Aclaris Therapeutics leverages its KINect® platform to discover covalent kinase inhibitors and dual‑targeting bispecific antibodies that modulate key inflammatory signaling pathways. It advances oral, topical, and biologic candidates for immuno‑inflammatory conditions such as atopic dermatitis, asthma, COPD, and inflammatory bowel disease, and provides licensing and co‑development partnerships to pharmaceutical and biotechnology companies.

Aeovian Pharmaceuticals is developing AV078, a selective mTORC1 inhibitor designed to treat refractory epilepsy associated with tuberous sclerosis complex (TSC), a rare genetic disorder characterized by hyperactive mTORC1 signaling. The company aims to provide a targeted therapeutic option that improves safety and efficacy compared to existing nonselective mTOR inhibitors, addressing the significant unmet medical need in managing TSC-related seizures.

Aerovate Therapeutics is developing AV-101, a proprietary dry powder inhaled formulation of imatinib, to target the hyperproliferation of cells in the narrowed arteries of patients with pulmonary arterial hypertension (PAH). This approach aims to provide significant health improvements for individuals suffering from this rare and progressive cardiopulmonary disease.

Aiolos Bio is developing therapies that target the underlying mechanisms of asthma and other respiratory diseases by leveraging advanced immunology and machine learning techniques. Their goal is to enable patients to achieve clinical remission, significantly improving quality of life for millions affected by these conditions.

Akouos develops gene‑therapy treatments for inherited hearing loss, using proprietary adeno‑associated virus vectors to deliver functional copies of defective genes directly to inner‑ear cells. Their platform combines precision, minimally invasive delivery with biomarker‑driven patient selection to restore or preserve auditory function, targeting otolaryngologists, audiologists, and pharmaceutical partners.

This biopharmaceutical company develops orally delivered compounds to treat serious eye diseases. Their lead drug candidate targets Stargardt disease and age-related macular degeneration, aiming to provide early intervention for irreversible vision loss.

Altamira Therapeutics provides the xPhore™ platform, a peptide‑based nanoparticle system that enables efficient delivery of siRNA, mRNA, circRNA, and DNA therapeutics to extra‑hepatic tissues for pharma and biotech partners. It also markets Bentrio®, a preservative‑free nasal spray for allergic rhinitis, and develops CNS treatments for inner‑ear and neurological disorders.

This company develops small molecule antiviral drugs targeting infections caused by HPV, herpes viruses, HIV, and animal retroviruses. Their medicinal chemistry platform aims to create direct-acting antivirals, helping patients recover from viral diseases.

Apellis Pharmaceuticals develops first‑in‑class C3‑targeted inhibitors to treat diseases caused by overactivation of the complement system. Its approved drug pegcetacoplan (SYFOVRE®/EMPAVELI®) blocks all three complement pathways and is used for geographic atrophy, paroxysmal nocturnal hemoglobinuria, and complement‑mediated kidney disorders, while its pipeline extends C3 inhibition to additional rare and renal diseases.

Arrivent is a clinical‑stage biopharmaceutical company that identifies, develops, and commercializes differentiated oncology medicines, focusing on hard‑to‑treat cancers. It leverages deep drug‑development and regulatory expertise to advance high‑quality candidates—including next‑generation antibody‑drug conjugates—through in‑licensing, partnerships, and strategic collaborations, aiming to bring effective therapies to global patients.

ARTBIO is developing a new class of alpha radioligand therapies that utilize Pb-212 isotopes to target and destroy cancer cells while minimizing damage to healthy tissues. This approach addresses the limitations of existing beta radioligand therapies by offering potentially improved efficacy and safety for patients with solid tumors.

Ascendis Pharma is a biopharmaceutical company developing novel therapies utilizing its proprietary TransCon technology platform. The company focuses on advancing product candidates in Endocrinology Rare Disease and Oncology through clinical development. This technology aims to improve treatment safety, efficacy, tolerability, and convenience for patients with unmet medical needs.

Atsena Therapeutics is a clinical-stage gene therapy company utilizing adeno-associated virus (AAV) technology to develop treatments for inherited retinal diseases, specifically targeting X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Their ongoing Phase I/II clinical trials aim to reverse or prevent vision loss in patients suffering from these genetic conditions.

Avenzo Therapeutics is developing targeted oncology therapies that utilize novel drug delivery systems to improve the efficacy of cancer treatments. The company focuses on addressing the limitations of current therapies by advancing a pipeline of potentially best-in-class options for patients with serious diseases.

Axonis Therapeutics is developing first-in-class oral KCC2 potentiator drugs, specifically targeting the KCC2 protein to restore functional inhibition in the brain. This approach aims to address neurological disorders such as epilepsy and pain by enhancing inhibitory signaling within the central nervous system.

BioAge is a clinical-stage biotechnology company utilizing a proprietary multi-omics platform that analyzes 65 million molecular measurements from 45 years of human aging to identify novel therapeutic targets for metabolic diseases. Its lead program, azelaprag, is an oral APJ agonist entering Phase 2 trials, designed to enhance weight loss and body composition in older adults undergoing obesity treatment with incretin drugs.

Capstan Therapeutics develops targeted lipid nanoparticles (tLNPs) that deliver mRNA and gene editing tools to specific cell types in vivo, enabling precise reprogramming of cells for therapeutic purposes. This technology addresses the need for effective treatments across various diseases, including oncology and genetic disorders, by facilitating safe and repeatable dosing with tailored payloads.

The startup develops a non-injectable somatostatin analog that provides an alternative to traditional injectable growth hormone inhibitors used in the treatment of acromegaly and neuroendocrine tumors. This therapeutic approach offers patients a non-invasive option for short-acting therapy, enhancing treatment accessibility and comfort.

Expedition Therapeutics is developing EXPD-101, an oral, once‑daily DPP1 inhibitor that selectively blocks neutrophil serine protease activation to reduce airway tissue damage and disease progression in COPD and other neutrophil‑driven respiratory conditions. The drug is designed for high potency, favorable pharmacokinetics, and a strong safety profile, with Phase 1 tolerability data and a global Phase 2 trial underway. It targets pharmaceutical partners, clinical researchers, and healthcare providers seeking disease‑modifying therapies for COPD, bronchiectasis, neutrophilic asthma, and related diseases.

Galera Therapeutics uses a proprietary platform to create novel agents that amplify the efficacy of existing breast cancer drugs, aiming to boost tumor response and enable lower dosages. Their preclinical pipeline focuses on combination candidates that synergize with chemotherapy, hormonal, and targeted therapies, offering pharmaceutical partners and clinicians enhanced treatment options.

InterMune discovers and advances small‑molecule inhibitors and monoclonal antibodies that block key profibrotic pathways such as TGF‑β and PDGF to treat idiopathic pulmonary fibrosis and other lung fibrotic diseases. The company integrates companion‑diagnostic biomarkers for patient stratification and employs adaptive Phase II/III trials to accelerate regulatory approval, generating revenue from product sales and licensing royalties.

IntrepidaBio is a preclinical biotechnology company focused on developing targeted therapies for cancer by utilizing proprietary drug discovery platforms that identify and optimize small molecule compounds. The company addresses the need for more effective and personalized treatment options in oncology, aiming to improve patient outcomes and reduce side effects.

Iterum Therapeutics is a clinical-stage pharmaceutical company focused on developing differentiated anti-infectives to combat multi-drug resistant pathogens. Their proprietary formulations aim to address the urgent public health crisis posed by resistant bacterial infections, enhancing treatment options for healthcare providers.

Kiniksa Pharmaceuticals is developing targeted therapies for inflammatory and autoimmune conditions, utilizing immune-modulating assets to address critical therapeutic needs. The company focuses on providing effective treatment options for patients suffering from debilitating diseases, such as recurrent pericarditis, where timely intervention is essential.