With an increasing number of U.S. biopharmaceutical firms feeling the financial constraints imposed by the Inflation Reduction Act (IRA) and subsequently shutting down or suspending their research programs in the rare disease domain, hundreds of millions of patients in both the U.S. and India face the grim prospect of not receiving a breakthrough treatment for their condition within their lifetimes. IndoUSrare, a US-based nonprofit committed to stanching rare diseases, launches the inaugural Bridging RARE conference to forge a path for affordable clinical innovation via strengthened Indo-US collaborations and more inclusive clinical trials for minimizing the devastating health consequences arising from this policy shift.

(HERNDON, Va.) October 10, 2023: Rare diseases, as defined by the World Health Organization (WHO), refer to some 10,867 medically significant conditions, each with a prevalence of one or less per 1,000 individuals. With only about 8% of them being treatable,¹ rare diseases include congenital malformations, autoimmune disorders, lysosomal storage disorders, thalassemia, muscular dystrophies, and infrequent forms of cancer and infections. Despite substantial progress during the 40 years since the enactment of the Orphan Drug Act of 1983, which incentivized the U.S. private sector to develop 1,100 FDA-approved orphan treatments,² the global thrust against rare diseases is on the verge of losing steam.

Imagine a world where unhealthy organs are replaced with transplants constructed with a 3D printer using the patient’s own cells. No more agonizing waits and desperate searches for a suitable donor organ, and, because the printed organs use cells from the patients themselves, no more fear of organ rejection. It might sound like science fiction set in the far off future, but thanks to the pioneering work of United Therapeutics , it may become a reality a lot sooner than you think, and it’s happening here in Maryland.

Co-headquartered in Silver Spring, United Therapeutics was founded in 1996 by Dr. Martine Rothblatt, a mom who wanted to save her 7 year-old daughter Jenesis who’d been diagnosed with a rare and serious disease called pulmonary arterial hypertension. Twenty seven years later, Jenesis has defied the odds and works at the company her mother founded to save her life.

Alex Philippidis, Senior Business Editor of Genetic Engineering and Biotechnology News, for the 4^th consecutive year, attended the 9th Annual BioHealth Capital Region Forum, presenting his expert insights into the “Top 10 U.S. BioPharma Clusters, 2023,” providing a detailed analysis and overview of the current state and future prospects of the BioPharma industry.

In his presentation, Alex highlighted the following:

Technological Innovations in BioPharma: Highlighting the increasing influence of Artificial Intelligence (AI) in BioPharma, Alex explored how these technological advancements are critically shaping research, development, and operational processes within the sector.

In the 9th Annual BioHealth Capital Region Forum, Pete Briskman, Executive Managing Director and Co-Lead for the Mid-Atlantic Life Sciences Practice, offered a thorough update on the BioHealth Capital Region (BHCR) and its significant strides in the life sciences sector.

BHCR’s Noteworthy Achievements:

• Securing the #3 rank among Life Science Clusters.
• Leading the nation in the number of biotechnology patents.
• Holding a prominent third position in total NIH grant funding.
• Establishing itself robustly as the third-largest science cluster.

Advances Amgen's Mission to Serve Patients With First-in-Class Rare Disease Medicines

THOUSAND OAKS, Calif., Oct. 6, 2023 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced that it has completed its acquisition of Horizon Therapeutics plc for $116.50 per share in cash, representing a transaction equity value of approximately $27.8 billion.

"Today marks an exciting milestone as we welcome Horizon employees to Amgen and begin working together to serve even more patients around the world suffering from serious illnesses," said Robert A. Bradway, Amgen's chairman and chief executive officer. "We have strong momentum in our core business and the addition of Horizon will further position Amgen as a leader across a broader range of diseases."

In a year that underscored “Global Breakthroughs for a Healthier Future,” Rich Bendis, President and CEO of BioHealth Innovation Inc (BHI), extended a heartfelt welcome to attendees, marking the 9th consecutive year of the Annual BioHealth Capital Region Forum.

Take a moment to reflect on Rich’s thorough review of the region’s transformative journey from its inception to its present-day stature, proudly holding a #3 ranking in the GEN Top 10 BioPharma Clusters in the United States. This achievement is a testament to its prowess in NIH Funding, Venture Capital Funding, Patents, Lab Space, and Jobs.

Rich highlighted the global impact of the term “BioHealth,” which has become more woven into the fabric of health discussions and innovations over nearly a decade.

Projects will broaden opportunities for traditionally underrepresented students in STEM

In a historic first for Montgomery College (MC), three different teams of faculty and administrators have won grant awards from the National Science Foundation (NSF) totaling over $3.24 million. The grants include EmpowerED: Building the Future Workforce Together, part of the new Enabling Partnerships of Increase Innovation Capacity (EPIIC) program; Beginnings: Democratizing Research and Experiential Education for Microelectronics (DREEM), part of the new Experiential Learning for Emerging and Novel Technologies (ExLENT) program; and AUSEM: Achieving Upward Socio-Economic Mobility, part of the competitive Scholarships in Science, Technology, Engineering and Mathematics program. Each project begins this month, and will expand opportunities for students who have been traditionally underrepresented in STEM fields.

ROCKVILLE, MARYLAND, October 5, 2023 – BioHealth Innovation, Inc. (BHI) is proud to announce a pivotal agreement with the Advanced Research Projects Agency for Health (ARPA-H) to provide Entrepreneurs-in-Residence (EIRs) in support of ARPA-H's mission. This collaboration aims to bolster the development and commercialization of transformative health solutions.

Under the agreement, BHI's EIRs will offer strategic guidance to ARPA-H Program Managers (PMs) in areas including technology development, intellectual property, and small business needs. They will assist in the development and execution of programs, evaluate partnership opportunities with government biomedical and health research agencies, and leverage industry associations to support funded performers.

Welldoc’s industry-leading holistic platform now incorporates AI-driven digital coaching and features specifically designed to support individuals throughout the complex journey of weight management

COLUMBIA, Md., October 4, 2023 — Welldoc^®, a digital health leader revolutionizing chronic care, today announced the expansion of its platform to support weight management. Welldoc’s comprehensive chronic care platform now provides multi-condition support across prediabetes, diabetes, hypertension, heart failure and weight management, along with integrated mental wellbeing and sleep apnea support. Welldoc’s weight management solution is currently available to all customers and will be launching in early 2024 with two of the leading health care companies in the U.S.

Nearly two-thirds of U.S. adults are overweight or obese, which increases their risk of developing chronic comorbid conditions like type 2 diabetes, hypertension, heart failure, and sleep apnea.  As part of Welldoc’s total health approach to chronic care, AI-driven, personalized digital coaching is available 24/7 to help individuals navigate weight management whether they are just beginning their weight management journey with lifestyle changes or are prescribed a GLP-1 medication. Welldoc enables individuals to understand and connect with their health data in a meaningful way, making the achievement of weight and overall health goals more attainable. Welldoc also provides clinical escalations and decision support for the care team when needed.

VaxTRIALS Adds a New Latin America Base and Greater Vaccine Research Depth

Rockville, MD, October 5, 2023 – Emmes, a global, full-service Clinical Research Organization (CRO) dedicated to supporting the advancement of public health and biopharmaceutical innovation, today announced the acquisition of VaxTRIALS. Headquartered in Panama City, Panama, VaxTRIALS’ 160+ staff members manage and monitor vaccine clinical trial activities throughout Latin America.

“Today’s clinical trials are both more complex and more global in nature than ever before,” said Emmes Executive Chairman Sastry Chilukuri. “Combining VaxTRIALS’ depth of expertise in Latin America and Emmes’ strengths, particularly in technology, biostatistics and data management, will give clients an unmatched partnership experience in conducting their clinical trials.”

First small molecule allosteric binder discovered with Gain’s computational drug discovery platform enters clinical development

BETHESDA, Md., Oct. 04, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced dosing of the first two subjects in a Phase 1 clinical trial of GT-02287, Gain’s lead drug candidate for the treatment of GBA1 Parkinson’s disease. The Company expects to complete this trial in the first half of 2024.

“Initiating first-in-human dosing with GT-02287 is an important milestone for Gain as we enter a new era as a clinical-stage company,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics. “I am very proud of the work accomplished by the entire Gain team to get us to this stage today, and we are eager to advance our understanding of the safety, tolerability and effect of GT-02287 in humans. This represents another major step forward toward providing a treatment for Parkinson’s patients and their families impacted by this devastating disease.”

GAITHERSBURG, Md., Oct. 3, 2023 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a global company advancing protein-based vaccines with its novel Matrix-M^™ adjuvant, today announced that the Novavax COVID-19 Vaccine, Adjuvanted (2023-2024 Formula) (NVX-CoV2601) has received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for active immunization to prevent COVID-19 in individuals aged 12 and older. Immediately upon authorization, Novavax's vaccine has also been included in the recommendations issued by the U.S. Centers for Disease Control and Prevention (CDC) on September 12, 2023.

Doses will be available nationwide at thousands of locations, including national and local retail pharmacies and physicians' offices, following the Center for Biologics Evaluation and Research release of vaccine batches, expected in the coming days.

ROCKVILLE, Md., Oct. 3, 2023 /PRNewswire/ -- Emmes, a global, full-service Clinical Research Organization (CRO) dedicated to supporting the advancement of public health and biopharmaceutical innovation, today announced that Matt Bond has become its chief financial officer (CFO).

Emmes Chief Executive Officer Peter Ronco said, "Matt combines a rich set of CFO experiences with a deep understanding of the biopharma and clinical technology solutions space. A seasoned executive with over 30 years of financial leadership experience, he has led the finance function for many successful, private-equity-backed companies in the pharmaceutical services and contract research market segment."

"This is a perfect time to join the company, as we continue to build our reputation with government and biopharma clients and ramp up our presence in new global markets," noted Bond. "I'm able to hit the ground running, because I've worked in this sector for decades and have been successful in generating profitable growth while partnering with great management teams."

Before joining Emmes, Bond was the CFO of Bracket (now Signant Health), a leading technology provider to the drug development market.  There, he led the organization through a period of sustained  growth, including a successful sale of the company to another global, private-equity-backed firm.  Before that, he was a founder and served as CFO of Aptiv Solutions,  a technology-focused CRO. Bond was part of the leadership team that grew the business from a startup to a large multinational organization through the acquisition of more than 10 companies. 

Previously, Bond was executive vice president and CFO of PRA, a global CRO, where he led a successful initial public offering. Earlier in his career, Matt was vice president of finance with Marriott International and a consultant with a leading, global financial consulting firm. He holds a B.S. degree from Wake Forest University.

Ronco said, "We are pleased to have Matt join our team. His hands-on experience with mergers and acquisitions will be a great help as we continue to identify, review and assimilate companies that complement our strengths and accelerate our global growth."

About Emmes

Founded more than 45 years ago, Emmes is a global, full-service Clinical Research Organization dedicated to excellence in supporting the advancement of public health and biopharmaceutical innovation. The company's clients include numerous agencies and institutes of the U.S. federal government and a wide range of biotechnology, pharmaceutical and medical device companies throughout the world. To learn more about how our research is making a positive impact on human health, go to the Emmes website at www.emmes.com.

Maryland has renewed its Memorandum of Understanding (MOU) with the Korea SMEs and Startups Agency, also known as KOSME.

As part of the MOU signed in 2021, Maryland and KOSME support Korean startups and small and medium-sized businesses interested in entering green and high-tech sectors in the U.S., with the potential for them to participate in Maryland’s Global Gateway program. The partnership also supports companies interested in the aerospace, biohealth, cybersecurity, and renewable energy industries.

The BioHealth Capital Region recently played host to a series of significant events that drew attention from the global biopharma community. Notably, several Israeli BioHealth companies, some traveling directly from Israel, participated in these events, underscoring the region's growing prominence, especially after being recognized by GEN as a Top 3 BioPharma Cluster in the United States. This remarkable participation was made possible with the dedicated efforts of Aisha Espey, Director of Commerce, Head of Science and Technology from the Embassy of Israel in Washington DC, and Andrea Yonah, Director of Business Development for the U.S. East Coast/Midwest at the U.S. Israel Binational Industrial R&D (BIRD) Foundation.

The BioHealth Capital Region Forum serves as the region's premier gathering for the biopharma sector, fostering collaborations and discussions that advance public health and economic outcomes. The Crab Trap, now in its 8th year, is a competition spotlighting entrepreneurs with transformative health solutions. The Investment Conference is a platform for companies and investors to create partnerships, featuring brief presentations highlighting BioHealth opportunities and innovations.

Innara Health has extended their recognition to BioHealth Innovation, Inc. (BHI) and Ethel Rubin for our significant contributions to Innara Health's journey. BHI is acknowledged for our confidence and insight in introducing Innara Health to the BioHealth Capital Region, while Ethel Rubin is thanked for her expert counsel, candor, and influential role on the Board, along with her valuable network. Our support has played a crucial part in Innara Health's mission to improve the lives of premature newborns and infants.

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How can patients help shape the future of scientific innovation? What's the vital role of the patient's voice in developing life-changing medicines? In this captivating episode, fresh off her thought-provoking appearance at the 9th Annual BioHealth Capital Region Forum, Dr. Theresa Podrebarac takes us on a journey through the dynamic landscape of autoimmune medicine.

Dr. Podrebarac is a renowned physician scientist with a wealth of expertise in the biotech industry. As the Senior Vice President of Clinical Development at Horizon Therapeutics, she's at the forefront of bringing hope to patients with rare, autoimmune, and severe inflammatory diseases. Join us as we explore pivotal questions:

• What is Horizon Therapeutics' mission, and what are Dr. Podrebarac's primary responsibilities in this quest for life-changing medicines?
• How has Dr. Podrebarac's background as a rheumatologist influenced her belief in the importance of the patient's perspective in drug development?
• Discover Horizon's impressive journey in clinical development, with a sharp focus on autoimmune diseases.
• Dive into Horizon's global presence and the crucial role of its Maryland operations.
• What are the clinical goals that will shape the future of autoimmune medicine?

The Greater Washington area has a long history of influential business leaders powering the growth of our region and the strength of their communities. On Dec. 5, the Washington Business Hall of Fame will welcome five such leaders to receive our region’s signature lifetime business achievement award. The Hall of Fame brings these visionaries (the best in business) to the table with the next generation of leaders (the next in business), through an experience that connects and inspires us all.

The 2023 Hall of Fame Laureates are no strangers to media coverage. If you live in the Washington region, chances are you’ve seen their names in headlines for years. What you may not have witnessed is these trailblazers telling their stories in their own words. That’s what this series brings you – the look back, lessons and life advice that these Laureates have to offer based on a lifetime of success.