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CNS

Vincere Biosciences

www.vincerebio.com

Vincere develops small molecule therapeutics to improve mitochondrial quality. Natural mechanisms of mitochondrial quality control decline with age, leading to the neurodegeneration seen in Parkinson's disease which affects 10 million patients worldwide. Biology is complex with a rapidly growing body of knowledge. Proprietary simulation and AI from NeuroInitiative enables the Vinere team to move faster and synthesize new discoveries into efficient therapeutic programs.

QurAlis

www.quralis.com

QurAlis is bringing hope to the ALS community by developing breakthrough precision medicines for this devastating disease. Quralis' stem cell technologies generate proprietary human neuronal models that enable them to more effectively discover and develop innovative therapies for genetically validated targets. They are advancing three antisense and small molecule programs addressing sub-forms of the disease that account for the majority of patients.

Evecxia Therapetucs

www.evecxia.com

Evecxia Therapeutics is the first company dedicated to realizing the therapeutic potential of 5-hydroxytryptophan (5-HTP) for the treatment of mental illness. Mental illness negatively impacts all aspects of life – from internal emotional states, to work and scholastic performance, to relationships with family and friends. Evecxia Therapeutics’ mission is to help people suffering from disabling mental illness, and not treated adequately by current therapies, to lead fuller, more meaningful lives.

Ophidion

www.ophidion.com

Ophidion, Inc. is a private biotechnology company with a pipeline of products to treat neurodegenerative diseases using gene silencing cargoes such as siRNAs or ASOs that are complexed to a patented Trojan horse technology that is capable to deliver these various cargoes to the brain following intravenous administration. The patented technology exploits an endogenous receptor-mediated transport system to deliver small molecule drugs and biologics into the brain. This technology can deliver therapeutics for the treatment of various CNS disorders and brain malignancies. Our lead program is targeting Huntington's disease where we deliver our proprietary siRNA or ASO, complexed to our Trojan horse, to the brain following IV administration. This is highly differentiated program relative to other competitors that deliver the cargoes via more invasive methods such as the intrathecal or intracranial routes. We have other pipeline products to treat Cognitive Impairment (e.g. Alzheimer’s disease, Parkinson’s disease). The platform technology is amenable to delivering multiple classes of drugs, including small molecules and macromolecules (gene therapeutics, siRNAs, ASOs, peptides, and cDNAs). Ophidion owns ten issued patents and is prosecuting additional patents.

Ribogenics

www.ribogenicstx.com

Ribogenics is developing small molecule RNA-medicines to treat cancer, neurological disorders and rare diseases. Their platform is based on over 30 years of research in RNA splicing and RNA binding proteins from Dr. Dreyfuss’ lab at UPenn with proof-of-concept from Merck in rare disease. The Ribogenics Platform generates small molecule drug candidates that can correct the function, presence or absence of proteins enabling cells to operate normally with a validated high-throughput phenotypic screening platform that brings compounds from discovery to optimization to build a proprietary pipeline of drug candidates and partner with Pharma.

Cardiovascular & Regenerative Medicine

Acelink Therapeutics

www.acelinktherapeutics.com

AceLink Therapeutics is a clinical-stage biopharma company with a focus on kidney diseases and genetic diseases with unmet clinical needs. Founded in 2018, AceLink is headquartered in San Francisco Bay area and has discovery and clinical development operations in US and Chuina. Our leading drug candidate AL01211 is a GCS inhibitor that offers a novel therapeutic approach for Fabry disease and other kidney diseases. AL01211 has successfully completed Ph1 clinical trial and is expected to enter phase 2 trial in patients with Fabry disease in 2022. The second drug program offers new treatment options for several neuropathic and neurodegenerative diseases. In addition to the above, our drug pipeline combines internal drug discovery, led by experienced drug development scientists, and licensing in external assets. AceLink has finished two rounds of VC fundraising.

Haya Therapeutics

www.hayatx.com

HAYA Therapeutics is a precision therapeutics company that discovers and develops innovative tissue- and cell-selective genomic medicines for fibrotic diseases and other serious health conditions associated with aging, including cancer. The company’s discovery engine focuses on long non-coding RNAs (lncRNAs) within the “dark matter” of the human genome -- key tissue and cell-specific drivers of fibrosis and other disease processes -- to identify novel targets and drug candidates with the potential for greater efficacy and safety than existing treatments.

Riparian Pharmaceuticals

www.riparianpharma.com

In nature, the riparian zone is a unique rich ecosystem created through the interaction of a river with its banks. In the human body, moving blood, or hemodynamics, interacts with the walls of blood vessels generating unique biological activity. This unique flow-sensitive biology is a critical and unexplored determinant of vascular health. Riparian’s therapeutic programs target new vasoprotective pathways to hopefully treat both prevalent and serious rare vascular diseases at an earlier stage than is possible today.

Basking BioSciences

www.baskingbiosciences.com

Clinical stage company developing a novel. reversible RNA therapeutic for acute thrombosis. The paired therapy combines an RNA aptamer targeting VWF and a direct acting reveral oligonucleotide that neutralizes the aptamer's pharmcologic activity in 5 minutes in the event of bleeding. We have completed Phase 1 and will start a Phase 2 study in acute ischemic stroke paitents in late 2022.

Morphogene SA

MorphoGene is an early stage Swiss company currently led by Professor Hubbell of the University of Chicago who has successfully founded and led other pharma startups in the past. MorphoGene specializes in re-engineering growth factors using a high affinity ECM binding domain to aid growth factor adherence to the ECM for better local wound retention and efficacy in the treatment of chronic wounds.

Immunology & Infection

IpiNovyx Bio

www.ipinovyxbio.com

IpiNovyx Bio is a New York-based biotech company, developing immunoproteasome inhibitors for the treatment of autoimmune diseases. Their leading compounds are highly selective and reversible immunoproteasome inhibitors, showed promising in vitro and in vivo efficacy, and a good safety profile. The leadership team is highly experienced industry veterans.

Karma Biotechnologies

karmabiotech.com/

Karma Biotechnologies is developing precision immunotherapies to treat autoimmune diseases.

Dorian Therapeutics

www.doriantherapeutics.com

Dorian Therapeutics is the leading company working on senoblockers, a new class of therapeutics that can rejuvenate cells and tissues. Based on a patented technology developed at Stanford University, Dorian’s team is bringing to the clinic an innovative solution for age-associated diseases. During aging, there is a decrease in the activity of stem cells, and therefore a reduced regenerative capacity. Additionally, senescent cells accumulate in the body and poison entire tissues. Dorian's Senoblockers act on epigenetic regulators to reverse both processes, reactivating programs of youthfulness and regeneration.

Saverna Therapeutics

www.saverna.com

Saverna Therapeutics was founded 2017 with a mission to change the way pharmaceutical companies discover and develop safe, orally active small molecules that target non-coding RNA-biology to treat diseases with unmet medical need. Saverna Therapeutics aims to discover highly active, selective and cell-permeable low molecular weight molecules that target RNA biology.

Arrepath

www.arrepath.com

ArrePath was founded upon the mission to discover new and differentiated classes of anti-infectives to address antimicrobial resistance (AMR) through the application of state of the art technologies and novel strategies. Arrepath's advanced machine learning (ML) and imaging-based drug discovery platform enables a rapid and efficient identification of new drug classes with desired activity profiles and clinical utility, coupled with a deep understanding of mechanism of action at the outset of the discovery process.

Xlement

www.liangzhunglobal.com

Established in 2016, Liangzhun Group focuses on smart biomedical technology. Since its inception, it has been committed to providing a full range of smart medical testing products and solutions for medical institutions and consumers. Over the years, it has cooperated with many top universities and research units in China and the United States. Through independent research and development of professional technology and products, it now has dozens of national and international patents and is continuously launching the most cutting-edge biomedical sensor products to benefit society and many families thus fulfilling their company mission.

Oncology

Deka BioSciences

www.dekabiosciences.com

Deka has developed the next generation of cytokine therapy - Diakine™. Diakines™ are purposely engineered therapeutic proteins that are designed to deliver clinically validated cytokine combinations to diseased tissue.
Deka uses Precision Medicine to identify patients that will benefit from their Diakines™, because not everyone responds to a given cytokine in the same way. They have developed the appropriate assays and discovered the underlying genetic signatures that are responsible for the differential responses to Diakines™.
Deka is focused on the development of novel cytokine therapies to treat cancer and inflammatory diseases such as Crohn’s, psoriasis, rheumatoid arthritis and sepsis.

Focus-X Therapeutics

www.focus-xtx.com

Focus-X Therapeutics, founded in 2020, is a preclinical stage biotechnology company committed to the discovery and development of cutting-edge radiopharmaceuticals. They focus on unmet needs in cancer diagnosis and therapy by applying their theragnostic approach to solid tumors with very low five-year survival rate. They bring proprietary radioligand therapy (RLT) to precisely deliver alpha or beta emitters to breakdown cancer cell DNA. As an emerging leader of RLT, their mission is to release the true potential of such modality by leveraging our world-class peptide drug discovery platform and joining force with top radiopharmaceutical partners.

NAKI Therapeutics

www.nakitherapeutics.com

Naki is developing car-NK therapeutics, based on their own NK cell expansion platform, which will be used for the treatment of solid tumors.

Synthis Therapeutics

www.synthistx.com

Synthis Therapeutics is an early-stage biotech company dedicated to the innovation of targeted therapies that harnesses the power of patients’ own immune system to eliminate metastatic cancer. Their novel therapeutic platform evolved from an idea to develop safer and more efficacious therapies that eliminate one of the most immunosuppressive pathways in virtually all cancers, known as TGF-β. By developing therapies that selectively target the immunosuppressive arm of the TGF-β pathway, they can safely drive efficacy and patient survival.

Phenomic AI

www.phenomic.ai

Phenomic is a pioneer in using advanced machine learning tools for processing imaging, RNA sequencing, and spatial transcriptomics data to understand the biology of single-cells sitting in complex multi-cell systems. This lets Phenomic explore how cells communicate with each other, both in human tissue samples, and disease relevant experimental models.

Exarta Therapeutics

Exarta is a pre-clinical biological start-up company located in New York, USA.

Reglagene

www.reglagene.com

Reglagene is an oncology company focused on discovering and developing novel medicines for difficult to treat and metastatic cancers. Reglagene’s first product is indicated for CNS cancers, including primary and secondary brain cancers. There is a massive unmet medical need in this therapeutic area.
Reglagene’s first product is a small molecule that is therapeutically effective, well tolerated, orally administered, and can freely cross the blood-brain barrier. We aim to reach patients diagnosed with CNS cancers as quickly as possible and utilize the FDA accelerated drug development and approval programs to reduce timelines and cost. Patients will no longer have to choose between palliative care and toxic medicines that barely extend their lives. Our medicine works through microtubule destabilization and is differentiated by being the only microtubule targeting agent that is orally administered, well-tolerated, and persists in the brain at micromolar concentrations for up to 8 hours.
Leveraging our discoveries from our first product, we believe there are other clinical applications that we can pursue, including COVID-19 and inflammatory diseases such as arthritis and coronary artery disease. The success of our first program will enable the Reglagene team to continue the development of our epigenetic medicine platform technology capable of discovering 1-2 new programs per year.

Amberstone Biosciences

www.amberstonebio.com

Amberstone Biosciences discovers and develops next generation immunotherapeutics with a tumor microenvironment-dependent activity switch to achieve improved safety profile and to address broad cancer patient populations. Our pipeline molecules are uniquley discovered in part through a cutting-edge patent-proteced single-cell functional discovery platform technology.

GT Apeiron Therapeutics

www.apeiron-bio.com/

Apeiron is strategically based in Shanghai and in the San Francisco Bay Area, with significant collaborations in the UK enabling us to tap into technologies and talent from all three continents. Our team and partners bring together deep expertise in oncology and drug discovery to enable efficient and scalable discovery and development of novel drugs.
Social media: LinkedIn https://www.linkedin.com/company/gt-apeiron-therapeutics/

F5 Therapeutics

www.f5therapeutics.com

F5 is evolving targeted protein degradation to transform the landscape of drug discovery by utilizing the cell’s normal protein regulation systems to remove disease causing proteins that cannot be targeted by traditional approaches. The NExMods™ Platform (NEosubstrate Expression Modulators) represents a novel approach to “molecular glues” capable of delivering impactful medicines to patients across diverse therapeutic areas: oncology, immuno-oncology, fibrosis, inflammation, and neurodegeneration.

GenHouseBio

www.genhousebio.com

Genhouse is focusing on the development of the world's leading small molecule anti-tumor drugs. The company’s management has many years of experience in corporate management, new drug research and development, and clinical medicine research, and they are committed to breaking through the target of "un-druggable drugs" and focusing on original innovation.

Sisu Pharma

www.sisupharma.com

Sisu Pharma is a preclinical-stage company exploiting cellular stress for the treatment of advanced and metastatic cancers. Our discovery platform drives the development of Heat Shock Factor 1 (HSF1) degraders - HSF1 is a highly validated cancer target that is critical for oncogenic stress resistance. Sisu is optimizing small molecules that selectively degrade the active form of HSF1, killing chronically stressed cancer cells while sparing normal cells. Sisu’s HSF1 degraders have demonstrated strong efficacy in multiple animal models of advanced, therapy-resistant cancers.

QureBio

QureBio Ltd. was established in July 2017 and aims to develop novel therapeutic drugs for the treatment of tumors, autoimmune diseases and metabolic diseases in China and around the world to benefit patients. The company has multiple technology platforms for antibody drug development including phage display, hybridoma as well as protein and antibody engineering. In addition, the company's goal is to make an effective and robust manufacturing process for biologics.