Published: 10/10/2023

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.