DrugBaron

FEBRUARY 7, 2022

Finding small molecule drugs is much harder than finding a needle in a haystack – discovering the right arrangement of atoms to bind precisely to a protein target to elicit a particular response is a problem of vast dimensionality. Yet the situation with small molecules is even worse.

Small Molecule

LifeSciVC

JANUARY 16, 2024

Small molecule GLP1s? My take is definitively no, though they will likely increasingly be used in early stages (F0-F2) and as backbones to combination therapy. Whether it’s small molecules to target the incretins (GLP1R ± GIPR), targeting well-validated signaling nodes in I&I (e.g.,

Small Molecule

FDA Law Blog: Biosimilars

JULY 11, 2023

To set the stage for this case, we need to go back to March 2020 , when a new definition of “biological product” threw the world of protein products into a tizzy. As a result of the Biologics Price Competition and Innovation Act (BPCIA) passed in 2010, the definition of a “biological product” expanded to include “proteins.”

FDA

Drug Target Review

JUNE 19, 2024

Because this programme is still in early research, we are not at a point we can say we definitely know what it is going to work best in setting X or setting Y. Transplanting cells could prove to be more beneficial than treating a particular condition with a small molecule or an antibody, and that really speaks to the power of cell therapy.

Therapies

DrugBaron

JANUARY 31, 2024

Consider target selection, which is probably the most critical decision for any drug R&D project (after all, if the target is wrong, then everything that follows will by definition be wasted). Can AI help make better decisions here?

Pharma R&D

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.

Small Molecule

Practical Cheminformatics

JANUARY 8, 2024

When given the prompt “Please estimate the binding affinity between the drug Afatinib and target EGFR”, GPT-4 suggested a docking study, which is definitely not a wise choice. These fingerprint bits then map to atom environments or bond paths, which often don’t correspond to widely accepted functional group definitions.

Drugs

FDA Law Blog: Biosimilars

FEBRUARY 26, 2024

Amongst other things, FDA co-opted many of the same definitions for key terms for implementation of the BPCIA. FDA borrowed this definition from 21 C.F.R. FDA replied that its definition of strength including concentration was clear even in 2009. mg) and the concentration (e.g., mg/mL) for liquid parenteral drug products.

FDA

The Pharma Data

SEPTEMBER 8, 2021

Sanofi has entered into a definitive merger agreement with Kadmon Holdings, Inc. It is the first and only approved small molecule therapy that inhibits the Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and fibrotic processes.

FDA Approval

Drug Target Review

AUGUST 10, 2023

Looking ahead: novel therapeutic avenues Dr Lee and her team are already exploring drugs that can inhibit GRP78 expression or activity, with ongoing studies indicating that certain small molecules, like YUM70, might even block GRP78’s nuclear activity. IOS Press; 2007 [cited 2023 Jul 26]. Available from: [link]

Cell Biology

The Pharma Data

JANUARY 17, 2021

As Crooke explained, “With small molecules, if you make any chemical change, you have a new ballgame. We are looking forward to more definitive guidance in the coming months.”. “I’m still involved with the science that we created and continue to advance,” he told BioSpace. That’s not the case with RNA-targeted drugs.

Disease

The Pharma Data

AUGUST 21, 2020

Novartis has agreed to contribute a carefully curated library of about 1300 small molecules with antiviral activity to the CARE project. I guess that’s the true definition of situational leadership. These will be screened against SARS-CoV-2.

Virus

Elrig

JUNE 4, 2024

The focus used to be more on small molecules, but we’re now starting to see antibody and cell therapeutics. All these ‘worlds’ can learn from each other – especially since the small molecule drug discovery process is much more mature than the antibody which is much more mature than the gene and cell therapy space.

Small Molecule

Vial

FEBRUARY 19, 2024

We can link small molecule drugs to a payload that would enter in the cell and kill it. We have the capability of different types of targets, whether small molecules or antibodies. Can you elaborate on how the platform allows your company to tailor therapy? Ahmed Hamby: We also have a very cool modification.

Small Molecule

The Pharma Data

SEPTEMBER 9, 2020

Its broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor T cells, bi-specific checkpoint immuno-modulators, targeted cancer antibodies and small molecules.

Vaccine

The Pharma Data

OCTOBER 28, 2020

Ridgeline develops small molecule inhibitors of nicotinamide N-methyltransferase (NNMT) to reverse Type 2 diabetes, obesity, muscular dystrophies and sarcopenia (age-related muscle degeneration). With the commanding presence of the MD Anderson Cancer Center , the largest cancer center in the U.S.,

Science

The Pharma Data

DECEMBER 12, 2020

Alexion ) have entered into a definitive agreement for AstraZeneca to acquire Alexion. AstraZeneca has developed a broad range of technologies, initially focused on small molecules and biologics and with a growing focus in precision medicine, genomics, oligonucleotides and epigenetics. This press release features multimedia.

Regulations

Agency IQ

JULY 26, 2024

in equipment) for products that are more complex or aren’t as well-characterized, such as antibody drug conjugates or recombinant DNA products, will be considered higher risk than they would if the change was made to the production of standard, small molecule products. Any CMC changes (e.g.,

Biosimilars

Agency IQ

JUNE 9, 2023

However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs. It also contains a definition of the term immunotoxicity— the unintended immunosuppression or stimulation (including hypersensitivity) —which was notably absent from the draft.

FDA

Drug Target Review

JULY 7, 2023

It normally binds to and recycles internalised immunoglobin G (IgG) antibodies, which are important for fighting infections, as well as albumin, which helps move many small molecules through the blood. Updated assessment of the prevalence, spectrum and case definition of autoimmune disease. Scientific American. EFPIA Homepage.

Disease

KIF1A

AUGUST 20, 2024

This study is not a definitive answer; it’s a snapshot, the first 300 days of a journey that Susannah is still pioneering today. In research we rely on operational definitions – highly specific, limited definitions that allow us to make testable hypotheses. What Symptoms were impacted?

Science

The Pharma Data

JANUARY 7, 2021

ONC201 is an orally administered small molecule dopamine receptor D2 (DRD2) antagonist and caseinolytic protease (ClpP) agonist in late-stage clinical development for recurrent gliomas that harbor the H3 K27M mutation. Cooley LLP served as legal advisor to Chimerix. Oncoceutics, Inc.

Clinical Trials

Agency IQ

OCTOBER 6, 2023

While the definition of NME has changed over the years, it can sometimes include a combination product consisting of at least one drug that has previously been approved. For example, these products face a much more arduous path for development of biosimilar or interchangeable products than that of generics for small molecules.

FDA

Codon

MARCH 19, 2024

While it may seem presumptuous to say that aging has a single major driver even at the single-cell level, this is not the first time the field has been presented with a seemingly definitive theory of the phenomenon it studies. John Gurdon (left) and Shinya Yamanaka (right). Credit: PA Images / Alamy.

DNA

Agency IQ

DECEMBER 11, 2023

In 2015, the FDA, along with the American Association for Cancer Research (AACR), held a workshop on dose optimization for small molecules. Just a few months ago, OCE Associate Director HARPREET SINGH noted , “I know definitely for older adults… there’s not enough work done.”

FDA

The Pharma Data

APRIL 27, 2021

Changes in net sales are expressed at constant exchange rates (CER) unless otherwise indicated (definition in Appendix 7) (1) In order to facilitate an understanding of operational performance, Sanofi comments on the business net income statement. Business net income is a non-GAAP financial measure (definition in Appendix 7).

Vaccine

Agency IQ

NOVEMBER 3, 2023

Unfortunately, there is no regulatory body that governs the terminology used in this space, although there are definitions that are generally accepted by the scientific community. Postbiotic: “Preparation of inanimate micro-organisms and/or their components that confers a health benefit on the host,” according to the ISAPP definition.

Therapies

Codon

APRIL 8, 2025

Enzymes are, by definition, functional proteins that impose their will through changes in structure. Similarly, research into prion protein-ligand interactions has revealed how small molecules can stabilize particular conformational states, potentially informing broader strategies for controlling protein shape.

RNA

Codon

APRIL 28, 2025

The prevailing hypothesis at the time was that Aequorea jellyfish produced light through luciferin, a small molecule sensitive to oxygen and moisture. In the 1960s, many scientists suspected that calcium ions played a critical role in living organisms, but they lacked definitive evidence. Shimomura agreed.

DNA