Drug Discovery World

MAY 15, 2024

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Specific numerical values (steps) denote the severity of NPDR, with 47-53 describing the moderately severe disease. orally administered compounds.

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Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. The team debuted a novel tool to instantly match microbes to the metabolites they produce earlier in 2024. 2023 February 5 [2024 March 11]; 15(1).

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KIF1A

FEBRUARY 18, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Read the Article The post #ScienceSaturday: February 17, 2024 appeared first on KIF1A.

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Elrig

APRIL 11, 2024

One of the great highlights of a:head’s work has been the ability to observe disease-specific phenotypes and profile clinically used therapeutics for their ability to alter neuronal network activity. He obtained his PhD from University of California, San Francisco, where he studied genetic regulation of neuron morphology using fruit flies.

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FDA Law Blog: Biosimilars

MARCH 31, 2024

Gibbs — On March 21, 2024, the House Energy and Commerce held a subcommittee hearing titled “Evaluating Approaches to Diagnostic Test Regulation and the Impact of the FDA’s Proposed Rule.” Van Meter also testified that this is even more consequential for pediatric patients, small patient populations and patients with rare diseases.

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Agency IQ

MAY 3, 2024

What We Expect the FDA to do in May and June 2024 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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FDA Law Blog: Biosimilars

DECEMBER 21, 2023

Both are celebrating their 75th anniversaries in 2024. 2024 brings a host of Hyman, Phelps & McNamara, P.C. 2024 brings a host of Hyman, Phelps & McNamara, P.C. (“HPM”) In other exciting news — in 2024 FDLI will be getting a make-over! What do Gene Autry’s Rudolph the Red-Nosed Reindeer and FDLI have in common?

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Agency IQ

MARCH 1, 2024

What We Expect the FDA to do in March and April 2024 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more. Read AgencyIQ’s analysis of a lower court’s ruling.

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Agency IQ

FEBRUARY 2, 2024

What We Expect the FDA to do in February and March 2024 (Updated) In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Drug Discovery World

APRIL 17, 2024

Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. What diseases are you currently targeting?

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Covalent Modifiers

JUNE 29, 2024

Commun Chem 7 , 144 (2024). Phosphorylation of their C-terminal RS domains by SR protein kinases (SRPKs) regulates their localization and diverse cellular activities. Dysregulation of phosphorylation has been implicated in many human diseases, including cancers.

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Agency IQ

FEBRUARY 12, 2024

What’s going on in the world of PFAS – early 2024 edition This feature from AgencyIQ focuses on the wide world of per- and polyfluoroalkyl substance regulation, legislation, and litigation around the United States. This edition reviews developments that occurred in late 2023 and early 2024.

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Drug Discovery World

NOVEMBER 22, 2023

Ascendis plans its first EU launch of YORVIPATH in January 2024 in Germany. “Each patient living with chronic hypoparathyroidism faces serious health and quality of life concerns,” said Professor Lorenz Hofbauer, Professor of Medicine, Geriatrics, and Endocrinology, Technical University of Dresden. “To

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Drug Discovery World

MAY 8, 2024

There has been an increasing focus over the past few years about the uses of AI and machine learning in drug discovery and development, particularly in their ability to identify molecules capable of treating disease and to simulate trial results. There is now growing pressure for regulators to take action.

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Agency IQ

OCTOBER 27, 2023

What we expect European regulators to do in November 2023 In this new recurring feature, AgencyIQ, through public data and previous analysis, determines what European medicine and device regulators will likely do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods, and more.

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SCIENMAG: Medicine & Health

JANUARY 10, 2024

10, 2024 – University of Pittsburgh Schools of Medicine researchers uncovered a fundamental mechanism that controls the body’s response to limited oxygen and regulates blood vessel disease of the lung. PITTSBURGH, Jan. Credit: Johnathan Wright PITTSBURGH, Jan.

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Drug Discovery World

JANUARY 5, 2024

The start of 2024 has seen quite a bit of regulatory activity, with the UK MHRA’s launch of a new procedure for assessing drugs, along with significant regulatory approvals for therapeutics for cytomegalovirus, melanoma, amyloidosis and multiple sclerosis. News round-up for 1-5 January by DDW Digital Content Editor Diana Spencer.

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Drugs.com

FEBRUARY 19, 2024

19, 2024 -- Raw milk cheese tainted with E. coli bacteria has sickened 10 people in four states, hospitalizing four, federal regulators warn. Centers for Disease. MONDAY, Feb. The cases have been tied to Raw Farm brand raw cheddar cheese, the U.S.

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Agency IQ

JUNE 26, 2023

FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. a disease or condition is defined as rare by the Orphan Drug Act (ODA) if it affects fewer than 200,000 people.

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Covalent Modifiers

MAY 9, 2024

Hopkins Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.4c00220 4c00220 Multiple sclerosis (MS) is a chronic disease with an underlying pathology characterized by inflammation-driven neuronal loss, axonal injury, and demyelination.

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Drug Discovery World

JUNE 7, 2023

s National Security and Investment Act, testimony to invoX’s continued willingness to build trust and work closely with regulators. McCarthy said: “At pHion Therapeutics, it is our mission to pioneer next generation, first-in-class vaccines for infectious disease and oncology through stealth-like delivery of mRNA.

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Drug Discovery World

MAY 21, 2024

This remarkable progress in antibody modalities for disease treatment is a testament to the effectiveness of these methods, and they remain key for many use cases. A significant fraction of GPCR receptors remain undrugged (around 80% of this receptor family) for an estimated 227 untapped targets with known disease links.

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Drug Discovery World

NOVEMBER 10, 2023

“This new technology offers hope to patients living with late stage inherited retinal diseases. Once inside the cell, KIO-301 localises within specific voltage-gated ion channels involved in regulating neural signalling.

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Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr

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Broad Institute

FEBRUARY 27, 2024

Broad-MIT Seminars in Chemical Biology By Rose Circeo February 27, 2024 Breadcrumb Home Research Broad-MIT Seminars in Chemical Biology MIT Chemistry and the Broad Institute host monthly seminars in chemical biology with the intent to hear from the thought-leaders and bring together chemical biologists from both academia and industry in the Boston/Cambridge (..)

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Drug Discovery World

MAY 6, 2024

The company is now investigating a promising gene therapy for Huntingdon’s disease in a Phase I/II trial, with patients already showing signs of preserved neurological function. The rights for etranacogene dezaparvovec were subsequently sold to CSL Pharmaceuticals. This pedigree has attracted beneficial international collaborations.

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FDA Law Blog: Biosimilars

OCTOBER 16, 2023

Three of these articles focus on medical applications of Artificial Intelligence and Machine Learning (AI/ML) and explore FDA’s role in regulating such products. Just a day earlier, FDA’s Center for Devices and Radiological Health (CDRH) released a list of the guidance documents the Center intends to publish in the upcoming 2024 fiscal year.

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Agency IQ

JUNE 7, 2024

Almost 10 years late, the new framework’s arrival will represent significant changes in the regulation of these pesticide substances. Active substances that have been approved for use in the EU are listed in the Annex of Regulation (EU) No 540/2011; currently, approximately 450 entries are listed.

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Drug Discovery World

FEBRUARY 6, 2024

However, many have cautioned against too great a reliance on these tools and emphasised the importance of checks and balances to ensure the systems are properly regulated and results are verified. Auransa, which harnesses available data to discover highly intractable diseases with poorly understood biology.

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Agency IQ

FEBRUARY 15, 2024

MHRA selects eight technologies to help it test its new innovative devices pathway The British regulator and its partners just released the list of products and companies chosen to participate in the pilot of its Innovative Device Access Pathway (IDAP). BY COREY JASEPH, MS, RAC | FEB 14, 2024 6:11 PM CST Quick background on the future U.K.

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Drug Discovery World

MAY 17, 2024

Yet, while the number of cancer clinical trials has soared from 19,211 in 2013 to a whopping 26,396 in 2022 3 , and they keep surging in 2024, the success stories are few and far between. There are more – regulations, validations and integrations. Accessed January 6, 2024. Accessed January 6, 2024. Cancer Key Facts.

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Agency IQ

JUNE 28, 2024

FDA draft guidance on addressing misinformation clears White House review A few months ahead of a potential administration transition, the White House has cleared (with changes) a new question-and-answer draft guidance document focused on “addressing misinformation” about regulated life sciences products.

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Broad Institute

MARCH 14, 2024

By Leah Eisenstadt March 14, 2024 Credit: Courtesy of the authors. Related groups Liu lab Choudhary lab Fischer lab Researchers studying the role of proteins in health and disease use experimental tools that inactivate proteins, destroy them, or prevent them from being made in cells. March 14, 2024.

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Targeted Protein Degradation Small Molecule Medical Schools Science 141

Agency IQ

MAY 3, 2024

What we expect European regulators to do in May 2024 In this recurring feature, AgencyIQ, through public data and previous analysis, determines what European medicine and device regulators will likely do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods, and more.

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Drug Discovery World

JANUARY 17, 2023

Genoscience Pharma, a clinical-stage biotech company developing lysosomotropic drug candidates for the treatment of cancer, fibrosis and auto-immune diseases through autophagy modulation has launched the ABE-LIVER study, along with trial sponsor Grenoble University Hospital. . The trial . Context .

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KIF1A

JUNE 10, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. CX32 likely regulates this cooperation, as knocking down CX32 weakened the connection between SFXN-1.2 Knocking down either SFXN-1.2

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Clinical Trials FDA Regulations Disease 98

DrugBank

MAY 6, 2024

This category encompasses a broad spectrum of treatments including vaccines, blood components, gene therapies, and more, each tailored to combat diseases in highly specific ways. A Wave of Innovations The year 2024 has already proven to be significant for the field of biologics, witnessing a notable increase in FDA approvals.

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