showcase of innovation and entrepreneurship!

https://otc.georgetown.edu/newsevents/research-and-innovation-showcase-2025/

COLUMBIA, Md., (February 25, 2025) — TEDCO, Maryland’s economic engine for technology companies, announced a recent $250,000 Venture Funds investment in Pathotrak. TEDCO’s evergreen Venture Funds are dedicated to funding and growing the next generation of early-stage technology and life sciences businesses in Maryland. Through this fund, companies are offered an experienced team that is focused on supporting the entrepreneurial journey.

“Ensuring food safety is a growing challenge, and current testing methods are often slow and costly for producers,” said Javier Atencia, CEO of Pathotrak. “With TEDCO’s continued support, we are advancing our technology in an effort to shorten the time required for pathogen detection, empowering manufacturers with what we hope are faster, more reliable solutions.”

By Benjamin Mueller -The Trump administration stormed into office, loudly firing workers and closing diversity programs. But behind the scenes, it has also brought biomedical research to the brink of crisis by holding up much of the $47 billion the United States spends on the field every year.

The world’s leading medical labs can be found in the United States, and they rely on grants from the National Institutes of Health. The agency has stopped vetting future studies on cancer, Alzheimer’s, heart disease and other ailments. Trump aides have said they just need time to review spending their predecessors had promised, but it’s unclear what they’re looking for at the N.I.H. or when scholars can expect to start receiving money again.

In today’s newsletter, I’ll walk you through what happened — and why it matters.

BALTIMORE, MD (January 24, 2025) – Governor Wes Moore today announced the appointment of Harry Coker, Jr. as secretary of the Maryland Department of Commerce. Coker will assume the post as acting secretary on Feb. 5 and his appointment is pending confirmation by the Maryland State Senate during the 2025 Legislative Session. Coker will succeed Secretary Kevin Anderson, who will transition to a new role as senior advisor to the governor for economic development.

“At the heart of our economic growth agenda is a commitment to investing in industries of the future—from cyber to quantum. Harry Coker understands that mission and brings a wealth of experience working on the leading edge of the very sectors Maryland stands to win,” said Gov. Moore. “Our entire administration is grateful for Kevin Anderson’s distinguished service and we look forward to this new chapter at the Maryland Department of Commerce under Harry Coker’s leadership.”

The Deloitte AI Institute has launched a new video series, You, Me, and Three: GenAI Questions, featuring industry leaders discussing the rapid transformation of federal health through generative AI and automation. BHI Board of Directors Member Juergen Klenk, Principal at Deloitte Consulting LLP, joined Beena Ammanath in this series to explore how organizations can cut through the AI hype and establish trust around AI. Watch the full series to gain insights from cross-industry experts on the future of AI in healthcare.

https://youtu.be/dNFsBSjBCFc

By Conor Gowder & Mark Skinner

The Feb. 7, 2025, memo from the NIH Office of the Director (NOT-OD-25-068), now on hold because of two federal judge actions, announced the implementation of a flat 15% Facilities and Administrative fee (F&A) “across all NIH grants.” While the historic average F&A, or indirect cost rate, paid for by NIH is between 27 and 28%, the memo stated, the agency has previously allowed private small businesses without a negotiated F&A rate to charge up to 40% on their SBIR/STTR awards without further justification, drastically lowering their administrative burdens. Thus, a flat 15% fee on F&A if ever implemented would likely lead to some hardship for the small businesses.

WASHINGTON, DC / BERN, SWITZERLAND – Children’s National Hospital and Compremium AG entered into a strategic partnership to develop and commercialize cutting-edge medical technologies for the pediatric population. This partnership will bring Compremium’s non-invasive and AI-enhanced technology to the forefront of pediatric healthcare.

Diagnosing pressure-related conditions without complicated, invasive procedures has long been a challenge in the medical community. Compremium’s technology aims to transform the diagnosis of pressure physiologies, including venous pressure, intracranial pressure, and tissue pressure conditions with their non-invasive, point of care solution. Through this strategic collaboration, Children’s National will serve as the Pediatric Innovation Hub for Compremium, leading clinical studies, research projects, and commercialization efforts focused on non-invasive pressure monitoring and other breakthrough technologies.

As we celebrate Women in Science Day and Women’s History Month, we are proud to spotlight the women of Volition.  From driving groundbreaking innovation to shaping strategic direction, our team do a fantastic job and we’d like to kick off the month of celebrations by featuring our female Directors on our Board.

The women on our Board of Directors are at the forefront of advancing science, fostering inclusivity, and paving the way for the future of health and medicine.

Dr. Ethel Rubin, who joined our Board in September 2024, has dedicated over 20 years to the life sciences sector, driving innovation and commercialisation strategies. Her advice to young women entering science? “Let your intellectual curiosity guide you.” She encourages future scientists to be true to themselves in choosing their career paths—whether they’re solving real-world problems or diving deep into basic biology. Ethel also highlights the importance of perseverance:

BALTIMORE, Feb. 13, 2025 (GLOBE NEWSWIRE) — Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on treating rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs). EXG-34217 is currently being studied in an ongoing Phase 1/2 trial (NCT04211714) for the treatment of patients with TBDs with bone marrow failure. Enrollment is currently open for participants aged 12 and older, regardless of gender or ethnicity.

What does it take to establish Maryland as a powerhouse for biotech commercialization? In this episode of BioTalk with Rich Bendis, Dr. Deborah Hemingway, Founder and Managing Partner of Ecphora Capital, shares her insights on the current investment landscape in the BioHealth Capital Region. She discusses the strategic advantages that make Maryland uniquely positioned for success, the critical gaps that must be addressed, and how state and local governments can provide essential support through tax credits, grants, and economic incentives.

Dr. Hemingway also highlights Ecphora Capital’s impact, having deployed $15 million in the last two years, and reveals the firm’s strategy for future investments, including SPVs and a dedicated fund. With deep experience in medtech commercialization, strategic partnerships, and early-stage investing, she offers valuable perspectives on where the industry is headed in 2025 and how companies can thrive in an evolving biotech ecosystem.

Listen via your favorite podcast platform:
Apple: https://apple.co/40QvxJg
Spotify: https://spoti.fi/3WWEvTZ
Amazon Music: https://amzn.to/3QbxZoH
YouTube Music: https://bit.ly/40T7xVF
TuneIn: https://bit.ly/42LITcb

Tune in for a compelling conversation on innovation, investment, and the future of biotech in the BioHealth Capital Region.

By Brian Darmody: While the Maryland economy is facing budget uncertainties, the state can help surmount these challenges by growing private growth industries, converting research into jobs, connecting the region, and attracting federal funding from those few federal budgets (defense and economic competitiveness) that are likely to grow.

Here’s how:

Winning the technology race with China will be a bipartisan effort in Congress and with the White House and state of Maryland has a role to play. The Department of Defense (DOD) R&D budget is expected to grow, including its University Affiliated Research Centers (UARCs) that ensure essential engineering and technology capability are maintained.

BALTIMORE, Jan. 23, 2025 /PRNewswire/ — LifeSprout, a privately held regenerative medicine company founded with technology licensed from Johns Hopkins University, today announced it has filed for CE Mark registration for Lumina.

Lumina is the first product from LifeSprout’s proprietary Regenerative Matrix platform. The technology comprises biomimetic, biocompatible, biodegradable materials engineered to look and feel like native soft tissue.

“The CE-mark submission marks a significant milestone for LifeSprout and brings Lumina an important step closer to benefiting patients,” stated Sashank Reddy, MD, PhD, co-founder of LifeSprout and Associate Professor of Plastic and Reconstructive Surgery and Biomedical Engineering at Johns Hopkins.

MONTGOMERY COUNTY, Md. — Businesses in Maryland’s Montgomery County experienced a surge in investment funding in 2024. Mergers and acquisitions, venture capital and private funding deals totaled $2.9 billion, a 36% increase over 2023’s total of $2.2b. Spanning 106 deals across 96 companies, this surge in mergers and acquisitions, venture capital, and private investment reflects Montgomery County’s continued prominence as a regional economic leader. 

Diverse Industries Driving Growth 

Over 100,000 patients in the United States have IPF, with only two approved therapies available that only slow the course of disease progression

Top line data expected in the first half of 2026

SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.–(BUSINESS WIRE)– United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, announced full enrollment of the TETON 1 study evaluating the use of Tyvaso® (treprostinil) inhalation solution (nebulized Tyvaso) for the treatment of idiopathic pulmonary fibrosis (IPF).

New Study Highlights Importance of Measuring Well-Being in Mental Health Research

WASHINGTON (February 10, 2025) – A new study published in Nature Mental Health underscores the critical role of assessing well-being in clinical research and treatment. 

Led by Fallon Goodman of the George Washington University, the study highlights how well-being, beyond the absence of mental illness, is essential for long-term health outcomes, including resilience, recovery, and overall quality of life.

Despite its importance, well-being is often overlooked in mental health research. The study identifies challenges in measuring well-being, such as inconsistencies in assessment methods, and offers recommendations to improve research approaches. By incorporating well-being metrics into clinical trials and treatment evaluations, researchers and clinicians can gain a more comprehensive understanding of mental health and enhance patient outcomes.

ROCKVILLE, Md., Jan. 30, 2025 (GLOBE NEWSWIRE) — MaxCyte, Inc., (Nasdaq: MXCT; LSE: MXCT), a leading, cell-engineering focused company providing solutions to advance the discovery, development and commercialization of next-generation cell therapeutics announced today the acquisition of SeQure Dx, a market leader of on-target and off-target editing assessment services for cell and gene therapies.

This strategic acquisition strengthens MaxCyte’s ability to serve ex vivo and in vivo cell and gene therapy (CGT) developers with an innovative suite of tools and services spanning early R&D through clinical development and commercialization. By integrating SeQure Dx into MaxCyte, Maxcyte will expand its service offerings and leverage its commercial and field application scientist teams to work with developers earlier in their research processes. SeQure Dx is revenue generating and expected to be accretive to MaxCyte’s revenue growth.

MONTGOMERY COUNTY, Md. — Businesses in Maryland’s Montgomery County experienced a surge in investment funding in 2024. Mergers and acquisitions, venture capital and private funding deals totaled $2.9 billion, a 36% increase over 2023’s total of $2.2b. Spanning 106 deals across 96 companies, this surge in mergers and acquisitions, venture capital, and private investment reflects Montgomery County’s continued prominence as a regional economic leader. 

Diverse Industries Driving Growth 

HANOVER, MD, Jan. 27, 2025 (GLOBE NEWSWIRE) — Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) (“Processa” or the “Company”), a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs with improved efficacy and safety, today announced the pricing of its “reasonable best efforts” public offering with participation from the Company’s Chief Executive Officer, certain board members, and existing institutional investors of the Company, along with a healthcare focused institutional investor, consisting of 8,050,672 shares of common stock (or pre-funded warrants in lieu thereof) and accompanying Series A warrants to purchase up to 8,050,672 shares of common stock (or pre-funded warrants in lieu thereof) and Series B warrants to purchase up to 4,025,336 shares of common stock at a combined purchase price per share (and accompanying warrants) of $0.615 for the institutional investors and $0.7975 for the Company’s Chief Executive Officer and certain board members.

ETC Baltimore, dedicated to elevating Baltimore as a national leader in tech startups, announces the opening of its inaugural ETC Venture Hub at Connect Labs Baltimore in the newly constructed 4MLK Building in the University of Maryland BioPark. This milestone marks a significant step in Baltimore’s efforts to strengthen its ecosystem for life science startups and attract high-potential companies to the city.

The ETC Venture Hub at Connect Labs Baltimore is a dedicated space designed to provide early-stage life science companies with the technology, capital, talent and resources needed to successfully grow and scale. Offering desk space, expert support, and a collaborative community, the hub serves as a launchpad for startups. This is part of ETC’s broader strategy to establish multiple footprints throughout the city. By expanding its reach, ETC aims to create a more inclusive and robust entrepreneurial ecosystem, ensuring innovators in all areas of Baltimore have access to the tools and support they need to thrive.

LEIDEN, Netherlands, January 28, 2025 / Biotech Newswire / — MIMETAS, a global leader in human disease modeling, has launched OrganoPlate UniFlow (UF), the first commercially available unidirectional, gravity-driven pumpless flow system for drug discovery and disease research. This innovative platform debuts at SLAS 2025 as a key addition to MIMETAS’ service portfolio.

OrganoPlate UF addresses challenges in traditional flow systems by enabling physiologically accurate tissue models that replicate natural biological conditions. With unidirectional, gravity-driven flow, this system eliminates the need for external pumps, simplifying operation while enhancing model accuracy. Supporting up to 512 chips in a single setup, the technology is ideal for rigorous scientific studies and large-scale screening, accelerating research without compromising biological relevance.

In this episode of BioTalk with Rich Bendis, Dr. Kolaleh Eskandanian, Vice President and Chief Innovation Officer at Children’s National Hospital, discusses her work driving pediatric healthcare innovation. Dr. Eskandanian introduces the BARDA SPARK Accelerator, a groundbreaking initiative focused on advancing medical countermeasures for children, and explains how it aligns with Children’s National’s mission to lead in pediatric healthcare innovation.

She also highlights the unique challenges in developing pediatric medical countermeasures and the importance of partnerships with organizations like Rainbow Babies, Mass General, and others. Additionally, Dr. Eskandanian shares insights into the hospital’s role in fostering innovation through the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) and the Children’s National Research and Innovation Campus.

Join us for an engaging conversation about the future of pediatric healthcare, the opportunities for innovation, and how the SPARK Accelerator is paving the way for advancements that will improve the lives of children worldwide.

Listen now via your favorite podcast platforms:
Apple: https://apple.co/4gcz9L3
Spotify: https://spoti.fi/3PHRYuP
Amazon Music: https://amzn.to/4h7PzWI
YouTube Music: https://bit.ly/4hp4WK0
TuneIn: https://bit.ly/42oIoVk

CES2025 once again brought the world’s brightest minds and groundbreaking innovations to Las Vegas. With over 4,500 exhibitors and more than 141,000 attendees, the event showcased technologies poised to shape industries across the globe. Among these, the health and wellness sector, centered at the Venetian Expo Center, stood out as a beacon of innovation. John Reinhart, Entrepreneur-in-Residence (EIR) at BioHealth Innovation (BHI), attended alongside colleagues from the National Institute on Aging (NIA) to explore the transformative field of AgeTech.

AgeTech—focused on enhancing the quality of life for an aging population—has rapidly emerged as a critical area of innovation. With 1 in 4 people currently over the age of 50, and that number projected to rise to 1 in 3 by 2050, the need for solutions that address aging-related challenges is urgent and global. Reinhart’s insights from CES2025 underscored the immense potential of this growing field.

The AgeTech Collaborative from AARP led the way in promoting innovation at CES2025. With its pavilion now in its third year, AARP highlighted the collaborative ecosystem it has built to foster solutions for the 50+ demographic. Boasting 111 investor organizations, 105 business services partners, 68 testbed collaborators, and 177 startups, the growth of the Collaborative has been remarkable. Its programming featured a pitch competition and networking reception, providing a platform for startups to connect and showcase their solutions.

COLLEGE PARK, Md., January 14, 2025–(BUSINESS WIRE)–IonQ (NYSE: IONQ), a leader in the quantum computing and networking industries, today announced a landmark partnership with the University of Maryland and the State of Maryland with the aim to establish Maryland as the “silicon valley” of the quantum computing industry. The proposed initiative will aim to fuel the growing quantum economy of the U.S. capitol region, provide access to IonQ’s cutting-edge quantum technology, and dramatically strengthen Maryland’s workforce with new quantum-focused jobs.

As part of today’s announcement of the “Capital of Quantum” initiative’s goal of catalyzing more than $1 billion in investments towards Maryland becoming the “Capital of Quantum,” IonQ intends to anchor a state-of-the-art quantum intelligence campus based at the University of Maryland at College Park.

GAITHERSBURG, Md., Jan. 14, 2025 (GLOBE NEWSWIRE) — Emergent BioSolutions Inc. (NYSE: EBS) announced today that it has entered into an agreement to obtain exclusive commercial rights in the U.S. and Canada to Hikma Pharmaceuticals’ KLOXXADO^® (naloxone HCl) Nasal Spray, an 8 mg naloxone agent that is approved for the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and/or central nervous system depression.¹ This six-year agreement complements and strengthens Emergent’s mission to protect, enhance, and save lives by helping to reduce opioid overdose deaths —providing compelling product options, combined with a commitment to increasing naloxone access, awareness, and education.

AIN becomes the second selected VCLP to support the next generation of technology innovation

COLUMBIA, Md., (January 15, 2025) — TEDCO, Maryland’s economic engine for technology companies, announces the selection of AIN Ventures as one of the early-stage venture capital fund managers supporting the management and investment of up to $10 million in U.S. Department of Treasury State Small Business Credit Initiative (SSBCI) funding.

“As a mission-driven organization, we are excited to work with early-stage entrepreneurs and TEDCO to strengthen Maryland’s innovation ecosystem and support the successful deployment of valuable SSBCI funding,” said Sherman Williams, co-founder and managing partner of AIN Ventures.

NORTH CHICAGO, Ill. and ROCKVILLE, Md., Jan. 13, 2025 /PRNewswire/ — AbbVie (NYSE: ABBV) and REGENXBIO Inc. (Nasdaq: RGNX) today announced updates to the ABBV-RGX-314 clinical program.

ABBV-RGX-314 in Wet Age-Related Macular Degeneration (wet AMD), Subretinal Delivery
Data from the ATMOSPHERE^® and ASCENT^™ pivotal trials evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with wet AMD are expected in 2026.

ABBV-RGX-314 in Diabetic Retinopathy (DR), Suprachoroidal Delivery
AbbVie and REGENXBIO will plan a Phase 3 clinical program. The clinical program will utilize the in-office SCS Microinjector^® to deliver gene therapy to the suprachoroidal space of the eye.

January 13, 2025 – By Ruxandra Draghia-Akli, MD, PhD, Executive Vice President, Head of R&D and Elaine O’Hara, Executive Vice President, Chief Strategy Officer

As we enter the new year, Novavax is embarking on an ambitious new corporate growth strategy to maximize the impact of our cutting-edge technology. We are focused on forging strategic partnerships for both our early- and late-stage research and development (R&D) assets and our Matrix-M™ adjuvant with the goal of driving vaccine innovation that protects the health of people.

Our Innovative Technology Platform

Novavax’s technology platform combined with our deep vaccine expertise is the fuel for future innovation and partnerships. The unique platform features two key elements:

• Recombinant protein-based nanoparticle: This technology enhances immune recognition by presenting multiple copies of viral antigens on the surface of each nanoparticle. Antigens displayed in this manner tend to lead to greater stimulation of the immune system, resulting in a more efficacious vaccine.^1-3
• Matrix-M™ adjuvant: Our proprietary adjuvant works together with the nanoparticle to induce potent, durable and broad immune responses, with the potential to be antigen-sparing.^4-7 Matrix-M increases both antibody and cell-mediated immune responses to the vaccine, and has demonstrated a favorable tolerability and safety profile in clinical trials across a variety of different antigens.⁷ Matrix-M is used in our authorized COVID-19 vaccine and the R21/Matrix-M malaria vaccine.

Like his fellow Type 1 diabetics, Alec Smith took insulin every day to regulate his blood sugar. Costs to treat his condition topped $1,000 per month, most of that going to insulin, and Alec made the difficult decision to ration his insulin to save money after aging off his mother’s health insurance plan.

Rationing insulin is a delicate process for diabetics. The body’s insulin needs can fluctuate significantly due to changes in diet, exercise, stress, poor sleep, or other medications, and diabetics must constantly monitor their glucose levels even when taking insulin as directed. When a person’s blood sugar gets too high, the body goes into diabetic ketoacidosis, which releases dangerous amounts of acid into the bloodstream and, if left untreated, can result in death — as it unfortunately did for Alec Smith in 2017. His mother, Nicole Smith-Holt, told CBS News, “I think if the price of insulin in 2017 had been $35, Alec would still be alive today.”

ROCKVILLE, Md., Jan. 7, 2025 /PRNewswire/ — Essex Management, an Emmes Group company and a leader in biomedical informatics and health information technology, is pleased to announce the appointment of David Loose as Chief Executive Officer (CEO), effective immediately. David succeeds Kevin Hurley, who has served as CEO since February 2019 and will transition to the role of Executive Advisor.

David Loose joined Essex Management as a partner in February 2009 and has played a pivotal role in the company’s growth. Starting as Vice President for Business Operations and Development, he later served as Chief Business Officer, overseeing critical operations, driving client-focused innovation, and delivering strategic initiatives such as NCI’s Clinical Trials Reporting Program (CTRP). His leadership and dedication to Essex’s mission have been instrumental in building its reputation as a trusted partner in the biomedical and health technology sectors.

“David’s leadership and dedication to Essex’s mission make him the ideal choice to guide the company forward,” said Sastry Chilukuri, CEO of Emmes Group. “His focus on innovation and client success will ensure Essex continues to deliver transformative solutions in biomedical informatics and health IT.”

*NOTE: Solution Summary due: February 14, 2025
 

The Big Question  

What if we could end the rare disease diagnostic odyssey?

The Problem  

Collectively, rare diseases are far from rare — more than 10,000 unique conditions affect over 350 million people worldwide, including one in ten Americans. The lengthy diagnostic “odyssey” endured by patients with a rare disease lasts six years on average but can extend for decades. Diagnostic delays stem from multiple factors, including overlapping symptoms, low disease incidence, and limited specialist expertise. It’s estimated that half of all individuals with a rare disease remain undiagnosed or misdiagnosed, leading to inappropriate care, irreversible disease progression, and rising medical costs.