ROCKVILLE, Md., Jan. 14, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) today announced a strategic partnership for the development and commercialization of RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome.

Under the terms of the agreement, REGENXBIO will receive $110 million at closing and up to an additional $700 million if certain milestones are achieved, consisting of $40 million in potential development and regulatory milestones and $660 million in potential sales milestones. REGENXBIO will also receive meaningful double-digit royalties on net sales in the U.S. and Asia (collectively, the "Licensed Territory").

GO Virginia, University of Virginia, Manning Family Foundation, and local Economic Development support the three-year project driving life sciences entrepreneurship.

Charlottesville, VA (January 14, 2025) --- The biotechnology and innovation landscape in Virginia is set for significant growth, thanks to a recent award from the state’s GO Virginia economic development program and a strategic partnership between CvilleBioHub and the University of Virginia (UVA).This award will inject a crucial business component into Virginia’s existing biotech ecosystem, bolstering the success of high-potential, early-stage life sciences companies. GO Virginia has allocated a $14.3 million funding package to three biotech organizations across Virginia as part of the multiregional initiative “Project VITAL: Virginia Innovations and Technology Advancements in Life Sciences.”

January 14, 2025 - Vicky Hatch - Osteosarcoma is a type of aggressive bone cancer that most commonly affects children and young adults between the ages of 10 and 20, during times of rapid bone growth. Although rare, it has a significant impact on young people and their families as treatment can require surgery or amputation. The cancer also has the potential to spread to other organs, most commonly the lungs. Because osteosarcoma is so genomically complex, it has been challenging to identify what genetic mutations drive the disease. As a result, there has been little advancement in treatment options over the past 40 years. 

New research, published in the journal Cell, solves the mystery of what drives the genomic rearrangements causing the aggressive development and evolution of osteosarcoma tumours. By analysing the largest collection of whole-genome data from osteosarcoma patients, the researchers identified a new mutation mechanism, called loss-translocation-amplification (LTA) chromothripsis, which is present in approximately 50% of high-grade osteosarcoma cases. 

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.

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.”

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.

Read more ...

GAITHERSBURG, Md., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) announced today that the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, executed a contract modification for the second option period, valued at approximately $16.7 million, for drug product process and analytical testing validation and long-term stability for Ebanga™ (ansuvimab-zykl). Ebanga™ is indicated for the treatment of infection caused by Zaire Ebola virus.

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*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.  

Read more ...

COLLEGE PARK, Md.--(BUSINESS WIRE)-- The following is a statement from Peter Chapman, Chairman and CEO of IonQ (NYSE: IONQ):

Today’s classical computing hardware is limited by computational capacity and power requirements in ways that will likely prohibit society from ever being able to solve some of its most pressing problems.

IonQ’s current #AQ 36 Forte Enterprise systems are already providing insight to solutions for customers today, and our upcoming #AQ 64 Tempo systems in 2025 and next-generation #AQ 256 systems will enable us to tackle increasingly complex problems to deliver near-term business value. One of the areas facing the most significant potential disruption is strong AI, where we believe natively quantum AI will outperform classical AI.

BELTSVILLE, Md., Jan. 10, 2025 (GLOBE NEWSWIRE) -- NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer, today announced the first patient has been dosed in its Phase 1 study of LNCB74, a B7-H4-targeting antibody-drug conjugate (ADC) as a therapeutic for treating multiple cancers.

FREDERICK, Md., Jan. 9, 2025 /PRNewswire/ -- Veralox Therapeutics, a clinical-stage biotechnology company developing a new class of therapies targeting the 12-lipoxygenase (12-LOX) pathway, announced today that it has entered into an exclusive agreement with Nudge Therapeutics to acquire the company and their preclinical cyclic AMP-GMP (cGAS) inhibitor compounds.  The agreement allows Veralox to continue development of Nudge's compounds and trigger acquisition of the company upon achievement of downstream milestones. Financial terms were not disclosed.

GERMANTOWN, Md., & VENLO, The Netherlands--(BUSINESS WIRE)--QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced the U.S. regulatory clearance of the first in a series of QIAstat-Dx Gastrointestinal Panel tests for clinical use.

Montgomery County, MD — The Montgomery County Economic Development Corporation (MCEDC) announced that Bill Tompkins will step down as President & CEO when his term ends in August 2025. Tompkins has served in this role since August 15, 2022.

“This marks a significant moment for MCEDC,” stated Elana Fine, Chair of the Board of Directors. “We extend our gratitude to Bill for his outstanding leadership over the past six years, as both the Chief Operating Officer and as President & CEO. His dedication has laid a strong foundation for future growth while fostering inclusion, driving collaboration, and ensuring stability during challenging times.”

In an unprecedented new study in the journal Cell Reports, researchers have shown neurotransmitters in the human brain are released during the processing of the emotional content of language, providing new insights into how people interpret the significance of words.

The work, conducted by an international team led by Virginia Tech scientists, offers deeper understanding into how language influences human choices and mental health.

Spearheaded by computational neuroscientist Read Montague, professor of the Fralin Biomedical Research Institute at VTC and director of the institute’s Center for Human Neuroscience Research, the study represents a first-of-its-kind exploration of how neurotransmitters process the emotional content of language — a uniquely human function.

The Washington D.C., Maryland, and Virginia (DMV) region has quickly become one of the nation’s top destinations for startups and tech innovation. With over 270,000 tech jobs and $5 billion in venture capital raised in 2023, the DMV stands out as a vibrant and rapidly growing ecosystem.

In a recent LinkedIn post, Christon Hill, Program Manager at Georgetown Tech Ventures (GTV) and the Office of Technology Commercialization (OTC) at Georgetown University, highlights the factors driving this growth. He points to the region’s proximity to federal agencies, cutting-edge research institutions, and a culture of collaboration as key ingredients for success.

GAITHERSBURG, Md., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) today announced the exercise of contract option and modification valued at approximately $20 million to supply BioThrax® (Anthrax Vaccine Adsorbed) to the U.S. Department of Defense (DoD). The first delivery, which was valued at approximately $7 million began in December 2024, and remaining deliveries are expected in 2025.

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."

COLLEGE PARK, Md.--(BUSINESS WIRE)-- IonQ (NYSE: IONQ), a leader in the quantum computing and networking industry, today announced the completion of its acquisition of substantially all of the assets of Qubitekk, Inc., a prominent quantum networking company. The acquisition brings Qubitekk’s esteemed team, advanced technology, and extensive patent portfolio into IonQ’s operations, solidifying IonQ’s position at the forefront of quantum networking and computing.

Quantum networks are poised to become important infrastructure, with the potential to drive transformative applications in sectors such as defense, finance, and energy. These networks facilitate highly secure communication between multiple quantum nodes using entangled qubits. The technology also enables precise timing synchronization, a transport layer for quantum sensing, and a backbone for distributed quantum computing. With the development and 2022 launch of the EPB Quantum Network in Chattanooga, Tennessee—the first commercially available quantum network in the U.S.—Qubitekk has established itself as a leader in quantum networking.

ROCKVILLE, MD, Jan. 6, 2025 /PRNewswire/ -- I-Mab (NASDAQ: IMAB) (the "Company"), a U.S.-based, global biotech company, focused on the development of precision immuno-oncology agents for the treatment of cancer, today highlighted its strategic outlook for 2025 and a re-prioritization of resources, with a focus on advancing its lead program, givastomig, a CLDN18.2 x 4-1BB bispecific antibody, targeting first-line metastatic gastric cancers, with further potential in other solid tumors.

In this episode of BioTalk, Bill Tompkins, President and CEO of the Montgomery County Economic Development Corporation (MCEDC), discusses the strategies driving Montgomery County’s position as a leading bioscience hub. Bill highlights MCEDC’s role in reinforcing the region’s standing as the third-largest bioscience cluster in the U.S. and shares insights into the new Institute for Health Computing. He also introduces two new venture funds—the Technology Innovation Fund and the Founders Fund—designed to accelerate innovation and support local entrepreneurs.

Bill outlines MCEDC’s priorities for the upcoming year, emphasizing economic growth, investment opportunities, and fostering collaborations that make Montgomery County a thriving ecosystem for business and life sciences.

Listen now via your favorite podcast platforms:

Apple: https://apple.co/4j6AA0e
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TuneIn: https://bit.ly/427OmtP

Kolaleh Eskandanian, chief innovation officer at Children’s National Hospital, said the partnership is meant to address the many challenges with developing devices for children and infants.

Published Jan. 2, 2025 - Elise Reuter, Reporter

A collaboration between a children’s hospital and the Food and Drug Administration aims to address challenges in developing medical devices for children and infants. 

Children’s National Hospital in Washington, D.C., struck a five-year research collaboration with the FDA’s Office of Science and Engineering Laboratories (OSEL) to build regulatory science tools to help evaluate pediatric devices. 

The partners will use de-identified clinical data, multimodal imaging and machine learning to develop open-source tools that can be used to design and test devices more efficiently. 

BHI is proud to support ARPA-H through its Partnership Intermediary Agreement (PIA), providing EIR commercialization services to advance groundbreaking technologies. Notably, BaySpec, one of BHI’s clients, was awarded ARPA-H’s first SBIR grant to develop cutting-edge diagnostics. Andy Kilianski, Program Manager at ARPA-H, recaps key 2024 milestones, including AI-driven drug discovery, RNA therapies, and the $204M APECx biologics program. These efforts underscore ARPA-H’s commitment to accelerating biomedical innovation.

Portfolio Views from 2024
Andy Kilianski
Program Manager at ARPA-H

December 30, 2024
Happy Holidays Everyone!

2024 was a BIG year for my team and our investments in the biomedical and biotechnology ecosystem. Our goal is to invest in transformative technology that allows biomedical R&D to be predictive of human outcomes, in ways that are currently impossible. I want to capture some of our key milestones (in semi-chronological order) to highlight the amazing work already underway with our team at the Advanced Research Projects Agency for Health (ARPA-H) . My team and our performers have been incredibly diligent and dedicated to get us to this point. There are a lot of efforts soon-to-be-announced from us as we head into next year, including the kick-off of the CATALYST program mid-2025.

Precigen has submitted a Biologics License Application (BLA) to the FDA, seeking priority review for PRGN-2012, which could become the first FDA-approved treatment for adults with recurrent respiratory papillomatosis (RRP)—a rare and chronic disease currently managed through repeated surgeries. PRGN-2012 has received Breakthrough Therapy and Orphan Drug Designations from both the FDA and the European Commission. The application, submitted under an accelerated approval pathway, is backed by Phase 1/2 study data showing over 50% of patients achieved Complete Response and more than 85% experienced fewer surgeries in the year following treatment. PRGN-2012 was also well-tolerated, with no dose-limiting toxicities and no severe treatment-related adverse events.

National Cancer Institute (NCI) has awarded a $357,000 SBIR grant to Arnold Consultancy & Technology, LLC to develop an open source model marketplace.

MIAMI BEACH, FL, UNITED STATES, December 30, 2024 /EINPresswire.com/ -- New cancer drugs have long been at the forefront of research and debate about rising healthcare expenditure in the United States; researchers and decision-makers rely on evidence to support fair pricing and ensure patient access. HE models support stakeholders in decision-making about the most cost-effective care for the available resources. Annually, stakeholders spend an estimated $1B on health economics and outcomes research (HEOR), much of it to create and recreate health economic (HE) models. Global government bodies use these models to formulate response and reimbursement decisions, aid resource allocation and suggest value-based pricing for new therapies. Sharing models by making their “source code” openly available would mitigate these problems, but prior initiatives to promote model sharing have had limited success in part because model authors fear the loss of their intellectual property and are not paid for their models being deposited.

BHI is excited to highlight Rachel Rath’s recent LinkedIn Post, where she reflects on her first 90 days as Head of JLABS @ Washington, DC. Rachel, who recently joined the BHI Board of Directors, discusses the Mid-Atlantic’s strengths—including its talent pool, investor activity, and opportunities for deeper collaboration.

We look forward to continuing to work with Rachel and JLABS to drive growth and innovation in the BioHealth Capital Region.

90 Days In: Reflections on an Evolving Life Science Landscape in the Mid-Atlantic

Children’s National Innovation Ventures is hosting the Symposium on Pediatric Medical Countermeasures (MCMs) and the SPARK for Innovations in Pediatrics Launch and Prize Competition—a premier event under the BARDA Accelerator Network 2.0. This exciting gathering will focus on advancing pediatric-focused MCMs, featuring insightful discussions and a live competition where finalists will pitch their groundbreaking solutions for a chance to secure non-dilutive funding from SPARK.

The event will also include a ribbon-cutting ceremony to officially launch the SPARK Hub, followed by a cocktail reception to celebrate this milestone in pediatric innovation.

BioHealth Innovation (BHI) is a proud partner in this initiative, supporting efforts to drive advancements in pediatric medical countermeasures and foster impactful innovation.

Event Details:

• Location: Children's National Research and Innovation Campus, 7144 13th Pl NW, Washington, DC 20012
• Date and Time: Tuesday, January 28, 2025, from 3:00 PM to 7:00 PM

For more information, visit https://lp.constantcontactpages.com/ev/reg/2p7f9t5.

Join us in celebrating this important step toward transforming the future of pediatric healthcare!

Italy, December 23, 2024. PQE Group is proud to announce its most recently-received official recognitions: ISO/IEC 27001 and A Women’s Business Enterprise (WBE) Certifications, which provide additional reasons to recognize PQE Group’s extensive capabilities; the ISO/IEC 27001 provides clients assurance that their information management systems are risk-aware and that PQE Group’s solutions can proactively identify and address weaknesses; the WBE Certification ensures that PQE Group has met the stringent eligibility criteria to be classified as a women-owned enterprise.

DEC 20, ANNAPOLIS, MD — Governor Wes Moore today signed an executive order to strengthen Maryland’s business climate and catalyze more economic growth in the state. The governor signed the order during a visit to the Frederick Hotel and Conference Center project site in Frederick—a public-private development project that is expected to generate $1.5 billion in new private sector spending over the next 25 years, more than 200 jobs, and more than $4 million in state and local tax revenue annually. 

SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced the world’s first transplant of a UKidney, which it produced, into a living person on November 25, 2024.

Guillermo Flor -
How to pitch to investors
The basics:
1. Venture Capitalists have an investment thesis they have to follow. If your startup doesn’t fit the investor’s thesis don’t bother pitching

2. Most times you’ll start pitching to associates. Then the associates will have to share the opportunity with the rest of their investment team. The easier you make their job the more chances you have.

3. How you pitch your startup gives out a lot of information about you to investors. Make sure your investment materials are organized, your pitch is concise and to the point and you are likeable.

GAITHERSBURG, Md., Dec. 16, 2024 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) today announced that the Biomedical Advanced Research and Development Authority (BARDA) within the Administration for Strategic Preparedness and Response (ASPR) at the U.S. Department of Health and Human Services has awarded a $50 million option to Emergent’s existing contract (HHSO100201600030C) for the acquisition of CYFENDUS^® (Anthrax Vaccine Adsorbed, Adjuvanted).

GAITHERSBURG, Md., Dec. 13, 2024 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a global company advancing protein-based vaccines with its Matrix-M™ adjuvant, today announced progress in Q4 2024 advancing its corporate growth strategy through its partnership with Sanofi. The Company has achieved a milestone associated with its Phase 2/3 clinical trial for its COVID-19 vaccine in children, triggering the first $50 million milestone payment from Sanofi.