Advanced Therapies

SeraGene has developed SG-001, a universal siRNA-based hemostatic agent aimed at treating a wide range of rare bleeding disorders. SG-001 is a first-in-class therapy that offers the convenience of monthly dosing, significantly reducing treatment burden while minimizing the risks of thrombosis and inhibitor development, making it a safer and more effective alternative to current options. Its unique approach of targeting mRNA rather than proteins further enhances its safety profile, distinguishing it from other therapies in the field.

SereNeuro Therapeutics is developing non-opioid cell and gene therapies to address the need for effective and safe treatments for chronic pain. SereNeuro uses mature and purifiable subsets of induced pluripotent stem cell-derived sensory neurons (iPSC-SNs) to decipher the functional heterogeneity of human pain perception and develop targeted therapies. This approach not only aims to provide pain relief but also promotes bone regeneration, offering a disease-modifying solution to chronic pain management.

StradBio is creating the next generation of immunotherapies by using computationally designed, precision-tuned
immune agonists. Systemic toxicity in immunotherapy development has limited the ability to achieve therapeutic doses for immune agonists. The venture’s computationally-designed cytokine mimetics have a broader range of potencies and can be engineered to create logic gates to regulate activation and widen the therapeutic windows, enabling highly targeted immune activation with a high therapeutic index, and making treatments more suitable for outpatient care.

Ernest Pharmaceuticals has engineered a patented
Bacterial Intracellular Delivery vector (BacID) that can produce and deliver biological drugs specifically to solid tumor cells. They have developed two bacterial therapies:
1) EBT-002 with a focus on heterogeneous tumors without biomarkers, such as liver cancer and
2) EBT-305, an immune strategy for ICI-resistant tumors.
Both therapies are in preclinical development, with proof of concept in mice. In addition to their in-house pipeline, they have engineered the BacID platform to easily implement other biological compounds for the development of partnered programs with other drug developers.

EpiCure Therapeutics is building circuit selective adeno-associated virus (AAV) gene therapies and optimized expression systems to
cure epilepsies, with an initial focus on Dravet syndrome. The venture uses its vectors to only target the brain circuit involved in the disease pathogenesis, enabling more precise and effective treatment. The delivery of full SCN1A gene supplementation offers enhanced efficacy and safety, particularly for patients with missense mutations, compared to competitors' upregulation approaches.

Antimmune is developing precision therapeutics for antibody-mediated diseases, beginning with autoimmune blistering skin diseases of which most have high relapse rates to immunosuppressants/steroids and no approved biologics. It differentiates itself through a proprietary platform that rapidly isolates and characterizes disease-causing antibodies and the cells that produce them, enabling the development of novel treatments. This technology offers greater scale, speed, cost-efficiency, and data dimensionality than competitive technologies currently in the market.

Angel City Bio has developed a drug delivery system that conjugates therapeutic small molecules to bile acids, enabling targeted and repeated cycling between the liver and intestine. This orally administered platform is designed to treat Crohn's disease without the use of immunosuppressive agents, offering a safer alternative to existing injectable therapies, particularly for mild-to-moderate cases. Its key differentiator lies in leveraging enterohepatic circulation (the movement of bile acid molecules from the liver to the small intestine and back to the liver) to enhance efficacy while minimizing systemic side effects, addressing a significant unmet need in the current treatment landscape.

Nyxion Therapeutics is developing a bacterial-based therapeutic delivery platform for the treatment of hypoxic diseases. The venture aims to address the challenge of targeted and controlled in vivo delivery through the use of a genetically engineered bacteria designed to durably colonize hypoxic pathologies while secreting a range of therapeutic factors. The key differentiation lies in the platform's ability to produce novel combinations and sequences of factors to effectively mimic real complex physiological events. The result is a scalable, cost-effective, and tissue-specific technology, with unique potential to address critical unmet clinical needs across a range of hypoxic diseases including vascular diseases, oncology, infectious diseases and rare disease.