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.

The startup develops RNA-only therapeutics that integrate genetic elements into safe harbor sites, enabling precise RNA-mediated insertion of transgenes. This approach addresses the need for targeted genetic medicine, offering patients a method for accurate and effective treatment options.

Akamis Bio develops T‑SIGn® therapeutics, systemically administered virus‑based oncolytic immunotherapies that selectively replicate in colorectal tumor cells and deliver immune‑modulating payloads directly to the tumor microenvironment. By combining direct oncolysis with intratumoral production of antibodies, cytokines or bispecific proteins, these agents aim to enhance antitumor immunity and reduce the need for invasive surgery in locally advanced and metastatic colorectal cancer.

Alumis develops oral therapies using a precision medicine approach to target immune-mediated diseases, focusing on the underlying drivers of immune dysfunction through genetic and translational insights. The company aims to improve clinical outcomes for patients suffering from autoimmune disorders by optimizing therapeutic interventions.

AM Pharma is developing ilofotase alfa, a proprietary recombinant human alkaline phosphatase, as an enzyme replacement therapy for adult hypophosphatasia (HPP). The subcutaneous treatment restores alkaline phosphatase activity, lowering disease‑related pyridoxal‑5′‑phosphate and inorganic pyrophosphate levels, which improves bone mineralization, muscle function, and pain. Phase 1b data show dose‑dependent normalization of HPP biomarkers and a favorable safety profile, positioning ilofotase alfa for Phase 2 trials to address the unmet need in adult HPP patients.

The startup develops antibody biotherapeutics targeting B cell cancers, inflammatory and metabolic disorders, and chronic hepatitis B infections. By providing clinical development support, the company enhances treatment options for patients facing these life-threatening diseases.

Antag Therapeutics is developing first‑in‑class GIP receptor antagonist peptides, such as AT‑7687, to treat obesity, type 2 diabetes, and related metabolic disorders. By blocking the GIP pathway that drives fat storage and insulin resistance, their approach aims to achieve meaningful weight loss and metabolic improvement while preserving lean mass and maintaining tolerability. The lead candidate is in Phase 1a trials as both a monotherapy and in combination with other obesity therapies.

Arcellx develops cell therapies utilizing its proprietary D-Domain technology and ddCAR platform to enhance the efficacy of immune cell treatments for cancer. The company targets relapsed or refractory multiple myeloma, aiming to provide patients with durable responses through its innovative CART-ddBCMA therapy.

ARS Pharmaceuticals develops neffy, the first FDA-approved needle-free epinephrine nasal spray, providing a portable and easy-to-use alternative for individuals at risk of severe allergic reactions. This product addresses the challenges of needle-related risks and the anxiety associated with traditional epinephrine delivery methods.

Asceneuron develops oral therapeutics that target tau pathology to treat neurodegenerative disorders, including Alzheimer’s disease and orphan tauopathies. The company addresses the high unmet medical needs in these conditions by blocking the progression of tau-related neurodegeneration.

Avalyn Pharma develops inhaled formulations of anti‑fibrotic drugs, such as pirfenidone (AP01), delivered via a handheld eFlow® nebulizer that targets the lung directly. This lung‑focused delivery enables lower systemic doses, reducing side effects and improving patient adherence while aiming to stabilize lung function in progressive interstitial lung diseases.

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.

Bicycle Therapeutics creates fully synthetic, bi‑cyclic peptide molecules that combine the high affinity of biologics with drug‑like pharmacokinetics, enabling precise targeting of protein‑protein interactions that are difficult for traditional small molecules or antibodies. Their platform delivers rapid tissue penetration, renal clearance, and flexible conjugation to payloads, supporting targeted drug conjugates and immune agonists for solid‑tumor oncology and a range of non‑oncology indications. The company partners with pharma, biotech, and academic groups to develop novel therapeutics for high‑unmet‑need disease targets.

CB Therapeutics utilizes precision fermentation and a proprietary yeast platform to biosynthetically produce cannabinoids, tryptamines, and other high-value compounds for various industries, including neuroscience and food technology. The company addresses the need for sustainable and scalable production of therapeutic and nutritional ingredients, ensuring consistent quality and purity.

CHARM Therapeutics utilizes its proprietary DragonFold technology, which employs 3D deep learning for protein-ligand co-folding, to develop small molecule inhibitors targeting previously undruggable proteins associated with cancer and other diseases. By addressing the challenge of the vast majority of the human proteome remaining undruggable, the company aims to create transformative therapies for patients with high unmet medical needs.

CRISPR Therapeutics develops curative gene‑editing medicines using CRISPR/Cas9 technology, creating both ex vivo cell therapies and in vivo lipid‑nanoparticle delivery approaches. Its pipeline targets serious genetic, hematologic, metabolic, oncologic and rare diseases, aiming to provide one‑time, disease‑modifying treatments for patients.

Crystalys Therapeutics focuses on developing new therapeutics to address significant unmet medical needs for people living with gout. The company is actively advancing its pipeline through clinical development, including ongoing Phase 3 studies. Their work aims to provide improved treatment options for patients managing this condition.

Design Therapeutics is developing small molecule therapeutic candidates through its GeneTAC™ platform to target the underlying causes of serious genetic disorders, such as Friedreich ataxia and Huntington’s disease. The company addresses the urgent medical need for functional cures in monogenic repeat expansion disorders, which currently lack effective treatment options.

Draig Therapeutics develops novel neuromodulators targeting Glutamate and GABA systems to restore synaptic balance in the central nervous system. Their pipeline of small molecules, including AMPA receptor PAMs and GABA A receptor NAMs/PAMs, aims to provide improved efficacy and tolerability for neuropsychiatric disorders like Major Depressive Disorder.

Dren Bio is a clinical‑stage biopharmaceutical company that develops antibody therapeutics designed to selectively deplete pathogenic cells using its proprietary Targeted Myeloid Engager and Phagocytosis Platform. Its pipeline includes two first‑in‑class clinical programs for oncology and autoimmune diseases, plus preclinical candidates across oncology, immunology, and neurology, aiming to improve efficacy while reducing off‑target toxicity.

Econic Biosciences is developing a healthcare platform that targets extrachromosomal DNA (ecDNA), a significant driver of oncogenic gene amplification linked to treatment-resistant cancers. By utilizing artificial intelligence to identify and characterize ecDNA, the company aims to create targeted therapies that improve clinical outcomes for cancer patients.

Elektrofi utilizes its hyper concentration microparticle technology, Hypercon™, to enable subcutaneous self-administration of biologic therapies, specifically targeting monoclonal antibodies and therapeutic proteins. This approach eliminates the need for intravenous delivery, allowing patients to receive their medications conveniently at home, thereby enhancing treatment adherence and patient autonomy.

This company pioneers a new approach to allosteric drug discovery by targeting natural hotspots on proteins to create differentiated medicines. Their proprietary Smart Allostery™ platform integrates structural biology, pharmacology, chemistry, and AI to systematically identify and drug these cellular on/off switches. The resulting novel allosteric medicines are being developed to treat diseases such as cancer and autoimmune disorders.

The startup develops investigational MDM2 inhibitors aimed at treating solid tumors and hematological malignancies. By targeting the MDM2 protein, the company provides a new therapeutic option that may enhance treatment outcomes for cancer patients.