DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. This can potentially correct the root causes of some diseases at the genetic level.

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Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. So, what makes these therapies unique?

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ASPET

SEPTEMBER 7, 2023

Pompe disease is a rare glycogen storage disorder caused by a deficiency in the lysosomal enzyme acid α-glucosidase, which leads to muscle weakness, cardiac and respiratory failure, and early mortality. Avalglucosidase alfa has received marketing authorization in several countries for infantile-onset and/or late-onset Pompe disease.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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DrugBank

MAY 15, 2024

Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.

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DrugBank

JANUARY 9, 2025

Targeted Therapies vs. Traditional Treatments Traditional cancer treatments, such as chemotherapy and radiotherapy, are inherently cytotoxic, targeting both cancerous and healthy cells. This lack of selectivity often results in significant side effects, including nausea and limited efficacy, particularly in advanced or metastatic disease.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. 2 It is this complexity that necessitates powerful, targeted combination therapies. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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Drug Target Review

OCTOBER 6, 2023

This interview explores how CoRegen’s innovative techniques are not only transforming cancer treatment but also hold promise for a wide range of medical applications, all while minimising the side effects often associated with traditional therapies. What are the key benefits of your approach?

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Drug Target Review

SEPTEMBER 11, 2024

How does morADC technology combine the properties of small molecules and monoclonal antibodies to enhance anti-aggregation effects for CNS applications? The morADC are able to cross the blood-brain barrier more efficiently and offer higher potency than individual parent molecules.

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Drug Target Review

JULY 23, 2024

Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. TPD engages the body’s natural protein recycling system by selectively eliminating disease-causing proteins, ultimately addressing the root cause of disease.

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KIF1A

JANUARY 27, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. The study found improved spasticity and movement in mice treated with ARL61P1 gene therapy; it also restored abnormalities observed in the brains of ARL61P1-deficient mice.

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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

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The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. We are now committed to pursuing other CGRP-mediated diseases through advancing novel investigational agents such as HTL0022562 into human studies.” TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,

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LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

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The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with neurodegenerative diseases for potential disease modification. Multiple System Atrophy.

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The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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New Drug Approvals

MAY 26, 2025

Ervogastat CAS 2186700-33-2 Non-alcoholic Steatohepatitis (NASH) with Liver Fibrosis (FAST TRACK – U.S.) 2-[5-[(3-Ethoxy-2-pyridinyl)oxy]-3-pyridinyl]- N -[(3 S )-tetrahydro-3-furanyl]-5-pyrimidinecarboxamide ( S )-2-(5-((3-Ethoxypyridin-2-yl]oxy]pyridin-3-yl)- N -(tetrahydrofuran-3-yl)pyrimidine-5-carboxamide PF 06865571 BSOIY5AKQW 407.4

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The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. Does it have to be in a disease model? The cell or gene therapy should be pharmacologically active in the animal model. The cell or gene therapy should be pharmacologically active in the animal model.

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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The Pharma Data

JANUARY 31, 2021

Patent US10835501B2 Begins Development of Indication BioSciences’ Intellectual Patent Portfolio for statin plus cannabinoid combination therapies. Current statin therapies are associated with adverse effects including myalgia. SILVERTHORNE, Colo.–(

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The Pharma Data

JUNE 7, 2022

AL01811 is a preclinical selective GBA2 inhibitor with first-in-class potential as an oral disease modifying treatment for Parkinson’s Disease Alectos to receive a $15 million upfront payment and is eligible to receive potential future development and commercial milestone payments. Biogen Inc. President and CEO at Alectos Therapeutics.

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The Pharma Data

AUGUST 7, 2021

Vividion’s platform is able to produce a variety of small molecule therapies across indications, with initial focus on targets relevant to oncology and immunology. The acquisition of Vividion strengthens Bayer’s small molecule capabilities and expands Bayer’s reach into new modalities.

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Drug Target Review

MAY 6, 2024

The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development. Melanoma was an ideal disease for studying this phenomenon because cutaneous tumour biopsies are readily available. This caused many scientists to study the mechanisms that drive drug resistance.

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Drug Target Review

JULY 12, 2023

These assays provide insights into the molecular mechanisms of disease biology and drug response, enabling the characterisation of gene expression profiles and deviations in diseased cells. Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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The Pharma Data

MARCH 30, 2021

Preclinical Program Targeting 15-PGDH has Potential use in a Broad Range of Therapeutic Applications Including Inflammatory Disease Indications. “The enzyme 15-PGDH plays a key role in many disease-relevant processes such as stem cell self-renewal and epithelial barrier repair. senior vice president of Global Research at Amgen.

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The Pharma Data

JANUARY 25, 2021

–( BUSINESS WIRE )– Veralox Therapeutics, a biotechnology company developing first-in-class small molecule therapeutics that treat the underlying pathologies of diseases with significant unmet medical needs, today announced that the U.S. 26, 2021 13:00 UTC. FREDERICK, Md.–( About VLX-1005.

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Drug Target Review

MAY 4, 2023

These studies are conducted by comparing the genomes of large populations to find variants that occur more often in those with a specific disease or trait. These studies are conducted by comparing the genomes of large populations to find variants that occur more often in those with a specific disease or trait. “A

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The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. There is a great need for a disease-modifying treatment for the 5.3 CLARKSVILLE, Md.,

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Drug Target Review

DECEMBER 6, 2023

Dear readers, Today’s drug screening methods predominantly rely on one or two types of data, limiting their ability to replicate the intricate nature of disease biology. We are committed to providing you with the highest quality content, and we believe that this report will deliver a different perspective on the topics covered.

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Drug Target Review

MAY 23, 2023

Inflammatory bowel disease (IBD) is an umbrella term that describes complex disorders that cause chronic inflammation in the digestive tract with alternating periods of relapse and remission. Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. More than 6.8

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The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. Bayer today announced that it has entered into an agreement to acquire Noria Therapeutics Inc. Noria) and PSMA Therapeutics Inc.

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We they can help many patients.”.

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