Drug Target Review

APRIL 7, 2025

The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions. References Sun D, et al. Acta Pharmaceutica Sinica B , 12(7), pp.

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DrugBank

JUNE 18, 2025

In 2020, the FDA halted Johnson & Johnson’s COVID-19 vaccine production at a contract manufacturing facility after discovering quality control issues. Regulatory bodies require strict adherence to Good Manufacturing Practices (GMPs) to ensure that drugs are consistently produced at high quality.

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PPD

DECEMBER 16, 2024

This struggle is further complicated for small sponsors conducting trials in rare disease areas where limited patient pools are available, and for all sponsors facing diversity requirements that mandate reaching underrepresented populations.

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Drug Development Clinical Trials Compliance Trials 52

SugarCone Biotech

MARCH 24, 2025

Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. These therapeutics share some characteristic measures of clinical efficacy, including high initial overall response rates but a low rate of complete responses and limited duration of response. doi: 10.1073/pnas.1716266115

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New Drug Approvals

APRIL 25, 2025

World Health Organization (2020). 3] Primary endocrine resistance was defined as relapse while on the first two years of adjuvant endocrine therapy (ET) and secondary endocrine resistance was defined as relapse while on adjuvant ET after at least two years or relapse within twelve months of completing adjuvant ET. [3] Foundation Medicine.

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New Drug Approvals

MAY 9, 2025

Eligible participants had experienced disease progression on one or two prior lines of endocrine therapy , including one line with a CDK4/6 inhibitor , and could have received up to one prior line of chemotherapy in the advanced or metastatic setting. [2] 1] [4] It is taken by mouth. [1] 3] [7] PATENTS Cruskie MP, et al.

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Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 Cell (2020). million people every year. Credit: Stokes J.M.

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Codon

JUNE 5, 2025

Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.

DNA 81

DNA RNA Clinical Trials Engineering 81

LifeSciVC

JUNE 12, 2025

Building medicines by activating biological pathways As I joined the Company in Q4 2020 as its CEO, I had a vision of creating protein variants to activate biological pathways. was based on experience building on the understanding that a single protein variant, could have markedly different outcomes for patients with chronic kidney disease.

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RNA Protein Expression DNA Science 67

BioPharma Drive: Drug Pricing

JUNE 11, 2025

Like many other biotechnology firms, it’s seen its stock price fall significantly during a sector-wide pullback, with shares losing more than three quarters of their value since 2020. Ibtrozi has also shown the ability to help people whose cancer has spread to the brain, a leading cause of disease progression, the company said.

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FDA Drugs Therapies Science 270

New Drug Approvals

JUNE 29, 2025

2020/0062765) was dissolved in ethanol (604 mL) at 50℃. 3] Participants may have received prior chemotherapy for advanced disease. [3] 3] Participants may have received prior chemotherapy for advanced disease. [3] obtained by using the method described in Example 5 of U.S. Application Publication No. Sodium hydroxide (68.1

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International FDA Clinical Trials Treatment 62

New Drug Approvals

JUNE 15, 2025

Its sodium salt is used for the treatment of generalised myasthenia gravis (a disease that leads to muscle weakness and tiredness) in adults whose immune system produces antibodies against acetylcholine receptors. [1] Zilucoplan is a 15 amino-acid, synthetic macrocyclic peptide with formula C172H278N24O55. 24 September 2024.

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FDA Treatment Clinical Trials Production 57

National Institute on Drug Abuse: Nora's Blog

NOVEMBER 25, 2024

2 Drug use contributes to the spread of HIV and other infectious diseases like hepatitis B and C in multiple ways, including the sharing of drug injection equipment. Interventions to prevent HIV and Hepatitis C among people who inject drugs: Latest evidence of effectiveness from a systematic review (2011 to 2020). Int J Drug Policy.

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Drugs Treatment Disease Science 119

Codon

NOVEMBER 1, 2024

Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. This essay focuses on how we might do both, specifically for the cell. Subscribe to Asimov Press.

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DNA RNA Molecular Biology Laboratories 132

BioPharma Drive: Drug Pricing

JULY 18, 2025

The FDA expanded Elevidys’ approval the next year, allowing a substantially larger group of patients to receive it, including those whose disease had eroded their ability to walk. Published July 18, 2025 • Updated 3 hours ago Ned Pagliarulo Lead Editor post share post print email license The U.S.

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Therapies FDA Clinical Trials Licensing 159

New Drug Approvals

JULY 8, 2025

US FDA granted fast track designation for pritelivir in 2017 and breakthrough therapy designation 2020. HSV in immunocompromised patients Although HSV infection is very common in the general population, it rarely causes serious disease and is effectively contained by the immune system. Infectious Diseases Community of Practice (2013).

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Virus DNA Clinical Trials Treatment 62

New Drug Approvals

APRIL 18, 2025

4] A medication used to reduce the amount of steroid replacement required in patients with a genetic disease that causes, amongst other symptoms, a steroid deficiency. 8 May 2020). . : 321839-75-2 Molecular Weight 519.50 1] It is taken by mouth. [1] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2]

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FDA FDA Approval Drugs Trials 62

Drug Patent Watch

JANUARY 13, 2025

. “Biosimilars represent one of the fastest growing segments in the pharmaceutical industry, with the potential to dramatically alter the treatment landscape for numerous chronic and life-threatening diseases.”[1] from 2020 to 2025[1]. billion by 2025, growing at a CAGR of 34.2%

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Biosimilars Marketing Treatment Small Molecule 61

BioPharma Drive: Drug Pricing

JULY 9, 2025

has agreed to pay $10 billion to buy London-based Verona Pharma, scooping up a potential multibillion-dollar new drug to treat chronic obstructive pulmonary disease in a deal announced Wednesday. Published July 9, 2025 By Kristin Jensen post share post print email license Merck & Co. Alamy Merck & Co.

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Clinical Trials Drugs Therapies Licensing 277

Broad Institute

JULY 17, 2025

She was previously a member of the Broad scientific community, beginning in 2006 as a core faculty member and later, Broad’s chair of the faculty from 2015 to 2020. This has led to a far deeper and more profound understanding of health and disease.& She is also co-founder of the Human Cell Atlas.

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FDA Law Blog: Biosimilars

OCTOBER 13, 2024

Tobolowsky — The Rare Pediatric Disease Priority Review Voucher program has had a bit of a tumultuous history in its 12 short years of existence. Over 50 rare pediatric disease priority review vouchers have been awarded to date. Notably, it was reauthorized in 2016 (until 2020) and in 2020 (until 2024).

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Disease FDA Approval FDA Government 64

Drug Patent Watch

JANUARY 7, 2025

” WHO.int, 2020. “The Use of Generic Drugs in Prevention of Chronic Disease Is Far More Cost-Effective Than Thought, and May Save Money.” . “The High Cost of Prescription Drugs in the United States: Origins and Prospects for Reform.” ” JAMA, 2016. World Health Organization. Shrank, W.H.,

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Drugs FDA Doctors Trials 71

New Drug Approvals

JULY 18, 2025

3] It was discovered for the treatment of hepatitis C [4] and has since been investigated for use against other viral diseases such as AIDS and COVID-19 , [2] [5] for which it was granted conditional approval in China. [6] 2020, 63, 8554−8566. (7) China Patent CN 111892636, 2020. Harrison C (April 2020). 7) Chang, J.

RNA 62

RNA Clinical Trials Trials Virus 62

Codon

JUNE 9, 2025

” The poet-scientist Goethe, in 1798, proclaimed that life seen from a high enough altitude looked “like a malignant disease.” As a passing rhetorical flourish, he reached for the now-familiar phrasing, asking whether we might look upon biology as “a disease which affects matter in its old age.”

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Science Disease Production Protein Production 95

Science Daily: Pharmacology News

SEPTEMBER 19, 2024

The study is based on a new analysis of metatranscriptomic data released by the Chinese Center for Disease Control and Prevention (CDC). The data come from more than 800 samples collected in and around the Huanan Seafood Wholesale market beginning on January 1, 2020, and from viral genomes reported from early COVID-19 patients.

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Marketing Virus Disease 308

Drugs.com

JULY 6, 2023

Centers for Disease Control and Prevention estimates that there were nearly 68,000 new cases of acute hepatitis C in 2020 and over 107,000 newly reported cases of chronic hepatitis C? THURSDAY, July 6, 2023 -- Did you know the U.S. To help you.

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BioPharma Drive: Drug Pricing

AUGUST 3, 2023

The biotech is acquiring rights to an RNA interference medicine Alnylam invented for a rare blood disorder, continuing a corporate makeover that began in 2020.

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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. We're excited to expand our data offering by launching a new Rare Disease add-on to complement our Clinical Trial dataset.

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Disease Clinical Trials Trials FDA 98

Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC).

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Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

Disease 59

Disease Clinical Trials Therapies Science 59

Broad Institute

JUNE 18, 2024

By Allessandra DiCorato June 18, 2024 Credit: Jon Arizti-Sanz SHINE, a rapid diagnostic test developed by Pardis Sabeti's lab in 2020, uses paper strips and CRISPR enzymes to identify specific sequences of viral RNA in samples. Tags: Infectious Disease Diagnostics Pardis Sabeti Paper cited Zhang YB, Arizti-Sanz J, et al. 2024.04.004.

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Drug Discovery Today

MAY 21, 2020

Glasgow, Scotland, 21 May 2020 - Amphista Therapeutics, a biopharmaceutical company creating first-in-class cancer therapeutics that harness the body’s natural processes to remove disease causing proteins selectively and efficiently, today announced the appointment of Dr Ian Churcher as Chief Scientific Officer (CSO).

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Disease 100

Drug Target Review

FEBRUARY 14, 2024

Researchers from the Michigan State University have created an advanced human heart organoid system that models the characteristics of pregestational diabetes-induced congenital heart disease found in mice and humans. The most common type of congenital defect in humans is congenital heart disease.

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Disease Research Drugs 59

Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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addgene Blog

FEBRUARY 13, 2024

Monoclonal antibody drugs are popular therapeutics for a plethora of disease conditions, from cancer to autoimmune disorders. Antibodies administered as drugs are still immunogenic, meaning that they elicit an immune response from the body.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Drug Discovery Today

JULY 15, 2020

Amsterdam, The Netherlands, 23 June 2020 - Scenic Biotech BV (“Scenic”), a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, today announced that it has been awarded a €3.1

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