Elrig

MARCH 25, 2025

It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.

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RNA Bioinformatics Science Pharmacokinetics 59

Covalent Modifiers

MARCH 1, 2025

Zhang, and Lun Kelvin Tsou Journal of Medicinal Chemistry 2025 DOI: 10.1021/acs.jmedchem.4c02665 4c02665 Pharmacological inhibition of the cGAS-STING-controlled innate immune pathway is an emerging therapeutic strategy for a myriad of inflammatory diseases. Here, we report GHN105 as an orally bioavailable covalent STING inhibitor.

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Broad Institute

APRIL 5, 2025

David Liu receives Breakthrough Prize in Life Sciences By Corie Lok April 5, 2025 Breadcrumb Home David Liu receives Breakthrough Prize in Life Sciences Liu is honored for the development of base editing and prime editing, two gene editing technologies transforming medicine.

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Science Clinical Trials DNA Disease 139

Drugs.com

JANUARY 16, 2025

16, 2025 -- The U.S. Food and Drug Administration has approved Omvoh (mirikizumab-mrkz) for the treatment of moderately to severely active Crohn disease in adults, according to a press release from Eli Lilly and Company.Omvoh targets. THURSDAY, Jan.

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Broad Institute

MARCH 11, 2025

By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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FDA Law Blog: Drug Discovery

JANUARY 30, 2025

To this end, the December 2024 draft guidance and a companion guidance published January 7, 2025, Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway (the January 2025 draft guidance ) describe FDAs latest thinking on what it means to conduct a confirmatory trial with due diligence.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Why are you focusing on liver disease? As a result, waiting lists continue to grow.

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Therapies Disease RNA Engineering 102

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Clinical Trials Disease Trials Therapies 52

National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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Treatment Trials Clinical Trials Drugs 69

H1 Blog

DECEMBER 11, 2024

In this edition, our CEO and co-founder, Ariel Katz, along with Regional VP of Trial Landscape, Ryan Brown, share their top predictions for 2025. While this approach aims to improve ROI before potential price controls kick in, it complicates trial design and recruitment, increasing the time it takes to get treatments to market.

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Broad Institute

FEBRUARY 25, 2025

Rare genetic diseases are collectively common: they affect 1 in 10 people in North America. While more than 8,000 genes are known to drive these diseases, fewer than 500 have an available treatment. For more information visit: Rare Disease Day

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Broad Institute

OCTOBER 25, 2024

Autoimmune Disease ML Challenge - Lecture 1, Part A - Biology - What is Autoimmune Disease? By Rose Circeo October 25, 2024 Breadcrumb Home Autoimmune Disease ML Challenge - Lecture 1, Part A - Biology - What is Autoimmune Disease? The challenge will run from October 28, 2024 - January 31, 2025. MLC-2024.

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Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Broad Institute

OCTOBER 17, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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Fierce BioTech

FEBRUARY 24, 2025

Transforming Early Detection in Oncology and Rare Diseases Using AI pesurya Mon, 02/24/2025 - 09:53 Wed, 04/09/2025 - 11:00 Resource Type Webinar Chris Tackaberry Dr. Will Ricketts Health systems around the globe face a difficult paradox: delivering more care with fewer resources.

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Quanticate

SEPTEMBER 13, 2024

With the implementation of technology rapidly increasing, stricter regulatory standards, and growing demand for innovative treatments, the trends within the industry continue to evolve, impacting the future of clinical trials both positively and negatively.

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Clinical Trials Trials Drug Development Treatment 95

Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Disease Treatment 52

Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The pipeline currently includes five drug candidates, all of which have the potential to revolutionise the treatment of PD.

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Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Disease Treatment 52

Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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Fierce BioTech

SEPTEMBER 19, 2024

Madeline Naylor, VP of Health Informatics RWD at Norstella, and Patrick Winniewicz, Executive Director of Healthcare Analytics Solutions at Quest Diagnostics, will discuss how today’s robust lab data sources can help improve patient access and time to treatment—and, ultimately, lead to better patient outcomes.

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Treatment Pharma Companies Disease 52

Broad Institute

OCTOBER 25, 2024

Autoimmune Disease ML Challenge - Lecture 1, Part C - Biology - How is a Diagnosis Made? By Rose Circeo October 25, 2024 Breadcrumb Home Autoimmune Disease ML Challenge - Lecture 1, Part C - Biology - How is a Diagnosis Made? The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024.

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Disease Treatment 52

Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

Disease 52

Disease Treatment 52

Broad Institute

OCTOBER 25, 2024

Participants will identify genes that serve as markers for potential cancerous regions in the gut, facilitating the early detection and treatment of colorectal cancer. The challenge will run from October 28, 2024 - January 31, 2025. Register and learn more at broad.io/MLC-2024. MLC-2024.

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The Pharma Data

MAY 26, 2023

More ways, to save more lives, for less money: World Health Assembly adopts more Best Buys to tackle noncommunicable diseases The World Health Organization has expanded the list of ‘NCD best buys’. This gives countries of every income level support to improve the health of their citizens. Source link: [link]

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Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. How does SRP-001’s safety profile, especially concerning hepatotoxicity and nephrotoxicity, compare to other common pain medications?

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SugarCone Biotech

MARCH 2, 2025

An emerging concept is to localize the inhibition of VEGF or the inhibition of TGF- within the disease microenvironment, e.g. in the context of cancer and the TME. The interest from analysts is driven by near-term readouts from the COMPANION-002 Phase 2/3 Study including ORR in March and median PFS later in 2025.

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Fierce BioTech

JANUARY 16, 2025

The complex clinical planning, potential for nuanced regulatory hurdles, payer scrutiny, and supply chain complexity all necessitate early and strategic planning to optimize patient access to these novel treatment options at launch. On Demand Start Date Thu, 03/06/2025 - 13:00 End Date/Time Thu, 03/06/2025 - 12:00

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Fierce BioTech

JANUARY 31, 2025

Healthcare and life sciences professionals, researchers, and pharma leaders will learn how to gain deeper insights into treatment impact, disease progression, patient experiences by collecting real-world data through a direct connection to individuals. On Demand Start Date Tue, 02/25/2025 - 15:00 End Date/Time Tue, 02/25/2025 - 15:00

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The Pharma Data

JANUARY 15, 2021

for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. Enhertu is approved with Boxed WARNINGS for interstitial lung disease (ILD)/pneumonitis and embryo-fetal toxicityEnhertu. In the U.S.,

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Treatment Trials Disease Therapies 52

National Institute on Drug Abuse: Nora's Blog

NOVEMBER 4, 2024

This is true across diseases and disorders, and addiction research is no exception. Another study aimed at reducing overdose and increasing engagement in harm reduction and treatment services had originally been limited to people who use methamphetamine. We are accepting application statements through January 10, 2025.

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Science Research Treatment Drugs 85

Broad Institute

FEBRUARY 11, 2025

Its T cells in particular are famous for their ability to attack tumors, leading to immunotherapies that have revolutionized cancer treatment. Studies of cancer immunotherapy have largely focused on T cells, but the Bod lab seeks to understand whether the immune system's antibody-producing B cells can also be leveraged for cancer treatment.

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Medical Schools Immune Response Hospitals Doctors 52

The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Billion by 2025. billion by 2025, according to Grand View Research 1. NEW YORK and LONDON, Nov. NEW YORK and LONDON, Nov.

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