Codon

JULY 28, 2025

That year, Terrence Flotte’s team at Johns Hopkins University used a natural strain of AAV, called AAV2, to introduce a cystic fibrosis transmembrane conductance regulator (CFTR) gene — defective in those with cystic fibrosis — into patients during a phase 1 clinical trial.

Immune Response

New Drug Approvals

JULY 10, 2025

4] Names Sebetralstat is the international nonproprietary name. [5] “Sebetralstat (KVD900): A Potent and Selective Small Molecule Plasma Kallikrein Inhibitor Featuring a Novel P1 Group as a Potential Oral On-Demand Treatment for Hereditary Angioedema” Journal of Medicinal Chemistry. Hz), 6.39 (1H, ddd, J=0.7,

FDA

New Drug Approvals

MAY 26, 2025

M in tetrahydrofuran) was added at room temperature at a rate to maintain the internal temperature below 30 C. 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] equiv) in tetrahydrofuran (1.3

Small Molecule

New Drug Approvals

APRIL 18, 2025

Compound 8A was previously described in International Publication Number WO2010/125414 by Sanofi-Aventis.Example 1: Preparation of 4-(2-chloro-4-methoxy-5-methylphenyl)-N-[(1S)-2-cyclopropyl-1-(3-fluoro-4-methylphenyl)ethyl]-5-methyl-N-prop-2-ynyl-1,3-thiazol-2-amine (Compound 1), See FIG. in DCM (682 kg, 513 L, 7.3 kg, 1 eq.)

FDA

Codon

AUGUST 4, 2025

A small molecule “barcode” is added to each droplet and attaches itself to the proteins within. Scientists have already sequenced the RNA molecules of millions of brain cells from people with Alzheimer’s. Credit: PTI Next, these protein-filled droplets are studied using mass spectrometry.

Laboratories

New Drug Approvals

JULY 21, 2025

Sodium nitrite aqueous solution (sodium nitrite to compound d molar ratio is 1:1) was added, and the internal temperature was kept at no more than 5°C during the process. Hetrombopag Olamine , RAFUTROMBOPAG OLAMINE Hetrombopag diolamine SHR8735 olamine Hetrombopag ethanolamine SHR-8735 olamine 580.6 kg), 7-aminobenzo[d]oxazol-2(3H)-one (2.00

Small Molecule

Drug Target Review

JULY 6, 2023

1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional small molecules. 1 The concept of TPD was first demonstrated with a heterobifunctional small molecule to degrader a protein of interest in the early 2000s.

Targeted Protein Degradation

LifeSciVC

JANUARY 16, 2024

The expectation that Pharma will continue buy-ups here is helping to fuel conviction for another strong year ahead for M&A, especially for those acquirers who will rely on inorganic acquisition to build in areas under-represented by internal R&D (e.g., Small molecule GLP1s? Additional trials (e.g.,

Small Molecule

The Pharma Data

JANUARY 19, 2021

The Phase 1 clinical trial is planned to be conducted in Canada and targeted to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple- ascending dose (MAD) cohorts, respectively. About ALS-4.

Small Molecule

The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.

Small Molecule

The Pharma Data

NOVEMBER 19, 2020

Program has advanced rapidly under an international collaboration of companies led by Sosei Heptares as part of its commitment to socially responsible investing. Sosei Heptares has successfully designed three distinct series of compounds with attractive anti-viral drug-like properties and advanced the most promising molecules.

Small Molecule

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. The lead candidate, HTL0022562, has advanced through preclinical development demonstrating promising and differentiated properties for further investigation in human trials. About Sosei Heptares.

Small Molecule

Drug Target Review

AUGUST 25, 2023

However, in tissues where the 3 Hs are not present, the small molecule has anti-toxicity activity through 1) direct inhibition of the NLRP3 inflammasome, an inflammation starter and perpetuator, and 2) upregulation of the master antioxidant transcription factor, Nrf2.

Small Molecule

Conversations in Drug Development Trends

APRIL 4, 2024

To help integrate these various regulatory standards, the International Council for Harmonization (ICH) of Technical Requirements for Pharmaceuticals for Human Use was founded in 2016. The post Demystifying the ICH M10 Bioanalytical Method Validation Guidelines appeared first on Worldwide Clinical Trials.

Small Molecule

LifeSciVC

APRIL 24, 2024

In the absence of a clinical trial result or FDA label to point to, how does one create the case and target product profile (TPP) around a new target? and whether a molecule’s pharmacology can help to mitigate safety risk. Human and mouse genetics can inform not only efficacy but also safety. in liver, in CNS)?

Production

Drug Target Review

NOVEMBER 6, 2023

These regimens frequently include one or two small molecule inhibitors and immuno-oncology (IO) therapies, such as monoclonal antibodies and T-cell engagers. To bring us closer to curing cancer, a combination of effective drugs with non-overlapping mechanisms of action is required.

Small Molecule

LifeSciVC

JULY 6, 2023

Although these results cannot prove that there is no possibility of finding a high-affinity small molecule binder of NBD1, they are discouraging…” It turns out the small molecules they were looking for are not only possible – they were ready for a dedicated team to push them ahead.

Small Molecule

DrugBaron

JANUARY 31, 2024

A simple example is called projection, where simple linear combinations of the data are used to extract the internal patterns within very large datasets – tools that were rightly called pattern recognition methods. And pattern recognition is not intelligence.

Pharma R&D

The Premier Consulting Blog

NOVEMBER 20, 2024

In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial small molecule or biologic product to expedite product availability for patients. The quality of data from Stage I validation activities. Prior PPQ knowledge and experience with similar products.

Small Molecule

Nvidia Developer: Drug Discovery

MARCH 21, 2023

New supercomputing-scale large language models (LLMs) that understand biology and chemistry text are helping scientists understand proteins, small molecules, DNA, and biomedical text. These state-of-the-art AI models help generate de novo proteins and molecules and predict the 3D structures of proteins.

Small Molecule

The Pharma Data

JANUARY 31, 2021

Convergent is currently conducting advanced human trials relating to prostate cancer treatments involving peptide receptor radionuclide therapy (“PRRT”) that targets the prostate-specific membrane antigen (“PSMA”) present on prostate cancer cells. or its ability to internalize PSMA.

Small Molecule

The Pharma Data

NOVEMBER 18, 2020

. “Based upon our anti-Globo H targeted approaches in cancers of high unmet needs, OBI Pharma is proud to have presentations on the progress of our trial presented at ESMO-Asia 2020 for our novel therapeutic cancer vaccine, OBI-833.” Title: A phase I cohort expansion trial of OBI-833 in non-small cell lung cancer patients.

Vaccine

Practical Cheminformatics

JANUARY 8, 2024

In November and December, several large pharmas held “AI Day” presentations featuring LLM applications for clinical trial data analysis. These information retrieval capabilities have many applications, from writing computer code and collating clinical trial results to summarizing papers on a specific topic.

Drugs

The Pharma Data

JANUARY 19, 2021

TC-210 is currently in a Phase I/II trial for mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. TC-110 is in a Phase I/II trial for CD19-positive adult acute lymphoblastic leukemia (aALL) and with aggressive or indolent non-Hodgkin lymphoma (NHL).

Small Molecule

Agency IQ

APRIL 19, 2024

Meet C3TI, FDA’s new clinical trial innovation hub On April 14, FDA announced that its Center for Drug Evaluation and Research (CDER) is launching a new clinical trial innovation hub, the CDER Center for Clinical Trial Innovation (C3TI). set specific standards and expectations for discrete components of clinical trials (e.g.,

Clinical Trials

New Drug Approvals

JULY 22, 2025

mol) was added dropwise, slowly enough to maintain the internal temperature below -55 °C. mol) in THF (1 L) was then added dropwise, slowly enough to maintain the internal temperature below -50 °C. mol) in ethanol was added at 25 °C, then the mixture was heated to 75 °C internal and stirred for 30 hours. Hz, 1H), 2.25 (dd, J=8.0,

International

The Premier Consulting Blog

MAY 31, 2023

In this blog post, we outline typical elements of an IND-enabling package for a single ascending dose (SAD)/multiple ascending dose (MAD) study of an oral small molecule for a non-cancer indication with a maximum of 14 days of dosing.

Packaging

The Pharma Data

DECEMBER 15, 2020

SetPoint Medical received FDA Investigational Device Exemption (IDE) approval for a multicenter, double-blind, randomized, sham-controlled pivotal trial that will enroll up to 250 patients at 40 clinical trial sites in the U.S. Small Molecule Inhibitors. Oral, Small Molecules.

Disease

The Pharma Data

OCTOBER 18, 2020

Berk completed his internship, residency, and fellowship in internal medicine, hematology, and medical oncology at the Weill Cornell Medical College and New York Presbyterian Hospital, where he also served as a faculty member. Inflection Biosciences Ltd is developing small molecule therapeutics for the treatment of cancer.

Small Molecule

The Premier Consulting Blog

JANUARY 31, 2023

Food and Drug Administration (FDA) adopted an addendum to the guidance titled “ S1B(R1) Testing for Carcinogenicity of Pharmaceuticals ,” which had previously been finalized by the International Council for Harmonization (ICH). 1,2,3 Its appendix offers case studies on the application of WoE in determining the utility of 2-year rat studies.

Small Molecule

The Pharma Data

MAY 15, 2023

Through the acquisition, Gilead gains rights to a portfolio of small molecule inhibitors targeting PARP1 for oncology and MK2 for inflammatory diseases that could enter clinical trials later this year. XinThera is backed by a group of international investors including Foresite Capital, OrbiMed Advisors, LLC and TTM Capital.

Small Molecule

The Pharma Data

OCTOBER 6, 2021

The positive opinion by the CHMP is based on the clinical trial data shared by the companies, real world evidence as well as literature data. If the EC grants the variation regarding a booster dose, the decision will be immediately applicable to all 27 EU member states.

Vaccine

The Pharma Data

JANUARY 3, 2021

Based on the strong scientific rationale for telaglenastat in KEAP1/NRF2 mutant non-small cell lung cancer patients, and the safety profile observed in CANTATA, we remain dedicated to advancing our randomized KEAPSAKE trial.”. The CANTATA trial enrolled 444 patients at multiple centers globally. SOUTH SAN FRANCISCO, Calif.,

Trials

The Pharma Data

OCTOBER 14, 2020

International Dial-in Number: (210) 874-7715. Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. Conference Call Information : Date : Thursday, November 5, 2020. Time : 4:30 p.m. Domestic Dial-in Number : (866) 913-8546.

Pharmaceuticals

The Pharma Data

MAY 16, 2022

About Digestive Disease Week® Digestive Disease Week (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery.

Disease

The Pharma Data

OCTOBER 18, 2020

“We diligently and thoughtfully prepared for this trial,” said William G. The Company’s small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. Rice, Ph.D.,

Clinical Trials

New Drug Approvals

JANUARY 31, 2024

PATENT The free base and salts of the compound of formula (I) may be prepared for example, according to the procedures given in International Patent Application No. It works as a colony-stimulating factor 1 ( CSF1 ) receptor inhibitor. [1] PCT/US2007/066898 filed on Apr. 18, 2007 and published as WO2007/121484 on Oct.

Small Molecule