Broad Institute

JULY 24, 2025

By Leah Eisenstadt July 24, 2025 Credit: CDC/ Frederick A. The approach could be used to explore the role of various proteins during infection with other pathogens, as a way to find new drugs for hard-to-treat infections. Murphy New treatments are sorely needed for Ebola virus infections, which cause rare, yet severe, outbreaks.

Virus

Drug Target Review

JULY 21, 2025

2 A raft of emerging therapeutic modalities sits at the centre of this boom, spanning advanced biologics, engineered platforms and next-generation small molecules. However, the advanced nature of the drugs being developed has brought new challenges. The global market for CNS therapeutics was worth an estimated $144.3

Drug Development

Collaborative Drug

JULY 28, 2025

PK Intravenous Dosing: Exponential Decay Equation Intravenous (IV) dosing refers to the administration of a drug directly into the bloodstream by injection (bolus) or continuous infusion. This approach is essential for understanding drug absorption, distribution, metabolism, and elimination (ADME).

Testimonials

Alta Sciences

JULY 3, 2025

Altasciences At CPHI Americas 2025 pmjackson Thu, 07/03/2025 - 13:41 In the ever-changing drug development world, staying agile is essential to enabling more informed decisions, faster. It’s a challenge that affects a large percentage of new drug candidates—one we’re passionate about helping to solve.

Small Molecule

Broad Institute

AUGUST 5, 2025

CDoT landing page By Rose Circeo August 5, 2025 Breadcrumb Home CDoT landing page Featured description text here Broad scientists are continually making new biological discoveries that have the potential to create new and transformative medicines.

Small Molecule

Drug Target Review

JULY 3, 2025

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This has led drug developers to unintentionally limit their potential within chosen therapeutic spaces.

Drug Development

Drug Target Review

JUNE 16, 2025

G protein-coupled receptors (GPCRs) are a highly validated drug target family – accounting for 30-35 percent of all approved drugs. Despite this, the therapeutic potential of targeting most GPCRs remains untapped, as only 10 percent of GPCRs have been drugged.

Disease

Drug Target Review

JUNE 11, 2025

Drug development is plagued by complex challenges, but multimodal AI is unlocking new opportunities. By integrating diverse data sources – from genomics to clinical insights – this approach is accelerating drug discovery, improving patient stratification and boosting success rates.

Drugs

Alta Sciences

JULY 18, 2025

Highlights From the 2025 American Society for Mass Spectrometry Conference | By Jean-François Larocque and Benjamin Charron aasimakopoulos Tue, 07/22/2025 - 18:05 We were thrilled to represent Altasciences at this year’s American Society for Mass Spectrometry (ASMS) conference in Baltimore. Curious to see this poster in action?

Assay Development

The Pharma Data

JUNE 12, 2025

The presentations highlight the ongoing progress and growing promise of the company’s innovative therapeutic platform, featuring its investigational oral CELMoD™ (Cereblon E3 Ligase Modulating Drugs) agents mezigdomide, iberdomide, and golcadomide, alongside its first-in-class, oral BCL6 (B-Cell lymphoma 6) ligand-directed degrader (LDD) BMS-986458.

Targeted Protein Degradation

Drug Target Review

JUNE 16, 2025

Unlike traditional pharmacokinetic (PK) studies that rely on plasma measurements alone, MSI, when combined with traditional histology, enables spatial mapping of drug distribution, metabolism and target engagement. Toxicological investigations play a key role in assessing drug safety and, importantly, inform development decisions.

Drugs

The Pharma Data

JUNE 25, 2025

The drug’s mechanism also aims to delay the overall progression of disease severity—an urgent need in a patient population that often progresses to end-stage renal disease. In March 2025 , Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinical trial for farabursen. Eastern Time on June 25, 2025.

RNA

Pharma Manufacturing

JUNE 17, 2025

Magazine ENEWS SUBSCRIBE WEBINARS PODCASTS EBOOKS ABOUT US Edit submissions Media kit Innovation Awards Articles Flow state: The evolving shape of continuous manufacturing June 17, 2025 While the pharmaceutical industry has been slow to adopt continuous manufacturing, it appears to be gaining momentum.

RNA

BioPharma Drive: Drug Pricing

JULY 8, 2025

Published July 8, 2025 Gwendolyn Wu Senior reporter post share post print email license Proteasomes are cellular machines for breaking down proteins. Unlike most approved drugs, which work by turning cell signaling up or down like a dimmer switch, this approach aims to rid cells of problem proteins entirely.

Targeted Protein Degradation

New Drug Approvals

JULY 2, 2025

Orforglipron ( LY-3502970 ) is an oral, non-peptide, small-molecule GLP-1 receptor agonist developed as a weight loss drug by Eli Lilly and Company. [1] Orforglipron ( LY-3502970 ) is an oral, non-peptide, small-molecule GLP-1 receptor agonist developed as a weight loss drug by Eli Lilly and Company. [1]

Small Molecule

New Drug Approvals

APRIL 2, 2025

1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 Journavx is the first drug to be approved in this new class of pain management medicines.

FDA

New Drug Approvals

JULY 10, 2025

g/mol O5ZD2TU2B7 FDA 7/3/2025, Ekterly, To treat acute attacks of hereditary angioedema N -[(3-fluoro-4-methoxypyridin-2-yl)methyl]-3-(methoxymethyl)-1-[[4-[(2-oxopyridin-1-yl)methyl]phenyl]methyl]pyrazole-4-carboxamide Sebetralstat , sold under the brand name Ekterly , is a medication used for the treatment of hereditary angioedema. [1]

FDA

The Pharma Data

JUNE 9, 2025

Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co., In parallel, we are working to file a New Drug Application (NDA) to the U.S. Food and Drug Administration, with additional filings planned in other markets.”

Treatment

Codon

JULY 28, 2025

Small molecule conjugation offers another approach , with molecules like GalNAc enhancing hepatocyte targeting through asialoglycoprotein receptors (useful for patients lacking LDLR), vitamin A derivatives targeting cells involved in fibrosis, and bisphosphonates targeting bone tissue. ” Asimov Press (2025).

Immune Response

New Drug Approvals

MAY 26, 2025

S2CID 253257260. ^ “With the right partner, Pfizer gains fast-track tag for previously shelved NASH drug” Retrieved 20 November 2023. Ervogastat CAS 2186700-33-2 Non-alcoholic Steatohepatitis (NASH) with Liver Fibrosis (FAST TRACK – U.S.) 1] Its development was previously halted by the company but resumed in 2022. [2]

Small Molecule

New Drug Approvals

APRIL 18, 2025

2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 1] [2] Adverse effects The US Food and Drug Administration prescription label for crinecerfont has a warning for acute adrenal insufficiency or adrenal crisis. [2] Retrieved 25 January 2025. Food and Drug Administration (FDA).

FDA

Alta Sciences

APRIL 24, 2025

Case Study: How Altasciences Overcame Pharmacological Challenges in a GLP-1 IND-Enabling Study pmjackson Thu, 04/24/2025 - 21:43 For emerging therapies with pronounced pharmacological effects, thoughtful study design is critical to ensuring reliable and interpretable data. This blog was originally published in April 2025.

Small Molecule

Drug Patent Watch

JANUARY 13, 2025

As we delve into the intricate world of biosimilar market dynamics, we’ll explore how these complex molecules are reshaping treatment paradigms across diverse patient populations. billion by 2025, growing at a CAGR of 34.2% from 2020 to 2025[1]. . billion by 2025, growing at a CAGR of 34.2% from 2020 to 2025[1].

Biosimilars

New Drug Approvals

JULY 6, 2025

It is a small molecule delivered orally. [1] “Prospect of acromegaly therapy: molecular mechanism of clinical drugs octreotide and paltusotine” Nature Communications. amcrasto@gmail.com NEW DRUG APPROVALS ONE TIME $10.00 amcrasto@gmail.com NEW DRUG APPROVALS ONE TIME $10.00 mmol) and DIPEA (1.19

Small Molecule

Fierce BioTech

MAY 27, 2025

jgrcic Tue, 05/27/2025 - 11:55 Tue, 05/27/2025 - 11:55 Resource Type Whitepaper Promotion Start Tue, 05/27/2025 - 12:06 Use of healthy volunteers in first-in-human trials with oncology small molecules. Can an alternative approach expedite clinical development of small molecule oncology drugs and benefit patients?

Small Molecule

Molecular Design

DECEMBER 31, 2024

My pulse will be quickenin' With each drop of strychnine We feed to a pigeon It just takes a smidgin To poison a pigeon in the park Tom Lehrer, Poisoning Pigeons in the Park | video ** Ill be reviewing the H2024 study (Occurrence of Natural Selection in Successful Small Molecule Drug Discovery) in this post.

Small Molecule

BioPharma Drive: Drug Pricing

JULY 14, 2025

Published July 14, 2025 Ben Fidler Senior Editor post share post print email license Bain Capital, Kohlberg and Mubadala joined to invest in biopharmaceutical CDMO PCI Pharma on July 14, 2025. PCI helps biopharmaceutical companies manufacture and package drug products used in clinical trials as well as commercially.

Production

Codon

AUGUST 4, 2025

Today, it is used in everything from chemical analysis to drug discovery, but it was not initially developed with biologists in mind. A small molecule “barcode” is added to each droplet and attaches itself to the proteins within. ” Asimov Press (2025). Niko McCarty is a founding editor of Asimov Press.

Laboratories

Codon

JULY 17, 2025

This observation, borne of accidental exposure, would eventually spur one of the first weight loss drug crazes — long before Ozempic and Mounjaro. In addition, upward of 20 wholesale drug firms are marketing the compound, which suggests that a considerable population is being medicated. Still, there wasn’t much the U.S.

Drugs

FDA Law Blog: Biosimilars

SEPTEMBER 11, 2024

Claud — Last week, FDA revised one of its two guidances relating to nitrosamines, Control of Nitrosamine Impurities in Human Drugs. Nitrosamines are impurities that can form during drug manufacturing and are considered potentially potent carcinogens. The revisions due in 2025 include testing for NDSRIs.

Small Molecule

LifeSciVC

JANUARY 16, 2024

Small molecule GLP1s? Whether trial design, execution, or otherwise, drug development even where there is precedent is a challenging road and should not be taken for granted. Whether it’s small molecules to target the incretins (GLP1R ± GIPR), targeting well-validated signaling nodes in I&I (e.g.,

Small Molecule

Alta Sciences

FEBRUARY 10, 2025

Let Us Guide You Through the Process blussier Mon, 02/10/2025 - 17:54 HTML Let Us Guide You Through the Process Altasciences' experts streamline the IND/CTA application process by designing your regulatory strategy and thoroughly planning and completing your required preclinical studies.

Small Molecule

Fierce BioTech

JANUARY 3, 2025

Optimizing Formulation Strategies for Poorly Soluble Compounds: Insights into Bioavailability Enhancement and Accelerated Development dwunderlin Fri, 01/03/2025 - 15:54 Wed, 02/19/2025 - 11:00 Resource Type Webinar Feng Zhang, Ph.D. On Demand Start Date Wed, 02/19/2025 - 12:00 Hong Li, Ph.D.

Small Molecule

New Drug Approvals

JUNE 26, 2025

Acoltremon AR-15512 CAS 68489-09-8 WeightAverage: 289.419 Monoisotopic: 289.204179113 Chemical FormulaC 18 H 27 NO 2 FDA 2025, 5/28/2025, To treat the signs and symptoms of dry eye disease Tryptyr WS-12 WS 12 (1R,2S,5R)-N-(4-methoxyphenyl)-5-methyl-2-(propan-2-yl)cyclohexane-1-carboxamide Fema No. Food and Drug Administration (FDA).

FDA

The Premier Consulting Blog

AUGUST 8, 2024

On July 31, 2024, the US Food and Drug Administration (FDA) announced Fiscal Year 2025 (FY2025) Prescription Drug User Fee Amendments of 2022 (PDUFA VII) fee rates for the review of human drug and biological product applications along with prescription drug program fees.

Drugs

New Drug Approvals

APRIL 30, 2025

1] [2] [3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C. [2] 2] The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review , fast track , orphan drug , and rare pediatric disease designations. [2]

FDA

The Pharma Data

JANUARY 19, 2021

On January 19, its investigational new drug (IND) for GC019F, a FasTCAR-enabled CAR-T therapy in relapsed or refractory adult B-cell acute lymphoblastic leukemia (B-ALL) was approved by China’s National Medical Products Administration (NMPA). Its first drug is PHVS416 for HAE, with plans to initiate a Phase II trial this year.

Small Molecule