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FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

FDA launches pilot program to accelerate rare disease therapies, improving communication between sponsors and FDA staff. #RareDiseaseTherapies #FDA #ClinicalTrials

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In a significant move to expedite the development of novel drug and biological products for rare diseases, the U.S. Food and Drug Administration (FDA) has introduced a pilot program aimed at fostering more frequent communication between sponsors and FDA staff. This initiative, known as the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program, aims to address clinical development challenges and generate high-quality data to support the future marketing applications of potentially life-saving therapies.

Enhancing Communication and Collaboration:
Under the START Pilot Program, a limited number of sponsors will have the opportunity to obtain regular ad-hoc communication and advice from FDA staff. This communication will focus on addressing product-specific development issues such as clinical study design, selection of control groups, and fine-tuning the choice of patient population. By facilitating improved communication, the FDA aims to streamline the development process for rare disease therapies and ensure that patients with unmet medical needs receive timely access to innovative treatments.

Eligibility and Application Process:
The program is open to sponsors of products currently in clinical trials under an active Investigational New Drug application (IND) regulated by the Center for Biologics Evaluation and Research (CBER) and/or the Center for Drug Evaluation and Research (CDER). Eligibility criteria differ between CBER and CDER-regulated products. CBER-regulated products must be gene or cellular therapies addressing an unmet medical need for a rare disease likely to cause significant disability or death within the first decade of life. CDER-regulated products must be intended to treat rare neurodegenerative conditions, including rare genetic metabolic disorders. Detailed eligibility requirements can be found in the Federal Register Notice.

The Road Ahead:
The FDA plans to accept applications for the START program between January 2, 2024, and March 1, 2024. Participants will be selected based on their application readiness and ability to progress their development programs towards a marketing application. The agency will choose up to three participants for each center. After evaluating the pilot program and receiving feedback from selected sponsors, the FDA may consider a second iteration, which would be announced in the Federal Register at a later date.

Commitment to Rare Disease Therapies:
The FDA’s efforts to advance the development of therapies for rare diseases extend beyond the START Pilot Program. The agency has recently requested feedback from stakeholders on scientific challenges and opportunities related to cellular and gene therapies for rare diseases. This input will help inform the planning of future meetings, workshops, and educational programs to facilitate the development of regulatory tools and frameworks.

Additionally, the FDA has created the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) program under the CDER Accelerating Rare disease Cures (ARC) initiative. Stakeholder feedback gathered through this program will contribute to identifying knowledge gaps in rare disease drug development and the creation of publicly available resources to guide stakeholders in designing and conducting clinical trials.


The FDA’s launch of the START Pilot Program demonstrates its commitment to expediting the development of rare disease therapies. By facilitating enhanced communication and collaboration between sponsors and FDA staff, the program aims to accelerate the availability of potentially life-saving treatments for patients with unmet medical needs. This pilot program, along with other ongoing initiatives, highlights the FDA’s dedication to improving the regulatory framework and recommendations for rare disease product sponsors, ultimately ensuring the safety, effectiveness, and quality of these important therapies.

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Read the press release: https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-further-accelerate-development-rare-disease-therapies?utm_medium=email&utm_source=govdelivery

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The Cosmic Dance: Understanding the Newly Discovered Asteroid 2024 YR4 and Its Risk to Earth

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Asteroid

Astronomy enthusiasts and casual stargazers alike are buzzing with excitement—and a hint of concern—over the recent discovery of a new asteroid, named 2024 YR4. This celestial wanderer has recently caught the attention of both the European Space Agency (ESA) and NASA, particularly due to its increasing risk assessment concerning a potential impact with Earth.

What’s the Buzz About 2024 YR4?

Discovered on December 27, 2024, by the Asteroid Terrestrial-impact Last Alert System (ATLAS) telescope in Chile, asteroid 2024 YR4 is now on the radar for its increased chance of impacting Earth. Initially assessed at a 1.2% risk, fresh observations have revised this figure to 2.2% for a possible encounter on December 22, 2032. While the mathematics behind such percentages can be daunting, they essentially mean that we have a somewhat higher—but still relatively unlikely—chance of an impact.

The Evolution of Risk Assessment

What’s fascinating about asteroids like 2024 YR4 is how their risk percentages can change significantly over time. According to ESA, as more observations and data are gathered, these numbers typically fluctuate. This rollercoaster of statistics can be observed in the case of the infamous asteroid Apophis, which went from a high-risk designation to much lower probabilities thanks to more refined measurements of its orbit.

Experts from the CNEOS (Center for Near Earth Object Studies) note that tracking 2024 YR4 for more data is crucial. The more astronomers can observe it and refine its trajectory, the better equipped they will be to predict its path and assess any potential risk.

What If It Hits?

While the current risk of impact is low, concerns remain about what could happen if an asteroid of this size were to strike. Measuring between 131 to 295 feet (40 to 90 meters) wide, 2024 YR4 is comparable to a large building. Dr. Paul Chodas from NASA highlights that, should it be on the larger end of its scale, the damage could be significant, potentially affecting an area up to 50 kilometers (31 miles) from the impact site. Comparatively, past asteroid impacts, such as the one in Tunguska in 1908, resulted in immense devastation over vast areas.

Asteroids of similar dimensions have been known to strike Earth every few millennia—bringing with them the potential for severe regional damage.

Keeping Our Eyes on the Sky

The ongoing monitoring of 2024 YR4 is a collective effort supported by international organizations, including the International Asteroid Warning Network (IAWN) and the Space Mission Planning Advisory Group (SMPAG). Together, these groups work to analyze the asteroid’s trajectory, assess potential threats, and formulate response plans if necessary. Already, discussions around mitigation strategies are underway. These could range from deflecting the asteroid through means like NASA’s Double Asteroid Redirection Test to even evacuating potentially impacted regions.

For now, astronomers are racing against time to observe 2024 YR4 as it travels farther away from Earth and becomes more challenging to track. Our best window to study it financially is until early April, after which it will disappear from our view—returning only in 2028.

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The Bigger Picture

While asteroid 2024 YR4 is getting a lot of attention, it serves as a reminder of the cosmos we inhabit, full of wonders and occasional threats. The collaborative efforts of international space agencies demonstrate how seriously we take the potential risks associated with near-Earth objects.

As the astronomical community keeps watch over 2024 YR4, we are also reminded of the technological advances that allow us to detect and track these celestial bodies. Thanks to the incredible human ingenuity dedicated to space exploration, we can keep an eye on our cosmic neighbors and stay informed about any future surprises from the universe.

So, while the chance of disaster is currently low, one thing is clear: the dance of celestial bodies continues, and it’s an exhilarating spectacle to behold! Keep looking up; you never know what you might discover next. 🔭✨

Check out the story on CNN: https://www.cnn.com/2025/02/07/science/asteroid-2024-yr4-earth-risk/index.html

The science section of our news blog STM Daily News provides readers with captivating and up-to-date information on the latest scientific discoveries, breakthroughs, and innovations across various fields. We offer engaging and accessible content, ensuring that readers with different levels of scientific knowledge can stay informed. Whether it’s exploring advancements in medicine, astronomy, technology, or environmental sciences, our science section strives to shed light on the intriguing world of scientific exploration and its profound impact on our daily lives. From thought-provoking articles to informative interviews with experts in the field, STM Daily News Science offers a harmonious blend of factual reporting, analysis, and exploration, making it a go-to source for science enthusiasts and curious minds alike. https://stmdailynews.com/category/science/

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NASA Brings Space to New Jersey Classroom with Astronaut Q&A

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In an exciting opportunity for young minds, NASA is bringing the wonders of space exploration directly to a New Jersey classroom. Students from the Thomas Edison EnergySmart Charter School in Somerset, New Jersey, will have the unique chance to connect with NASA astronaut Nick Hague aboard the International Space Station (ISS). During a 20-minute space-to-Earth call, Hague will answer prerecorded questions from students, focusing on science, technology, engineering, and mathematics (STEM) topics.

The event, scheduled for 11:10 a.m. EST on Tuesday, February 11, will be broadcast live on NASA+, NASA’s streaming platform. This interactive session promises to inspire the next generation of explorers and highlight the importance of STEM education in shaping the future of space exploration.

How to Watch

The live Q&A session will be available to the public, offering a rare glimpse into life aboard the ISS and the work being done to advance human knowledge and capabilities in space. Viewers can tune in via NASA+ or follow NASA’s social media channels for updates and streaming options. For those unable to watch live, the event will likely be archived for later viewing.

Media Coverage

Media representatives interested in covering this event must RSVP by 5 p.m. EST on Thursday, February 6, to Jeanette Allison at [email protected] or 732-412-7643. This is a fantastic opportunity to showcase how NASA is engaging with students and fostering interest in STEM fields.

The International Space Station: A Hub of Innovation

For over 24 years, astronauts have continuously lived and worked aboard the ISS, conducting groundbreaking research and testing technologies that benefit life on Earth and pave the way for future exploration. The station serves as a microgravity laboratory where astronauts perform experiments in fields such as biology, physics, and materials science, while also developing the skills needed for missions to the Moon, Mars, and beyond.

Communication between the ISS and Earth is made possible through NASA’s Space Communications and Navigation (SCaN) program, specifically the Near Space Network, which ensures 24/7 connectivity with Mission Control in Houston. This seamless communication allows astronauts like Nick Hague to share their experiences and insights with audiences worldwide, including students eager to learn about space.

Inspiring the Artemis Generation

This event is part of NASA’s broader efforts to inspire the Artemis Generation—the next wave of explorers who will carry humanity’s mission of discovery forward. Through the Artemis program, NASA aims to return astronauts to the Moon and prepare for future human exploration of Mars. By engaging with students and educators, the agency hopes to ignite curiosity and passion for STEM, ensuring the United States remains a leader in space exploration and innovation.

A Lifelong Impact

For the students at Thomas Edison EnergySmart Charter School, this Q&A session is more than just a chance to ask questions—it’s an opportunity to dream big and see themselves as part of humanity’s journey into the cosmos. By connecting with an astronaut in real-time, they’ll gain a deeper understanding of the challenges and rewards of space exploration, as well as the critical role STEM plays in solving the problems of tomorrow.

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Don’t miss this inspiring event! Tune in on February 11 to witness the magic of space come alive in a New Jersey classroom.

For more information about NASA’s missions, educational initiatives, and streaming options, visit NASA’s official website.


What are your thoughts on NASA’s efforts to engage students in STEM? Share your comments below!

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Celebrate Transit Equity Day: Ride Metrolink Free on February 4, 2025!

On February 4, 2025, Metrolink will offer free rides in celebration of Transit Equity Day, honoring Rosa Parks’ legacy and emphasizing public transportation’s role in community access and equity, while encouraging regional exploration.

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Transit Equity Day
Image: Metrolink

Transit Equity Day

Los Angeles – Mark your calendars! On February 4, 2025, Southern Californians will have the unique opportunity to ride Metrolink free of charge, as the region’s six-county passenger rail provider celebrates Transit Equity Day. This annual event honors the legacy of civil rights pioneer Rosa Parks and underscores the essential role public transportation plays in fostering access and equity for all communities.

On this special day, riders can board at any of Metrolink’s 67 stations without needing a ticket and can travel systemwide, including the Arrow service. To further celebrate Transit Equity Day, numerous local transit carriers will also waive fares, providing additional options for those seeking to explore the region.

Transit Equity Day

A Statement of Purpose

Metrolink Board Chair and Orange Orange County Fourth District Supervisor Doug Chaffee expressed the significance of Transit Equity Day, stating, “This day honors Rosa Parks’ enduring legacy, while highlighting the vital role public transportation plays in ensuring people from all communities have access.” He continued, “When Metrolink introduced the largest service expansion in our history last fall, it was to create a more equitable system that works for all Southern Californians. Offering free rides on Transit Equity Day helps fulfill that promise.”

Exciting Changes in Service

In October 2023, Metrolink increased weekday service by nearly 23%, adding more midday and late-evening options for those needing to travel outside traditional work hours. Thanks to this expansion and recent improvements, Metrolink has seen a surge in ridership, setting multiple post-pandemic, single-day records. Moreover, a new San Bernardino Line schedule implemented on January 27 aims to enhance on-time performance along this high-volume corridor, allowing riders to experience efficient travel firsthand on Transit Equity Day.

A Commitment to Accessibility

Metrolink is not just about free rides on special occasions. The agency is dedicated to making public transit accessible to everyone year-round. In addition to offering three free-ride days each year, Metrolink provides everyday discounts for children, seniors, riders with disabilities, low-income riders, and active military members. For students, the Student Adventure Pass program allows K-12, college, and trade school students to ride free with a valid student ID, an initiative extended through June 30, 2025.

Metrolink ticketholders also enjoy free transfers to many connecting local transit providers, further enhancing accessibility across the region.

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Explore the Region

If you’re looking to discover new places, Metrolink has curated a special repository of destinations that are easily accessible from its stations. Check out these exciting travel options at metrolinktrains.com/explore and start planning your adventure on Transit Equity Day!

Join Metrolink, LA Metro, Omnitrans, the Orange County Transportation Authority, the Riverside County Transportation Commission, the San Bernardino County Transportation Authority, the Ventura County Transportation Commission, and other agencies in taking part in this celebratory day of equitable access to public transportation.

Ride Free, Celebrate Equity!

Transit Equity Day is more than just a free ride; it’s a reminder of the importance of public transport in promoting equity, access, and community connection. We invite all Southern Californians to take advantage of this opportunity, explore the region, and honor the legacy of Rosa Parks. This February 4th, hop aboard Metrolink and experience the freedom of transit—at no cost!

Metrolink website: Travelers looking for inspiration can view Metrolink’s specially curated repository of destinations easily accessible from Metrolink stations at metrolinktrains.com/explore.

https://stmdailynews.com/groundbreaking-for-a-sustainable-future-la-metros-southeast-gateway-line-light-rail-project/

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