AAVantgarde Bio develops gene therapies utilizing proprietary Adeno-Associated Viral (AAV) vector technology to enable the delivery of large genes for the treatment of inherited retinal disorders. The company addresses the limitations of traditional AAV vectors, which restrict the payload size, thereby expanding the potential for genetic medicine in underserved patient populations.

AAVantgarde Bio has developed an engineered adeno‑associated virus (AAV) platform that can package and deliver transgenes larger than 5 kb to photoreceptor and retinal pigment epithelium cells, overcoming the size limitation of standard AAV vectors. The system provides high retinal transduction efficiency, sustained expression, and a GMP‑compatible manufacturing process, supporting clinical programs for Stargardt disease (ABCA4) and Usher 1B syndrome (MYO7A).

Develops small molecule therapeutics that inhibit RNA-modifying proteins (RMPs) to target critical intracellular dependencies in multiple cancer types. This approach addresses the limitations of mutation-specific treatments by offering first-in-class and best-in-class candidates for patients with significant unmet medical needs.

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.

Akero Therapeutics is developing Efruxifermin (EFX), a therapeutic agent aimed at treating metabolic dysfunction-associated steatohepatitis (MASH) by restoring metabolic balance in patients. The company conducts clinical trials to evaluate the efficacy of EFX in reversing the progression of this serious liver disease.

Alnylam develops RNA interference medicines that silence disease‑causing genes using chemically modified siRNAs delivered via proprietary platforms such as GalNAc conjugates for liver targeting and lipid nanoparticles for systemic use. Its portfolio includes FDA‑approved treatments for rare genetic and protein‑misfolding disorders and a pipeline of additional indications, with revenue from product sales, technology licensing, and patient‑support services.

AntaresRX develops precision medicines by integrating predictive sciences and advanced medicinal chemistry to address previously undruggable targets. The company focuses on creating first-in-class allosteric inhibitors for validated targets, accelerating the discovery of novel small molecule therapeutics for serious diseases.

The startup develops neurodegenerative medicines that utilize insights into progranulin and lysosomal biology to enhance cellular health in the brain. Their therapies aim to delay the progression of neurodegenerative diseases and the onset of associated symptoms.

Be Biopharma is developing engineered B cell therapies to treat serious diseases, focusing on conditions like hemophilia B. Their approach utilizes protein-based treatments to enhance patient outcomes and improve quality of life for those affected.

Cogent Biosciences develops bezuclastinib, an oral, highly selective KIT inhibitor that targets activation‑loop mutations such as KIT D816V while sparing off‑target kinases. The drug is being evaluated in registration‑directed trials for systemic mastocytosis and imatinib‑resistant gastrointestinal stromal tumors, offering mutation‑specific therapy with reduced class‑related toxicities. Its design also supports combination with sunitinib to address resistance.

Develops an investigational siRNA-based therapy targeting the root cause of preterm preeclampsia, a life-threatening pregnancy complication. This approach aims to provide safe, evidence-based, and affordable treatment options, improving maternal and neonatal health outcomes globally.

Day One Biopharmaceuticals develops targeted therapies for pediatric patients with relapsed or refractory low-grade glioma, utilizing precision medicine approaches to enhance treatment efficacy. The company addresses the critical need for effective cancer treatments in children, aiming to improve survival rates and quality of life for young patients.

Diagonal Therapeutics utilizes a proprietary platform that combines computational and experimental techniques to rapidly identify and produce agonist antibodies that activate specific receptor complexes. This approach addresses the challenge of developing therapies for debilitating diseases by reactivating critical biological pathways, offering potential treatments for conditions like hereditary hemorrhagic telangiectasia and pulmonary arterial hypertension.

Disc Medicine develops targeted therapies for hematologic diseases and rare blood disorders using advanced biological approaches. The company focuses on creating treatments that aim to save lives and improve the quality of life for patients suffering from these conditions.

Dyne Therapeutics develops nucleic acid therapies using its proprietary FORCE™ platform to enhance muscle tissue delivery for patients with genetically driven muscle diseases. The company focuses on creating targeted treatments that aim to halt or reverse the progression of serious muscle disorders.

Exelixis discovers and develops next‑generation oncology therapeutics using both in‑house small‑molecule platforms and biotherapeutic modalities such as bispecific antibodies and antibody‑drug conjugates. By targeting diverse molecular mechanisms and partnering with pharmaceutical companies, it advances a pipeline of novel cancer medicines from preclinical research through clinical trials to expand treatment options for advanced and resistant tumors.

F‑Star provides a proprietary antibody engineering platform that creates tetravalent, bispecific and trispecific IgG‑like biologics for oncology and immuno‑oncology applications. The technology enables simultaneous engagement of multiple tumor antigens with native IgG pharmacokinetics, Fc‑engineered half‑life extension, and scalable cGMP manufacturing, delivering higher potency and reduced resistance risk for pharma and biotech partners.

Generation Bio provides a cell‑targeted lipid nanoparticle (ctLNP) platform that delivers siRNA specifically to T cells in vivo, enabling sequence‑specific knockdown of disease‑relevant genes. The stealth formulation minimizes off‑target organ uptake and supports repeat dosing with predictable exposure, offering a modular solution for pharmaceutical and biotech developers of T‑cell‑driven autoimmune therapies.

GRO Biosciences has developed a synthetic biology platform that utilizes non-standard amino acids (NSAAs) to enhance protein therapeutics, improving their potency, stability, and immunogenicity. The platform specifically targets the limitations of traditional protein therapies, enabling more effective treatments for autoimmune diseases.

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.

IFM Therapeutics develops first-in-class small molecules that precisely target the innate immune system to regulate immune responses in patients with inflammation-driven diseases and cancer. Their approach addresses the need for effective biological treatments by advancing novel drug candidates from discovery to clinical trials.

Ikena Oncology develops targeted therapies that focus on the Hippo and RAS signaling pathways to address cancer growth and resistance. Their biomarker-driven approach aims to provide effective treatment options for specific patient populations with high unmet medical needs.

Intellia Therapeutics develops in vivo CRISPR‑Cas9 gene‑editing medicines that directly correct disease‑causing mutations in patients with severe monogenic disorders. Its platform integrates guide‑RNA design, lipid nanoparticle/AAV delivery, safety screening, and GMP manufacturing to advance candidates from target discovery through clinical trials, aiming for single‑dose curative treatments.

This company develops and provides innovative medicines and therapies across areas like Oncology, Immunology, Neuroscience, and Cardiopulmonary. It also focuses on medical devices and technology for Cardiovascular, Orthopaedics, Surgery, and Vision applications. The organization applies rigorous science to address complex diseases and advance personalized treatment options in healthcare.