Search results for Small Molecule Therapy

Small Molecule Therapy: The Breakthrough That Could Transform How We Treat Disease

There are moments in medicine where something shifts. Not gradually. Not subtly. But […]

With $97M, Latus pursues a different kind of Huntington’s gene therapy

The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.

Madrigal Pharma Adds Another Genetic Medicine to Its Drug Combo Strategy in MASH

Madrigal Pharmaceuticals has licensed rights to an Arrowhead Pharmaceuticals small-interfering RNA therapy designed to address a particular genetic mutation associated with the fat...

Small Molecule Drug Candidate Completely Restores Survival in Lethal Mouse Model of Rare Kidney Stone Disease with No Ex...

A groundbreaking discovery from the Buck Institute for Research on Aging may soon rewrite the medical landscape for patients suffering from Primary Hyperoxaluria Type 2 (PH2), an a...

High-Throughput Platform for Fast-Acting Covalent Protein Therapies

Using this new platform, researchers developed a covalent antagonist targeting PD-L1, termed IB101, which was found to form a defined binding pocket that precisely positions the ac...

AI Designs Miniprotein Switches for GPCR Targeting

New AI‑designed miniproteins precisely modulate GPCR signaling and reveal a new "receptor diversion" microscopy-based screening system for targeting receptors long considered diffi...

Relay drug shows early promise against rare blood vessel diseases

While early, the findings suggest the therapy could be superior to Novartis’ Vijoice at treating “vascular anomalies,” a cluster of chronic conditions with few available treatments...

Transforming neurodegenerative disease treatment

Discover how Cognition Therapeutics is redefining hope for Alzheimer’s and Dementia with Lewy bodies [JG1] with innovative, accessible therapies.

A Cancer Driven by Ultra-Rare Mutation Gets Its First FDA-Approved Therapy

Partner Therapeutics’ Bizengri is now FDA approved for treating advanced cases of cholangiocarcinoma driven by NRG1 gene fusions. Partner acquired U.S. rights to this bispecific an...

Prostate Cancer Therapy Targets Disordered Region of Androgen Receptor

A new study designs drugs that bind to intrinsically disordered proteins, which play a central role in cancer, neurodegenerative disorders, heart disease, and autoimmune conditions...

Drug Target for Fragile X Syndrome Identified Through Preclinical Study

Researchers identified the synaptic protein EPAC2 as a potential therapeutic target for fragile X syndrome, and showed that blocking EPAC2 in an FXS mouse model restored abnormal p...

Blackstone invests $250M in Anagram to reduce burden of cystic fibrosis complication

For people living with exocrine pancreatic insufficiency due to cystic fibrosis, the only treatment option is a regimen that can include up to 40 pills a day. With a $250 million i...

Self-assembling peptide helps liver cancer drugs escape lysosome traps

A self-assembling peptide disrupts liver cancer lysosomes, helping lenvatinib and other therapies work better in mouse models without reformulating the drugs.

Lab-made small molecule may help slow brain disease progression

A small, lab-made molecule called SK-129 reduced the formation of toxic clumps of the alpha-synuclein protein, a hallmark of Parkinson’s disease, a study found. SK-129 was able to...

Modular design of dual-targeted, enzyme-responsive peptide carriers for gene drug delivery

Nanoscale Horiz., 2026, Accepted ManuscriptDOI: 10.1039/D5NH00803D, CommunicationXinhao Jiao, Mingshan Han, Zixuan Wang, Jie Min, Rongxin Su, Hang Tie, Wei Qi, Yuefei WangGene-base...

FDA grants breakthrough designation for efimosfermin for MASH

The FDA granted breakthrough therapy designation to efimosfermin — an investigational, once-monthly fibroblast growth factor 21 analogue — for treatment of patients with metabolic...

Oral Small-Molecule GLP-1s Linked to Deep Brain Activity and Reduced Cravings in Mice

New findings in mice suggest that oral small-molecule GLP-1 drugs may directly influence deep brain pathways involved in desire, revealing how these therapies reduce cravings and f...

Neurocrine Is Making a $2.9 Billion Bet on a New Kind of Drug

The company is acquiring Soleno Therapeutics to add a newly approved Prader‑Willi syndrome treatment to its portfolio.

Developing a Drug To Reverse Heart Disease

We have spoken with Reason from Repair Biotechnologies about his company’s lead candidate, REP-0004, a drug targeting the liver to reduce excess intracellular free cholesterol. The...

Breakthrough Parkinson’s Drug Targets Disease at Its Genetic Roots

An experimental gene-silencing therapy safely lowered LRRK2 protein levels in an early Parkinson’s trial, but its effect on symptoms remains untested. An experimental medicine buil...

Neurocrine’s $2.9B Soleno Buyout Brings What Could Become Its Next Blockbuster Drug

Soleno Therapeutics brings Neurocrine Biosciences Vykat XR, a drug commercialized for treating the rare genetic disease Prader-Willi syndrome. This blockbuster prospect complements...

<![CDATA[Beyond the Molecule: The New Competitive Battlefield in Oncology]]>

A protective gene sent to the liver cleared amyloid from mouse brains, sparking hope for an Alzheimer’s gene therapy

The calling card of Alzheimer’s disease is a buildup of misfolded amyloid protein in the brain, but many medicines struggle to penetrate the barrier protecting the mind from outsid...

Skape Bio Unlocks Generalizable GPCR Drugs Using AI Protein Design

David Baker’s latest company is making medicines for a huge protein family once deemed undruggable. A new study generated miniproteins that target GPCRs across a diversity of recep...