Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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The Pharma Data

NOVEMBER 17, 2020

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . This includes therapies against the nuclear receptor ROR?t,

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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. In some cases, cell therapy can be curative for the patient.

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Broad Institute

OCTOBER 4, 2023

By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new small molecule inhibitor (green) sits inside the PTPN2 protein, where acidic sites are marked in red and basic sites are marked in blue. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.

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DrugBank

MAY 15, 2024

This method was more about serendipity than science. 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. One approach is to look beyond the traditional drug molecule.

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Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. It is somewhat like how PROTACs have changed the perspective on what is a tractable therapeutic target.

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DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle. These complex molecules require precise engineering to ensure optimal efficacy and safety.

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Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Pushing the boundaries of science always opens the door to new, impactful developments, although there are always challenges along the way.

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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

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

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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. Small molecule GLP1s?

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Drug Target Review

MAY 6, 2024

Luckily, my love for science remained. The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development. We found that, indeed, cancer’s earliest defence mechanisms are triggered almost immediately after exposure to a targeted cancer therapy.

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LifeSciVC

APRIL 2, 2024

The quality of science – and the ability to distinguish it from other approaches – was the entry ticket to serious engagement. The science presents an opportunity and a challenge for us at Sionna. The science presents an opportunity and a challenge for us at Sionna. That science resonates with investors.

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The Pharma Data

NOVEMBER 15, 2020

In a deal set to be finalized early in 2021, Urovant Sciences has agreed to be fully acquired by largest investor and close partner, Sumitovant Biopharma. The company hopes to follow vibegron to the market with URO-902, a novel gene therapy for patients whose OAB has eluded oral pharmacologic therapy.

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KIF1A

JANUARY 27, 2024

Rare Roundup KRIBB develops new gene therapy candidate for hereditary spastic paraplegia This week we’re flipping the format and starting with our Rare Roundup, after families contacted us with this article about a gene therapy candidate for hereditary spastic paraplegia.

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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. The development of imatinib to treat chronic myeloid leukemia (CML) serves as a hallmark for targeted therapies.

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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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The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS to Gilead Sciences K.K., the Japan subsidiary of Gilead Sciences, Inc. .–(BUSINESS to Gilead Sciences K.K., the Japan subsidiary of Gilead Sciences, Inc.

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The Pharma Data

MARCH 30, 2021

(NASDAQ:AMGN) and Rodeo Therapeutics Corporation (Rodeo) today announced an agreement under which Amgen will acquire Rodeo, a privately held biopharmaceutical company based in Seattle that develops small-molecule therapies designed to promote regeneration and repair of multiple tissues. About Rodeo Therapeutics.

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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. Noria was founded by Dr. John Babich, Chief, Radiopharmaceutical Sciences in Radiology at Weill Cornell Medicine.

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NIH Director's Blog: Drug Development

OCTOBER 11, 2022

That’s quite a testament to the world-leading science that NIH pursues and its continued impact on improving human health and well-being. Barry Sharpless, Scripps Research, La Jolla, CA, October 5, 2022 marked the second time that he’s received an early-morning congratulatory call from The Royal Swedish Academy of Sciences.

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Olympian Clinical Research

MAY 6, 2024

As medical understanding grew, so did the sophistication of the therapies. These targeted therapies revolutionized treatment, offering relief to severe cases by specifically addressing the underlying immune responses causing the disease.

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Precision for Medicine

JUNE 26, 2023

Baseline Control’s expertise will become part of Project Farma, Precision for Medicine’s existing biomanufacturing strategy and execution group, expanding the scope of its manufacturing solutions for life science companies and driving greater speed and efficiency in bringing new drugs to market. For more information, visit projectfarma.com.

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Dark Matter Blog

JULY 19, 2021

Or should I say back to retirement, as he was comfortably relaxing at home back in 2016 when Jen Petter lured him back into the fray with the siren call of developing a new class of RNA-targeted small-molecule medicines here at Arrakis. I won’t dwell on Jim’s many accomplishments here. Don’t be a stranger!

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The Pharma Data

JULY 15, 2021

This extension will further help connect life-science innovators in Victoria to accelerate their healthcare solutions across the globe,” said Stacy Feld, Regional Head, Johnson & Johnson Innovation LLC, West North America, Australia & New Zealand.

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The Open Targets Blog

SEPTEMBER 21, 2023

I then moved back to the UK to work as a process chemist at GSK, designing the manufacturing routes for small molecule drugs. I knew I wanted to stay close to science, but I wanted a new adventure. The Open Targets role was in a different area of science, at the other end of the drug discovery process.

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KIF1A

MAY 27, 2023

Goodbye with Gratitude: Dr. Dominique Lessard It is with heavy hearts that this week we say goodbye to Dr. Dominique Lessard, Chief Science Officer of KIF1A.ORG. We are humbled to have had such an incredible champion spearheading Team Science, and wish her well-deserved success in her next stage.

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Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

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The Pharma Data

MAY 31, 2022

“More than 10 million people worldwide are affected by Parkinson’s disease, and there is a significant medical need for therapies to slow or stop the progression of this devastating neurodegenerative disease,” said Carole Ho, M.D., BIIB122 is an investigational small molecule inhibitor of LRRK2 that was discovered and developed by Denali.

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The Pharma Data

JANUARY 24, 2021

–( BUSINESS WIRE )– Faze Medicines , a biotechnology company pioneering therapeutics based on the groundbreaking new science of biomolecular condensates, today announced that it has appointed Philip Vickers, Ph.D., 25, 2021 12:00 UTC. CAMBRIDGE, Mass.–( as its chief executive officer (CEO). He earned his B.Sc.

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Dark Matter Blog

JULY 16, 2021

My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I was enticed by Arrakis’ founder Jen Petter to serve in one more operational role, leading the research group at Arrakis as the company launched as one of the first biotechs to target RNA with drug-like small molecules.

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Dark Matter Blog

JULY 19, 2021

This was a great gig – good people, good science. However, in June I happened to attend the Gordon Research Conference on Chemical Biology and High-throughput Chemistry where I saw a session on small molecules and RNA. Making small-molecule drugs against RNA structures looked like a really cool problem – I want to do THAT!

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The Pharma Data

MAY 15, 2023

Gilead Strengthens Early Pipeline in Oncology and Inflammation Through the Acquisition of XinThera Gilead Sciences, Inc. 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.

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The Pharma Data

JUNE 7, 2022

This collaboration combines Alectos’ expertise in small-molecule therapeutics with Biogen’s development capabilities in movement disorders. Currently, there are no approved disease modifying therapies for PD, creating significant unmet needs for patients seeking to slow disease progression.

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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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. We at Teva are excited about collaborating with the MODAG team and look forward to future developments as we continue to follow the science and explore additional indications for both partnered compounds.”.

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The Pharma Data

APRIL 6, 2021

Approvals were based on the overall response rate (ORR) from the open-label, single-arm Phase II CHRONOS-1 (NCT01660451) trial of copanlisib monotherapy in 104 adult patients with follicular B-cell NHL who had relapsed disease following at least two prior systemic therapies. PSMA-TTC is currently being investigated in Phase I studies.

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