In 1996, Martine Rothblatt’s six-year-old daughter was diagnosed with pulmonary arterial hypertension, a rare lung and heart disease without a cure. Undeterred, Rothblatt, a cofounder of Sirius Satellite Radio, launched her own biotech firm with the goal of finding one.

“There is nothing worse than being told that your daughter is going to die,” https://www.forbes.com/sites/chloesorvino/2018/06/20/how-ceo-martine-rothblatt-turns-moonshots-into-earthshots/" aria-label="Rothblatt told Forbes">Rothblatt told https://www.forbes.com/sites/chloesorvino/2018/06/20/how-ceo-martine-rothblatt-turns-moonshots-into-earthshots/">Forbes in 2018. “I just said I will find a way, or she was going to die, because all of the previous people with this illness had died.”

Nearly three decades later, Rothblatt’s daughter is healthy and in her 30s. Meanwhile, shares of the now publicly traded firm, United Therapeutics, are up 54 times its price at its 1999 IPO. This year alone, shares have surged, up 50% so far in 2024 and up 40% since April 30—enough to make Rothblatt the world’s newest billionaire.

By: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research

An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments. Fundamental to the mission of the U.S. Food and Drug Administration is to engage patients and caregivers – to understand their unique perspectives and experiences and keep these front of mind as we review medical products for rare disease patients.

The Rare Disease Innovation Hub
Recent rapid advances in the identification of promising drug targets and development of gene therapies offer momentum and potential to meet the needs of patients with rare diseases. In 2023, over half of all the novel drugs and biologics approved by the FDA’s Center for Drug Evaluation and Research (CDER) and the FDA’s Center for Biologics Evaluation and Research (CBER) were to prevent, diagnose or treat a rare disease or condition.

D.C.-based maternal mental health startup Seven Starling has raised $10.9 million in an oversubscribed round to expand its virtual care services — which include supporting postpartum, early parenthood, infertility, miscarriage and loss and other experiences — further across the country.

San Francisco women’s health-focused venture firm RH Capital led the round and existing investors Menlo Park, California's Pear VC; San Francisco’s Expa; Santa Monica, California’s Magnify Ventures; Palo Alto, California’s Emerson Collective; and Emeryville, California’s Inflect Health also took part.

Emerson Collective is led by Laurene Powell Jobs, who is an investor in D.C.'s Monumental Sports & Entertainment, the owner of the Washington Wizards, Capitals, Mystics and other sports ventures. Emerson counts The Atlantic magazine, which is based in the District, among its holdings.

COLUMBIA, Md., (July 16, 2024) — TEDCO, Maryland’s economic engine for technology companies, announced a recent $500,000 investment in OmniSpeech. This investment was made in two parts, with $300,000 coming from TEDCO’s Venture Funds and the remaining $200,000 coming from TEDCO’s Social Impact Funds.

“After decades of research and development, we are excited to expand our reach to improve more apps and devices with AI/ML-powered speech,” said Carol Espy-Wilson, founder and chief technology officer at OmniSpeech. “With TEDCO’s generous investments, we are excited to advance our innovative AI and machine learning technologies.”

Foreign investors highlighted innovative and high-quality human resources as a strength of Korean biotech companies. They also recognized advantages in Korea’s active government investment and robust infrastructure for clinical trials and development. However, they also pointed out that Korea’s conservative corporate culture and limited funding needed improvement.

During the 'Bioplus-Interphex Korea 2024 (BIX 2024)' conference at COEX in Samsung District, Seoul on July 12, in a special session themed 'The charm of Korean biohealth according to foreign investors', Hwee Juin Lee, Vice President of Asia Pacific Region at Flagship Pioneering, emphasized, “Seoul is a cosmopolitan city as the premier global hub for clinical trials, hosting a diverse array of cutting-edge research centers.”

CHARLOTTESVILLE, VA – July 15, 2024 – Today AMPEL BioSolutions, with collaborators Lupus Therapeutics and Aker Biomarine, revealed peer-reviewed results that supplementation with Omega3-rich Krill Oil improves disease activity of autoimmune Lupus patients. 

Omega3 lipids called polyunsaturated fatty acids (PUFAs) that are found in microscopic shrimp-like crustaceans called Krill from the waters around Antarctica are key to maintaining a low inflammatory state.  Some studies indicate that Krill contains Omega3 in an easily digested phospholipid form that may achieve more effective integration into cellular membranes compared with the triglyceride-rich form predominant in fish oil.  And the concentrated Superba Boost Krill Oil (AKBM-3031) was highly ranked by the evidence-based CoLTs (Combined Lupus Treatment Scoring) published approach for testing in the ORKIDS clinical trial.

As we gear up for the 10th Annual BioHealth Capital Region Forum on September 17^th-18^th, 2024, at the US Pharmacopeia in Rockville, MD, we invite your organization to become a sponsor. This year’s forum marks a decade of innovation and collaboration within the biohealth sector and offers unique sponsorship opportunities that promise substantial visibility and engagement. You can become a sponsor with unique new sponsorship opportunities this year, including “Lunch Sponsor” and “Reception Sponsor” for both days.

Sponsoring the forum ensures that your organization’s logo will be prominently displayed on all event materials, daily presentation decks, and standees positioned strategically throughout the venue. This visual branding extends throughout the event, ensuring your message reaches all attendees. Furthermore, all sponsors are invited to an exclusive VIP Leadership Dinner, providing a prime networking opportunity with top industry leaders.

In this episode of BioTalk with Rich Bendis, we have the honor of speaking with the U.S. Ambassador to the Republic of Tunisia, Joey R. Hood. With an impressive diplomacy and international relations background, Ambassador Hood sheds light on the vital collaboration between the U.S. and Tunisia in scientific research, innovation, and technology transfer.

Ambassador Hood discusses the current status of U.S.-Tunisia cooperation in scientific research and technology transfer, emphasizing these areas’ critical role in Tunisia’s development. He highlights the importance of scientific research, innovation, and technology transfer in Tunisia and explains how the U.S. can support and enhance these efforts.

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RNA therapies developer miRecule is partnered with Sanofi on the development of a treatment for facioscapulohumeral muscular dystrophy, or FSHD. MiRecule, winner in the biopharma track of MedCity News’s Pitch Perfect startup competition, is now pursuing a Series A round of financing.

For biotech companies with platform technologies, one common goal is finding a big pharmaceutical partner interested in jumping onto that platform sooner or later. For miRecule, the collaboration came sooner. Within four years of its formation and still preclinical, the startup found itself working with Sanofi to develop an RNA therapy for a rare muscle disorder.

Most research-intensive universities in the US — including globally renowned institutions such as Stanford, Cornell and Yale — do not have their own venture fund to back spinouts.

Just 50 out of the 146 universities that are at the top rank of the Carnegie classification — a scale that measures the amount of doctoral research they produce — have an investment vehicle.

These universities, by and large, produce the most spinouts and typically have infrastructure, like incubators or maker labs, in place to support fledgling startups. Many of the tech transfer offices run entrepreneurs-in-residence programmes to bring in the right expertise to run spinouts.

Maryland-based company seeks to revolutionize green algae growth factors 
COLUMBIA, Md., (July 11, 2024) — TEDCO, Maryland’s economic engine for technology companies, announced a recent $200,000 Pre-Seed Builder Fund investment in Phycin, Inc. TEDCO’s Pre-Seed Builder Fund is housed under TEDCO’s Social Impact Funds which were created to engage and invest in economically underserved founders and communities.

“At Phycin, we are focused on revolutionizing growth factor production, allowing researchers to continue their efforts with an efficient and cost-effective alternative,” said Jun Wang, CEO of Phycin, Inc. “Thanks to TEDCO’s investment, we can continue our research and development and hopefully offer more affordable and efficient solutions for various industries.”

As the first of its kind in South Africa, through a partnership between Eduvos and Task and supported by Wesgro, the inaugural Bio Centrifuge Africa competition will be selecting the winners on 17 July at the exclusive live pitching event in Cape Town.

The competition was launched this year to highlight South African talent in the expanding biotech sector and serve as a bridge between the country and the USA's BioHealth Capital Region (BHCR). This groundbreaking event promises to propel visionary health-focused innovators and biomedicine students onto the global stage.

South Africa, through the Western Cape’s biotech hub, has forged a partnership with the BioHealth Capital Region in the US. As part of this collaboration, the winner of Bio Centrifuge Africa will have the opportunity to compete at the prestigious Crab Trap competition, scheduled for September 18, 2024, in Rockville, Maryland.

COLUMBIA, Md., (July 11, 2024) — TEDCO, Maryland’s economic engine for technology companies, announces the appointment of Graham Dodge as the vice president of the Innovation Connectors department. Dodge will be responsible for providing leadership and oversight to TEDCO’s Cultivate Maryland and Innovation Connectors.

The Innovation Connectors department, previously known as TEDCO’s Entrepreneur & Ecosystem Empowerment (E3) department, is responsible for driving strategic collaborations, supporting entrepreneurial innovation and defining strategies to leverage networks and resources for innovative success. The department houses influential programs intentionally created to support entrepreneurial growth, such as the Rural Business Innovation Initiative, Urban Business Innovation Initiative, Network Advisors, Prelude Pitch and more.

Are you ready to showcase your company to some of the top minds in the industry? The 9^th Annual Crab Trap competition, a highlight of the annual BioHealth Capital Region Week, is now accepting applications from emerging companies eager to demonstrate their groundbreaking solutions. Scheduled for September 18th at the US Pharmacopeia in Rockville, MD, this competition is an unmissable opportunity for startups looking to make a significant impact.

A Platform for Pioneers

The Crab Trap provides a unique platform for startups to present their innovations before a panel of esteemed judges and a live audience. This year’s competition promises exciting prizes, including cash and business services, to accelerate the winning entry’s growth and success.

Why Enter Crab Trap?

Participating in the Crab Trap competition offers numerous benefits:

• Visibility: Gain exposure to a broad audience of industry professionals, investors, and potential partners.
• Feedback: Receive valuable insights and feedback from leaders in the biohealth industry.
• Prizes: Compete for a chance to win cash and services that can catalyze your company’s growth and development.

Apply Now

RICHMOND, Va., July 8, 2024 – Activation Capital, an innovation ecosystem development organization, announced today that Chandra Briggman, president and chief executive officer of Activation Capital, will depart the organization to pursue new opportunities to build innovation ecosystems and drive economic development.  

“Chandra Briggman’s pivotal time at Activation Capital has been defined by impact, and her impressive body of work has energized our region,” said Michael Rao, chairman of the Virginia Biotechnology Research Partnership Authority Board and Virginia Commonwealth University (VCU) president. “In 2020, Chandra was recruited from Boston/Cambridge, MA, to grow the economic impact of Activation Capital. In four years, two of which were at the height of the COVID-19 pandemic, she helped reenergize the organization with a new vision and strategy. Chandra executed a bold roadmap that has since grown our regional innovation economy and strengthened Activation Capital’s sustainability model for the future.” 

Artificial intelligence is keeping the venture capital ecosystem afloat. It accounted for more than 40% of new private U.S. "unicorns" in the first half this year, and over 60% of the increase in total venture-backed valuation, per PitchBook.

The big picture: There were 13 new U.S. AI unicorns — private companies that reached valuations of over $1 billion — between Jan. 1 and June 25 of this year.

• Elon Musk's xAI on its own added $24 billion in valuation, while all AI unicorn companies combined added $116 billion in aggregate value.
• Overall U.S. unicorn value has grown by $162 billion so far this year.

Follow the money: Over the past few years, AI accounted for about 15%-17% of venture dollars invested in North America and Europe. That number increased to about 20% in 2023 and is rising even more in 2024, according to PitchBook senior emerging technology analyst Brendan Burke.

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HONG KONG and GERMANTOWN, Md. and SUZHOU, China, July 8, 2024 /PRNewswire/ -- Sirnaomics Ltd. (the "Company", together with its subsidiaries, the "Group" or "Sirnaomics"; stock code: 2257), a leading biopharmaceutical company engaging in discovery and development of advanced RNAi therapeutics, today announced the interim results for successful completion of the second cohort of its Phase I clinical study of GalNAc-based RNAi therapeutic STP122G, targeting Factor XI as a novel form of anticoagulation agent.

ARLINGTON, Va.--(BUSINESS WIRE)--Kerecis, the company pioneering the use of sustainably sourced fish skin and fatty acids in cellular therapy and tissue regeneration and protection, today announced the availability of Shield Spiral, an extension of the Kerecis Shield silicone fish-skin combination product range.

ROCKVILLE, Md. and SUZHOU, China, July 3, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing both first-in-class and best-in-class therapies for hematological malignancies, announced today that on July 2, 2024, in relation to the Exclusive Option Agreement with Takeda for the third-generation BCR-ABL inhibitor olverembatinib (HQP1351), Ascentage Pharma has received the US$100 million option payment called for by the Exclusive Option Agreement.

GAITHERSBURG, Md., July 02, 2024 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) today announced it has received more than $250 million in contract modifications from the Administration for Strategic Preparedness and Response (ASPR) at the United States Department of Health and Human Services (HHS), to deliver millions of doses of four medical countermeasures (MCMs). These contract modifications will help ensure continued supply/stockpiling of critical MCMs to address biological threats and emergencies against anthrax, smallpox and botulism.

WASHINGTON, DC — The Biden-Harris Administration, through the U.S. Department of Commerce’s Economic Development Administration (EDA), announced another funding round of approximately $504 million in implementation grants to 12 Tech Hubs to scale up the production of critical technologies, create jobs in innovative industries, strengthen U.S. economic competitiveness and national security, and accelerate the growth of industries of the future in regions across the United States.

The Tech Hubs Program is a flagship initiative of President Biden’s Administration to invest in and grow the economies in communities across the country, advancing America’s global leadership in critical technologies, and strengthening our national and economic security.

GAITHERSBURG, Md., July 02, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today announced that it has entered into a securities purchase agreement for a private investment in public equity (“PIPE”) financing that is expected to result in gross proceeds of approximately $130.0 million to the Company, before deducting placement agent fees and other offering expenses. The PIPE financing is expected to close on or about July 3, 2024, subject to satisfaction of customary closing conditions.

The PIPE financing included participation from both new and existing investors, including HBM Healthcare Investments (Cayman) Ltd., Invus, Schooner Capital, Surveyor Capital (a Citadel company), Timothy A. Springer, Ph.D., a leading mutual fund manager, and other institutional investors.

GAITHERSBURG, Md., July 02, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (“Cartesian” or the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today announced positive topline results from its Phase 2b trial of Descartes-08 in patients with generalized myasthenia gravis (MG).

Descartes-08, Cartesian’s lead product candidate, is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) directed against the B cell maturation antigen (BCMA). It is designed to be administered as an outpatient treatment without the need for lymphodepleting chemotherapy required to achieve activity with conventional CAR-T cell therapies. Descartes-08 was previously granted Regenerative Medicine Advanced Therapy (RMAT) Designation and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of MG.

BEL AIR, Md., (July 1, 2024) – Maryland Department of Commerce has awarded $500K to Baltimore-based Early Charm to develop a biomanufacturing incubator at a Harford County-owned facility near Aberdeen Proving Ground (APG). The funding from the state’s Build Our Future grant will be used to upgrade the CONVERGE Innovation Center, formerly known as the HEAT Center, located at the I-95/Aberdeen interchange.

Biomanufacturing is the use of biological processes to build new materials such as body armor made from spider silk, three times the strength of Kevlar. This technology has the potential to revolutionize manufacturing.

Professor Ramzi Maalej, CTO and Founder of FluoInk, recently traveled from Tunisia to Maryland to attend the SelectUSA Investment Summit. FluoInk is currently working on its innovative Bacti Cleaner technology, which adds antibacterial properties to paints. It is making strides in the eco-friendly and non-toxic product space with the development of this product.

BioHealth Innovation Inc. (BHI) is collaborating with FluoInk, along with partners from The Tunisian Health Center for University Research Excellence and Sustainability (CURES) and FHI 360, to help grow their business. While in Maryland, Professor Maalej connected with key figures in the BioHealth Capital Region, including Brian Castleberry, Senior Regional Manager - Middle East, Africa, and UK at the Maryland Department of Commerce, and Brian Darmody, AURP Chief Strategy Officer.

"Visiting Maryland and participating in the SelectUSA Investment Summit was an incredible experience. The networking opportunities and the collaborative environment, facilitated by BHI and FHI 360, were beyond my expectations," said Professor Ramzi Maalej, CTO and Founder of FluoInk. "This summit has opened new doors for transforming our academic research into impactful commercial products."

Join us for an episode of BioTalk with Rich Bendis featuring Dr. Bradley Maron, Senior Associate Dean for Precision Medicine and Executive Co-Director of the University of Maryland-Institute for Health Computing at the University of Maryland School of Medicine. With his extensive background in cardiovascular research and precision medicine, Dr. Maron offers a wealth of knowledge and innovative perspectives.

In this episode, Dr. Maron shares his professional journey, discussing his various roles at the University of Maryland and what drew him to the region. He provides a comparative analysis of the ecosystems in Boston/Harvard and Maryland/University of Maryland, highlighting the unique attributes and opportunities within each.

Dr. Maron introduces the Institute for Health Computing (IHC), explaining its creation, mission, and strategic partnerships with the University System of Maryland and Montgomery County. He outlines the significance of data science in improving health and wellness, addressing its increasing role and challenges.

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EyeControl improves clinical outcomes, prevent cognitive decline, and enhance the patient and family experience.“We are honoured to be able to support every patient – but especially our soldiers."

When Yoav Tzivoni, a combat soldier seriously wounded in the northern Gaza Strip, awoke after six months in the intensive care unit of the Samson Assuta Ashdod University Hospital, he shockingly asked the nurse for his favorite heavy metal music.

“I was shaking from excitement when the nurse texted and said Yoav is asking for his music!” recounts Michal Finkelstein, product specialist, and speech and language pathologist by training, in charge of customer relations and success at the Israeli company EyeControl.

“What? Is he awake?” Finkelstein wondered, saying that she was “shaking from excitement.”

Tzivoni and his fellow wounded ICU soldiers are benefiting from a recently discovered additional and potentially internationally game-changing use of EyeControl, a wearable device that enables patients – including those on ventilators – to communicate with medical staff and family members via eye gestures and a bone conduction headset, to hear the recorded messages of loved ones – and to listen to one’s favorite music.

EyeControl helps improve clinical outcomes, prevent cognitive decline, and enhance the patient and family experience. It is currently being tested in prestigious university hospitals in and out of Israel to see if it reduces delirium in ICU patients.

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BETHESDA, Md., June 27, 2024 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (Nasdaq: GANX), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announces that Matthias Alder has departed the Company in order to pursue other opportunities. Upon Mr. Alder’s departure, the Company appointed Gene Mack, the Company’s current Chief Financial Officer, as interim Chief Executive Officer, effective June 25, 2024. Mr. Mack will retain his role as Chief Financial Officer. Khalid Islam, Ph.D., founder and Chairman of the Company’s Board of Directors, will bolster management as Executive Chairman until a permanent CEO is appointed.

Chicago, May 2024 — miRecule, a cutting-edge startup focused on developing antibody RNA conjugate therapeutics, clinched the top spot at the prestigious MedCity INVEST Biopharma track competition held at the Ritz Carlton in Chicago. The event, presented by MedCity News and the Mid-America Healthcare Investors Network (MHIN), brought together active investors and promising entrepreneurs from the healthcare industry.

miRecule’s innovative approach to personalized RNA therapeutics captured the attention of venture capital judges, who recognized the company’s potential to revolutionize healthcare.

Agency seeks proposals for innovative research and development from small businesses 

The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services (HHS), today announced new funding opportunities through the Small Business Innovation Research (SBIR)/ Small Business Technology Transfer (STTR) program. 

The ARPA-H SBIR/STTR solicitation seeks proposals from small businesses that aim to rapidly achieve better health outcomes across patient populations, communities, disease, and health conditions.

18 projects will receive funding to support their growth and development

COLUMBIA, Md., (June 27, 2024) — TEDCO, Maryland’s economic engine for technology companies, announced the 2024 Maryland Makerspace Initiative Program (“Makerspace Program”) awardees. Funding will be awarded to 18 projects spanning across nine (9) counties and Baltimore City.

“TEDCO is committed to supporting the continued growth of innovation in Maryland. The Makerspace Program is just one tool that TEDCO has to promote this expansion,” said Terry Rauh, chief finance and operations officer at TEDCO. “And after witnessing the success and growth of last year’s awardees, we are hopeful that these awardees will continue the trend.”

WASHINGTON, D.C. — Amazon Web Services committed $10 million to fund pediatric rare disease research, including cancer, on Wednesday at its annual summit in the nation's capital.

The tranche of cash is first being allocated through three, $1 million grants to Children’s National Hospital in Washington, D.C.; Nationwide Children’s Hospital in Columbus, Ohio; and the Children’s Brain Tumor Network, located at the Children’s Hospital Of Philadelphia. 

AWS says the unrestricted cash grant will allow the children’s cancer centers, which are already using AWS software, to continue innovation in accelerating pediatric research, improving well-being for children and empowering the professional pediatric workforce and caregivers of children.