Covalent Modifiers

FEBRUARY 6, 2024

The most common adverse effects are hyperphosphatemia caused by FGFR1 inhibition and diarrhea due to FGFR4 inhibition, as current therapies are not selective among the FGFRs.

Small Molecule 89

Small Molecule Treatment Science Therapies 89

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,

Small Molecule 52

Small Molecule Disease Immune Response Licensing 52

The Pharma Data

JANUARY 19, 2021

Precision medicine developer Kura Oncology is positioned to make a meaningful difference in the treatment of cancer in the next few years with its two, wholly-owned, small molecule compounds. As with any innovative therapy, “It takes multiple iterations and time to get things right,” he pointed out. “We Photo courtesy of Kura.

Small Molecule 52

Small Molecule Therapies FDA Approval Trials 52

Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. Using gene therapy, their approach was beautifully straightforward – “Missing an important gene? But we have not been the only people on this mission. We will replace it.”

Small Molecule 52

Small Molecule RNA Clinical Trials Drugs 52

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.

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

Therapies 105

Therapies Research Small Molecule Clinical Trials 105

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?

Therapies 98

Therapies Small Molecule Production Disease 98

Science Daily: Pharmacology News

NOVEMBER 30, 2023

Researchers have succeeded in restoring lost brain function in mouse models of stroke using small molecules that in the future could potentially be developed into a stroke recovery therapy.

Small Molecule 127

Small Molecule Therapies Research 127

DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed.

Small Molecule 83

Small Molecule Drugs Clinical Trials Therapies 83

Covalent Modifiers

AUGUST 14, 2024

Rullo Molecular Therapy 2024 DOI: [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova,Joanne A. Hammill,Jonathan L. Bramson, Anthony F.

Small Molecule 71

Small Molecule Engineering Science Therapies 71

The Premier Consulting Blog

NOVEMBER 20, 2024

For drugs with FDA Orphan Drug Designation (ODD), Breakthrough Therapy Designation (BTD), and EMA PRIority MEdicines designation (PRIME), all clinical, non-clinical, and CMC requirements are still required for regulatory approval, despite being on pathways for an expedited approval process.

Small Molecule 52

Small Molecule Drugs FDA Production 52

ASPET

SEPTEMBER 7, 2023

This recently approved enzyme replacement therapy (ERT) was glycoengineered to maximize CIMPR binding through high-affinity interactions with ~7 bis-M6P moieties. Avalglucosidase alfa has received marketing authorization in several countries for infantile-onset and/or late-onset Pompe disease.

Small Molecule 100

Small Molecule Therapies Disease Treatment 100

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

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.

Small Molecule 52

Small Molecule Disease Therapies Clinical Trials 52

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.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

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.

Therapies 52

Therapies Clinical Trials Small Molecule Immune Response 52

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. The cell or gene therapy should be pharmacologically active in the animal model. Note that there is no strict requirement of toxicity studies in two species, which is often needed for small molecule programs.

Therapies 52

Therapies Small Molecule Production Clinical Trials 52

Covalent Modifiers

JUNE 24, 2024

Rullo, Molecular Therapy: Oncology , 2024 , 200842, [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova, Joanne A. Hammill, Jonathan L. Bramson, Anthony F.

Small Molecule 52

Small Molecule Engineering Science Therapies 52

Covalent Modifiers

JANUARY 29, 2024

3c01835 Building on recent advances in peptide science, medicinal chemists have developed a hybrid class of bioconjugates, called peptide–drug conjugates, that demonstrate improved efficacy compared to peptides and small molecules independently. Moore Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.3c01835

Small Molecule 68

Small Molecule Drugs Science Disease 68

Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

Vaccine 52

Vaccine Therapies Clinical Trials Small Molecule 52

DrugBank

MAY 15, 2024

One approach is to look beyond the traditional drug molecule. Researchers are experimenting with biologics—larger biological molecules that can do things small molecules can't, like targeting larger, more complex structures on cell surfaces or even inside cells.

Small Molecule 82

Small Molecule Molecular Biology Drugs Disease 82

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.

RNA 96

RNA Small Molecule Disease Assay Development 96

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. We look forward to working on this new collaboration with Biohaven, a world leader in the clinical development of CGRP-targeted therapies.” TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,

Small Molecule 40

Small Molecule Licensing Licensing Treatment 40

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.

Small Molecule 128

Small Molecule Clinical Trials Engineering Trials 128

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?

Small Molecule 111

Small Molecule Engineering Protein Expression Therapies 111

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 –(BUSINESS WIRE)– Kite Pharma, Inc., to Gilead Sciences K.K., the Japan subsidiary of Gilead Sciences, Inc. Kite’s manufacturing facility in El Segundo, California, U.S.

Therapies 40

Therapies Marketing Small Molecule Science 40

Vial

DECEMBER 11, 2023

In the decades since its identification in 1989, advances in clinical research for new Hepatitis C therapies and the introduction of direct-acting antiviral agents (DAAs) have led to viral eradication in more than 98% of patients, resulting in HCV crossing from manageable to being the first curable , chronic viral infection.

Clinical Research 52

Clinical Research Therapies Research Vaccine 52

The Pharma Data

APRIL 22, 2023

While no cure is available for MS, existing disease-modifying therapies in the form of small molecule and protein drugs either directly target the self-reactive immune cells or broadly dampen inflammation. “Current MS therapies do not specifically target myeloid cells.

Therapies 40

Therapies Immune Response Small Molecule Engineering 40

The Pharma Data

OCTOBER 15, 2020

“Manufacturing cells is not like manufacturing small molecules,” Brian Culley, CEO of Lineage Cell Therapeutics , told BioSpace. For cell therapy products to mature into ‘real’ products that deliver on the promises of 10 years ago, they must be scalable – which drives affordability – and they must solve their purity issues.”.

Therapies 52

Therapies Small Molecule FDA FDA Approval 52

Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise. Autophagy boosters, in contrast, are conventional small molecule drugs.

Disease 113

Disease Treatment Small Molecule Therapies 113

Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

RNA 133

RNA Vaccine Small Molecule Therapies 133

New Drug Approvals

SEPTEMBER 23, 2024

There are no current small molecule therapies targeting the underlying cause of the disease, leaving a high unmet need for medications that can be used for treating or ameliorating HD. Consequently, there remains a need to identify and provide small molecule compounds for treating or ameliorating HD.

Small Molecule 57

Small Molecule RNA DNA Disease 57

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.

Drug Development 71

Drug Development Metabolic Stability Clinical Trials Drugs 71

Alta Sciences

SEPTEMBER 27, 2024

The growing understanding of the immune system's role in various diseases, along with advancements in drug development, have led to a natural evolution from traditional vaccines, small molecules , and cytokines , to increased targeted biologics and innovative approaches in gene therapy and vaccines.

Clinical Trials 52

Clinical Trials Trials Pharmacokinetics Small Molecule 52

PPD

NOVEMBER 11, 2024

As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8

Drug Development 95

Drug Development Clinical Trials Drugs Small Molecule 95

thought leadership

SEPTEMBER 29, 2023

Government begins its fiscal year on October 1, it signifies the annual revisions in FDA User Fees, which have an impact on applications and facilities associated with Prescription Drugs (including small molecules, vaccines, cell and gene therapy products, biologics, etc.), Generic Drugs, Biosimilars, and Medical Devices.

FDA 45

FDA Small Molecule Biosimilars Government 45

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).

Small Molecule 111

Small Molecule Therapies Treatment Disease 111