ADARx Pharmaceuticals develops a modality-agnostic RNA targeting platform that utilizes base editing of mRNA transcripts to create therapeutics for genetic, cardiometabolic, and central nervous system diseases. The company aims to provide effective treatments for intractable diseases by leveraging oligonucleotides for inhibition, degradation, and editing, along with advanced delivery technologies.

Adcendo is developing a first‑in‑class antibody‑drug conjugate (ADC) that targets the uPARAP receptor, which is overexpressed on mesenchymal cancer cells such as soft‑tissue sarcoma. The ADC links a humanized anti‑uPARAP antibody to a microtubule‑disrupting payload via a cleavable linker, enabling tumor‑specific delivery and reduced systemic toxicity. ADCE‑D01 is in a Phase I/II trial with a companion diagnostic to select uPARAP‑positive patients.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies using its proprietary Affinia Rationally-designed Therapies (ART) platform, which employs AI and structural modeling to enhance targeting and efficacy. The company focuses on providing transformative treatments for patients with severe diseases affecting the heart and nervous system.

Aktis Oncology develops precision radiopharmaceuticals that utilize targeted alpha-emitting particles to treat solid tumors in cancers with limited treatment options. Their technology aims to enhance the efficacy of cancer therapies by delivering localized radiation directly to cancer cells, minimizing damage to surrounding healthy tissue.

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.

Develops proprietary genomic editors that target the root causes of genetic diseases by selecting optimal editing tools based on disease-specific pathology. This approach enables the discovery and development of potentially curative genomic medicines, with a focus on liver and central nervous system disorders.

The startup develops targeted cancer therapies that exploit DNA damage response pathways to induce synthetic lethality in cancer cells. By focusing on specific mutations, their treatments aim to enhance the selective destruction of malignant cells while minimizing harm to healthy tissue.

Artios Pharma develops oral small‑molecule inhibitors that target DNA damage response pathways in solid tumors, using its DcoDeR platform to align drugs with biomarker‑defined patient groups. Its lead ATR inhibitor alnodesertib, which has FDA Fast Track status for ATM‑deficient colorectal cancer, is intended to boost low‑dose chemotherapy, while the Polθ inhibitor ART6043 blocks micro‑homology‑mediated end joining and is being evaluated alone and with PARP inhibitors. The company provides combination‑ready, first‑in‑class DDR agents for oncology clinicians and pharmaceutical partners.

Beacon Therapeutics develops AAV gene therapy to treat blinding retinal diseases, including X-Linked Retinitis Pigmentosa, by introducing functional copies of mutated genes into patients' cells. This approach aims to restore vision and improve the quality of life for individuals affected by these conditions.

Candid Therapeutics develops targeted therapies for autoimmune diseases, focusing on novel mechanisms of action. Their pipeline includes candidates designed to modulate immune pathways and improve patient outcomes. The company aims to address unmet clinical needs with scientifically validated drug candidates.

Carmot Therapeutics develops next‑generation GLP‑1 and dual GLP‑1/GIP receptor agonists that use biased signaling to minimize β‑arrestin recruitment, reducing receptor desensitization and extending drug activity. Its pipeline includes a once‑weekly injectable (CT‑388), a Phase 2 GLP‑1/GIP candidate for overweight/obesity with type 1 diabetes (CT‑868), and an oral daily GLP‑1 small‑molecule (CT‑996), all aimed at delivering stronger glycemic control, greater weight loss, and improved tolerability for metabolic disease patients.

CG Oncology develops Cretostimogene, an intravesically delivered oncolytic immunotherapy for non‑muscle invasive bladder cancer (NMIBC). The therapy uses a genetically engineered virus to directly lyse tumor cells and stimulate a systemic immune response, aiming to reduce recurrence and preserve the bladder. It is being evaluated in a comprehensive clinical program for safety and efficacy, targeting urologists, oncologists, and healthcare systems seeking bladder‑sparing treatments.

Eikon Therapeutics develops a proprietary single-molecule tracking platform that utilizes super-resolution microscopy to measure protein dynamics in living cells, enabling detailed analysis of molecular interactions. This technology addresses the challenge of understanding protein behavior, which is critical for identifying new drug targets and advancing therapeutic development for serious diseases.

Entrada Therapeutics develops intracellular therapeutics using its proprietary EEV™ Platform to target previously inaccessible disease-causing mechanisms within cells. The company focuses on treating devastating diseases by creating potential therapies that span from discovery to clinical stages across various therapeutic areas.

Mineralys Therapeutics is developing targeted therapies for hypertension and related cardio-renal-metabolic diseases driven by dysregulated aldosterone. Their approach focuses on improving patient outcomes by addressing the underlying mechanisms of aldosterone's role in these conditions.

The startup develops controllable cell and gene therapies that utilize pharmacologic operating systems to regulate protein activity in cells. This technology enables physicians to enhance the efficacy of adoptive immunotherapy for cancer patients by using safe, orally active drugs to exert precise control over cell functions.

Pathalys Pharma develops upacicalcet, a novel intravenous calcimimetic designed to treat secondary hyperparathyroidism in patients with late-stage chronic kidney disease. This therapeutic addresses the critical issue of elevated parathyroid hormone levels resulting from kidney failure, which can lead to severe complications in affected patients.

Pheon Therapeutics specializes in developing Antibody-Drug Conjugates (ADCs) to target and treat difficult-to-manage cancers. Their ADCs enhance the precision of cancer therapy by delivering cytotoxic agents directly to tumor cells, minimizing damage to healthy tissue.

Plexium develops a targeted protein degradation platform utilizing proprietary ultra-high throughput cell-based screening technology to create monovalent targeted protein degrader therapeutics. The company focuses on discovering new therapies for cancer and neurological conditions by rationally designing protein degraders that selectively eliminate disease-causing proteins.

RayzeBio develops targeted radiopharmaceutical drugs that utilize radioisotopes to deliver treatment directly to solid tumors, including metastatic cancer. By focusing on precise drug delivery, the company aims to improve survival rates for cancer patients through its robust pipeline of drug candidates in various stages of clinical development.

Synthekine is developing selective cytokine therapies using cytokine partial agonists, orthogonal cytokine and cell therapies, and surrogate cytokine agonists to enhance treatment efficacy for cancer and autoimmune diseases. The company addresses the limitations of current cytokine therapeutics by engineering solutions that minimize pleiotropy and improve therapeutic specificity.

Tourmaline Bio is developing targeted biologic therapies to treat immune and inflammatory diseases, focusing on establishing new standards of care for conditions with significant unmet medical needs. Their approach aims to improve patient outcomes by addressing the underlying mechanisms of these diseases through innovative therapeutic modalities.

Upstream Bio is developing verekitug, a clinical-stage monoclonal antibody that targets an upstream driver of inflammatory response to treat severe asthma. This therapy aims to provide improved treatment options for patients suffering from this chronic respiratory condition.

Versanis Bio develops treatments targeting activin type II receptors to regulate fat and muscle metabolism, addressing obesity and cardiometabolic diseases. By blocking activin signaling, the company aims to reduce visceral fat accumulation and promote muscle growth, improving body composition and metabolic health for individuals affected by these conditions.