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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

Published

1 year ago

September 29, 2023

Daily News Staff

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.

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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Space and Tech

NASA Coverage of the Upcoming Progress 90 Cargo Resupply Mission

Published

12 hours ago

November 21, 2024

Charles Elliot

Excitement is building as NASA prepares for the upcoming launch of the Progress 90 spacecraft, a critical mission that will deliver essential supplies to the crew aboard the International Space Station (ISS). With a launch scheduled for 7:22 a.m. EST on Thursday, November 21, from the Baikonur Cosmodrome in Kazakhstan, this unpiloted Roscosmos cargo spacecraft will transport nearly three tons of food, fuel, and vital equipment to the Expedition 72 crew.

Tune In for Live Coverage

NASA is offering live launch coverage starting at 7:00 a.m. EST on the day of the launch, available on NASA+ and their official website. This is a fantastic opportunity for space enthusiasts and curious minds alike to witness firsthand the relentless efforts of international space collaboration. Not sure how to catch the excitement? NASA has made it easy! Their live coverage can be accessed through various platforms, including social media, so you won’t miss a moment of this thrilling event.

Mission Timeline

Following the launch, the Progress 90 spacecraft will embark on a two-day journey to the ISS, a remarkable feat of engineering and logistics. It is scheduled to autonomously dock at the space-facing port of the Poisk module on Saturday, November 23, at 9:35 a.m. EST. Make sure to tune in for NASA’s coverage of the docking process, which will begin at 8:45 a.m. that same day. Observing the intricate choreography of rendezvous and docking showcases the ingenuity of modern space missions.

Before Progress 90 arrives, it’s important to note that the previous Progress 88 spacecraft will undock from the Poisk module on Tuesday, November 19. Although NASA will not stream this undocking event, it marks another step in efficient cargo operations at the ISS.

What Happens Next?

Once docked, the Progress 90 spacecraft will remain attached to the ISS for approximately six months, providing continuous support to the crew. When it completes its mission, the spacecraft will carry away any used supplies and cargo, ensuring a tidy and efficient living environment aboard the station. It will then safely re-enter Earth’s atmosphere, disposing of invaluable trash generated during the mission.

The Role of the ISS in Space Exploration

For over 24 years, NASA has played a key role in maintaining a continuous human presence in low Earth orbit, enabling groundbreaking research and scientific exploration that simply cannot be achieved on our home planet. The ISS serves as a unique laboratory that allows for experiments in microgravity, helping scientists make discoveries that will not only benefit life on Earth but also pave the way for future space exploration.

The station is poised to support NASA’s ambitious goals for the Artemis program, which aims to return humans to the Moon and expand our presence in the solar system. Ultimately, this sustained effort will guide us toward the dream of human exploration of Mars.

Join the Journey!

As we look forward to another exciting chapter in space travel with the Progress 90 mission, we encourage everyone to join the conversation. Share your thoughts, watch the launch, engage with fellow enthusiasts online, and marvel at the boundaries that humanity is pushing through science and collaboration. Space is within our reach, and every mission takes us one step closer to understanding our universe!

Together, let’s celebrate the spirit of exploration and the collaborative advances in human innovation that make missions like this possible. See you on launch day! 🌌🚀✨

Get breaking news, images and features from the space station on Instagram, Facebook, and X.

Learn more about the International Space Station, its research, and its crew, at:

https://www.nasa.gov/station

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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Space and Tech

Blue Origin’s New Shepard NS-28: Aiming for the Stars on November 22nd

Blue Origin’s New Shepard NS-28 mission, launching on November 22, features a significant crewed aspect, promoting broader space access. The mission patch symbolizes the journey’s educational and inspirational goals. Join the excitement!

Published

3 days ago

November 19, 2024

Rod Washington

New Shepard 28

Blue Origin has once again captured the public’s imagination with the announcement of its ninth human flight, the NS-28 mission, set to launch from Launch Site One in West Texas on Friday, November 22. With the launch window opening at 9:30 AM CST (15:30 UTC), excitement is brewing as space enthusiasts and potential future astronauts gear up for another exhilarating journey into the cosmos.

One of the highlights of this mission is the crewed aspect, making it another significant step in Blue Origin’s mission to democratize access to space. Following the success of its previous flights, NS-28 promises to deliver an experience that will inspire and ignite passion for space exploration. The live webcast on BlueOrigin.com will commence at T-30 minutes, offering spectators a front-row seat to this groundbreaking event.

New Shepard 28 — Image Credit: Blue Origin

The NS-28 Mission Patch: A Story of Meaning and Symbolism

Accompanying the NS-28 mission is a beautifully designed mission patch that encapsulates the spirit and purpose of this historic flight. Each element of the patch carries deep significance, painting a portrait of the crew and their aspirations in space.

Emily Calandrelli is represented by the color pink and a girl in signature pink overalls, acknowledging her influential role as a science communicator and advocate for space education.
For Sharon and Marc Hagle, this flight marks their second journey on New Shepard, a milestone aptly symbolized by the centerline in the “2,” fostering a sense of continuity and commitment to space travel.
The stars in the mission patch signify Austin Litteral’s enduring vision of humanity’s place among the stars, highlighting the adventurous spirit that drives our quest for knowledge beyond our planet.
At the bottom of the patch, a representation of people embodies J.D. Russell’s hope for future generations to expand their understanding of the universe, underscoring the mission’s educational and inspirational goals.
Lastly, the wolf in the crew capsule’s window stands as a tribute to Hank Wolfond, symbolizing the strength, courage, and tenacity that is required for exploration beyond our earthly confines.

Looking Ahead: The Future of Space Tourism and Exploration

As Blue Origin prepares for the NS-28 mission, more than just a flight is at stake. This mission represents a significant leap in commercial space travel and signifies a broader movement towards making space accessible and enjoyable for everyone. Each launch brings us closer to a future where experiencing space is a possibility for many, rather than just a select few.

With the countdown ticking down to November 22, stakeholders, space enthusiasts, and the general public alike are encouraged to tune in to the live webcast and partake in the excitement surrounding human spaceflight. Innovations in technology and a shared passion for exploration are paving the way for a new era of discovery—one where the stars are no longer out of reach.

Join us in celebrating the NS-28 mission—an emblem of human ingenuity, curiosity, and the indomitable spirit of exploration. See you on launch day—let’s reach for the stars together!

For more information about the crew, please see our previous blog post here

Or read the post from Blue Origin here.

Follow Blue Origin on X, Instagram, Facebook, LinkedIn, Threads, and YouTube, and sign up at BlueOrigin.com to stay updated on all mission details.

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unknown

UFO Debris Discovered on Lake Huron’s Shoreline: A Look Back at the February 2023 Incident

Published

4 days ago

November 17, 2024

Rod Washington

In a remarkable turn of events, recently released documents have confirmed the recovery of debris from a UFO that was shot down by a U.S. F-16 fighter jet over Lake Huron on February 12, 2023. This incident marked the third notable UFO occurrence in North America within that month, following a high-profile engagement involving a Chinese surveillance balloon just days earlier.

The octagon-shaped object, which had strings dangling from it, raised eyebrows when U.S. military forces targeted it. The F-16 pilot fired at the unidentified flying object twice, successfully striking it with one missile before it plummeted into Canadian waters. The incident sparked immediate attention from both American and Canadian authorities.

According to a Freedom of Information request filed by CTVNews.ca, the wreckage was located on the shoreline of Lake Huron, yet this significant discovery went largely unreported to the public at the time. The Royal Canadian Mounted Police (RCMP), alongside the American and Canadian Coast Guard, initiated a brief search for the debris. However, they concluded their efforts prematurely, citing deteriorating weather conditions and a low likelihood of recovery as the main factors.

🚨 UFO debris found on Lake Huron! Shot down by a U.S. fighter jet in Feb 2023, this octagon-shaped object sparks intrigue. What’s the truth? 👽✨ #UFO #UAP

In March 2023, the RCMP managed to recover the UFO wreckage from its crash site. In communications obtained through the documents, a member of the RCMP informed a Canadian military general that the module was traced back to a company specializing in weather monitoring equipment, shedding light on the mysterious object.

The series of UFO incidents in February 2023 has piqued the interest of lawmakers and the public alike, especially in light of the U.S. military’s actions against the Chinese surveillance balloon. The House Committee on Oversight and Accountability has held multiple hearings on unidentified anomalous phenomena (UAPs), with the latest session occurring on November 13, 2023. Experts like Michael Gold, a former NASA leader and member of the independent UAP study team, emphasized the importance of courage in seeking the truth about such phenomena.

As the conversation surrounding UAPs continues to evolve, the recovery of debris from Lake Huron serves as a reminder of the ongoing mystery and intrigue that surrounds unidentified objects in our skies. The implications of these incidents extend beyond mere curiosity, potentially impacting national security and public perception of aerial phenomena. Stay tuned for further developments as authorities continue to unravel the truth behind these intriguing occurrences.