Cayuga Biotech is a preclinical therapeutics company developing therapies for bleeding disorders. Our lead candidate is a platelet-like drug that accelerates clotting in the presence of injury. Cayuga has already held its pre-IND meeting with the FDA and received guidance on the GLP trials required for IND approval. The Cayuga team is made up of PhD scientists who developed CAY001 during their PhD research. We have two KOLs who are experts in the field of hemostasis and have overseen clinical trials on anticoagulant reversal drugs. Our regulatory strategy is developed by an advisor who spent 23 years at the FDA in CDER. Our manufacturing advisor has overseen 9 drugs reach clinical trials and 4 drugs receive approval. Our business advisors have overseen 4 successful business exits worth over $1 B. Cayuga has raised over $2 MM from DARPA and the Army. We are raising a round to complete the remaining IND-enabling studies, add experienced drug developers to the team, and prepare for the first in man trial.
Working men and women lift every day. They do it in warehouses and on production lines. In offices and combat zones. Every day, they lift the economy worldwide. Whether it’s the day-to-day grind or responding to a crisis, their strength supports us all. And they deserve gear that sustains their strength, for work and for life. That makes their job safer. The HeroWear Apex means that their weight is over. The world’s first exosuit for all, built from the ground up for both men and women, the Apex is a back-assist exosuit that uses a proprietary band-based mechanism to reduce strain on the back without getting in the way.
Zeit Medical develops a platform health monitoring technology that enables continuous neurophysiological assessment for a wide gamut of neurological diseases affecting Americans of all ages. Our first target is stroke (severe cerebral ischemia). Stroke is a devastating disease. In the United States alone, it is the leading cause of long-term disability and cognitive impairment, as well as the fifth leading cause of death. Someone in the United States suffers a stroke every 40 seconds and an estimated $72 billion are being spent annually on direct medical care of stroke patients. Our alert system is capable of reliably recognizing neurological events within minutes and alerts the patients, caregivers and 911.
Researchers at Stanford have developed a new water-based disinfectant with the potential to destroy a wide variety of pathogens and significantly improve healthcare settings. AquaROS, a novel and broad-spectrum disinfectant, is based on the formation of micron-sized water droplets that cause the spontaneous generation of highly reactive oxidant species (ROS). Access to safe and effective disinfecting solutions like AquaROS is more critical than ever – in the U.S. approximately the same number of people die from healthcare-associated infections (HAIs) as from AIDS, breast cancer and auto accidents combined. Inexpensively produced by atomizing water, the new disinfectant has been shown to outperform other commercial methods at inactivating Salmonella typhimurium and E. coli. Based on these and other promising results, the researchers anticipate AquaROS can kill bacteria, fungi, mycobacteria and bacterial spores on critical surfaces such as medical instruments.
There are 230 million surgeries performed per year worldwide. Surgical experience is the primary factor dictating outcome of each operation. However, surgical experience is not quantifiable, not accessible as a data source, and not easily distributable between users. This leads to variability in outcomes, complications, inefficiency, and high costs in surgical care delivery. What if an artificial intelligence (AI) platform could be designed that would quantify and learn from surgical experience, then customize and distribute tailored information back to all surgeons in real-time?
Using patent-pending technology developed at Vanderbilt University, our technique overcomes cost and scaleability limitations associated with traditional manufacturing techniques, while also giving commercial viability to carbon conversion technologies through the introduction of a high-value secondary material produced from greenhouse gas inputs. Our technique relies on electrochemistry, rather than solely catalysis, and results in a highly efficient process to convert atmospheric carbon dioxide into useful functional nanomaterials.
The human immune system participates in complex interactions with virtually all other systems in the body. In particular, the B-cell component of the adaptive immune response plays a role in various disease settings, including infectious disease, cancer, autoimmunity, cardiovascular, hematologic, neurologic diseases, and others. In addition, antibodies (a product of B cells) are effectively used in diagnostics, therapy, and prevention. To address these significant challenges with current technologies for B cell characterization and antibody discovery, we recently developed a novel technology that, for a given sample, enables the mapping of antibody sequence to antigen specificity from a single high-throughput experiment for a large number of antigens and B cells at a time.
Immuno-Modulators of Alzheimer’s Disease (I.M.A.D.) was founded by Dr. Elizabeth Bradshaw and Dr. Wassim Elyaman from Columbia University’s Department of Neurology. The team is pioneering genetically-driven strategies to treat Alzheimer’s disease by directly targeting susceptibility genes in the CNS innate immune cells, called microglia. Their lead program is a preclinical non-antibody biologic which has been shown to target the CD33 genetic susceptibility signaling pathway in human microglia and clear β-amyloid from the brain of treated mice.
Cache is a MIT startup from Prof. Mark Bathe’s lab is seeking an entrepreneur to help lead the next phase of customer discovery/hypothesis validation. Their technology enables massive and low-energy storage, up to millennium timescales, and random access of nucleic acid samples from broad sources including ecological, forensic, or archival DNA data storage. They are seeking a passionate, adventurous, and creative entrepreneur to join their early-stage venture to help launch this technology into the 21st-century biotechnology space. Their immediate aim is to recruit an entrepreneur who will be excited to mature their business plan using market discovery and research on product-market fit. They are ready to test the market’s appetite for this technology, developing partnerships along the way.
Designer biopolymers have numerous biotech applications due to their superior specificity and efficacy. However, their large size and complexity hindered the development of effective engineering methods, leaving developers with suboptimal molecules. This is most prevalent in drug discovery and diagnostics: patients routinely develop strong adverse reactions to biologics (e.g., many SARS-CoV-2 IgG tests have weaker specificity and affinity, leading to dubious results). Optimer is changing the status quo through its hybrid computational and experimental biopolymer engineering platform capable of designing DNA, RNA, and proteins with superior properties - especially target activity and specificity - for a broad range of biotech solutions. Optimer is currently rigorously testing and aggressively pursuing patent protection for its methodology and won a pilot award from Columbia’s Translational Therapeutics (TRx) Lab-to-Market accelerator program.
Sharper Sense is developing noninvasive neuromodulation technology that enhances users’ senses. Accurate perception of details in sensory stimuli heavily impacts ability to perform tasks correctly and safely. Unfortunately, impaired senses can arise from a variety of causes including fatigue, aging, and injury. Sharper Sense’s technology is based on research that elucidated neural circuitry which rapidly improved sensory processing and perceptual sensitivity when activated. In subsequent research, our team developed a method of using peripheral nerve stimulation to activate this sensory- sharpening circuitry and enhance perception on demand.
Rapid, reliable, and widespread testing is required to curtail the ongoing COVID-19 pandemic. Current gold standard diagnostic assays are hampered by supply shortages in critical reagents including nasal swabs, RNA extraction kits, personal protective equipment (PPE), instrumentation, and labor. This WUSTL team lead by Jeffrey Milbrandt and William Buchser overcome these challenges with the development of a rapid colorimetric assay using reverse-transcription loop-mediated isothermal amplification (RT-LAMP) optimized on human saliva samples without an RNA purification step. This optimized reaction has been prototyped and tested and can provide for an affordable point-of-care diagnostic of SARS-CoV-2.
Keren Therapeutics is focusing on pre‐clinical and early clinical development of recently discovered hormone osteocalcin as treatment for sarcopenia, cancer cachexia, and cognitive impairment due to aging. Osteocalcin is a biological construct that can be synthesized on commercial scale and delivered via depot administration. Development of osteocalcin benefited from more than $7 million in grant support. Columbia patented the composition of matter and its utility through 2034 and 2031, for muscular and cognitive impairment indications, respectively. KEREN intends to file an IND within 18 months from funding. Cost of development through completion of first-in-man studies is expected to be $8,200,000. Osteocalcin is addressing blockbuster market with unmet medical need in case of sarcopenia. We anticipate selling KEREN or executing a co‐development agreement with pharma within 6 years from funding. The Company’s value at that time could exceed $50 million, providing investors with 6x ROI.
Veramorph is developing a polymer-based pre-formed oral dosage technology as a more effective drug delivery vehicle for poorly soluble small molecule drugs. Our technology is capable of improving oral drug delivery performance of a much broader range of small molecule drugs the existing formulation methods. Our product, disintegrating polymer oral dosages (DPODs), can enable more effective drugs to be brought to clinical trials that will improve success rates and reduce the overall cost of successfully commercializing a drug product. Veramorph is developing an internal pipeline of reformulated products and is also seeking product development partnerships through licensing agreements with pharmaceutical companies.
A research team at the UMN has developed a system that predicts and optimizes the charge needed for commercial electric vehicles. The system is currently being applied to “last mile” delivery vehicles. The technology is a cloud-based software service that uses prior vehicle performance and external information like traffic and weather to train machine learning algorithms that, in turn, predict remaining vehicle range and battery state of charge. The result is a connected energy management system (C-EMS) that reduces range anxiety for fleet operators and enables greater penetration of electric vehicles into commercial fleets. The team, in conjunction with the UMN Venture Center, is seeking a business executive to form and launch a business with the research team and then lead and operate the business. C-VEM is already serving as the backbone to several companies providing solutions in this space.
AimRNA’s novel RNA bioengineering technology produces a wide variety of biologic RNA molecules through a large yield, cost effective bacterial fermentation process, among them a number of leads have been demonstrated efficacy and safety for the treatment of lung and liver cancer in animal models. We have been awarded with SBIR Phase I grant in 2018, and we are currently raising $500,000 to fund IND-enabling studies and $7 million for clinical investigations.
Theranostec have developed a novel, nanoparticle treatment platform allowing for targeted and effective chemotherapy, which can be enhanced with phototherapy, while also lowering toxicity.
VasoBio is startup company focusing on vascular disease treatment & medical devices. VasoBio has developed a proprietary ligand-based coating technology, VasoCoat, to improve vascular grafts and other blood contacting devices. Our technology overcomes the major barriers of currently available vascular grafts and possesses optimal functionality. The current focus of VasoBio is to apply VasoCoat to improve patency of hemodialysis vascular graft and reduce thrombosis, stenosis, and infection.
Viruses engage receptors on a host cell to initiate infection. Soluble versions of the receptors act as decoys that bind and block receptor-binding sites on the virus. However, human receptors often bind viruses weakly and in most cases also interact with endogenous factors in the human body as part of their normal physiology. These competitive off-target interactions adversely impact safety and efficacy. There is therefore an affinity and specificity problem that must be solved before soluble receptors can achieve their therapeutic potential. Orthogonal Biologics uses deep mutagenesis to resolve these challenges, engineering receptors that are orthogonal to normal human biology and with affinities that rival monoclonal antibodies. At this time, the company has lead biologic drug candidates for SARS-CoV-2 (the virus responsible for COVID-19) and human cytomegalovirus (the leading non-genetic cause of birth defects), as well as ongoing interests in zoonotic pathogens with pandemic potential and herpesviruses.
Sierra Biopharma is developing therapeutics that selectively neutralize, remove, and destroy immune components that lead to autoimmune diseases, starting with the orphan indication myasthenia gravis (MG). Sierra Biopharma has developed an antigen-specific therapeutic for MG that targets pathogenic antibodies (Abs) and the memory B-cells (mBCs) that lead to their production. This biologic compound currently binds to and neutralizes the main immunogenic region (MIR) in directed pathogenic Abs and should eliminate their mBCs.
FatesEDS is developing a compact, sensitive X-ray differential phase contrast (DPC) imaging system that improves field of view, increases fringe visibility and shortens imaging times. This system surpasses the performance of 3-grating DPC systems used today with a single grating that is combined with a specialized X-ray source and detector. By eliminating the two expensive grating components, the overall size and potential cost of the system is reduced along with enabling a single-step image acquisition with a field-of-view of ~35 degrees and fringe contrast ~25%. FatesEDS is focused on developing a next-generation baggage inspection system to identify hazardous materials, with a potential medical imaging application. FatesEDS is seeking an experienced entrepreneur to help lead the commercialization of this system.
MS patients have an imbalance of Effector and Regulatory cells which causes nerve damage. The solution is a Novel small molecule STAT3 inhibitor, LLL12B, that improves Effector/Regulator cell balance. LLL12b significantly suppresses the disease progression in three murine MS model. There is a great opportunity for this solution as MS is prevalent in 300 people per every 100,000 in the US and is onset in the age range of 20-40.
Early indication of fluid accumulation to trigger adjustments to medical therapy preventing costly readmissions and avoiding life threatening complications.
Many diseases are managed or treated by invasive IVs or frequent injections. We are all familiar with the difficult routines of chemotherapy to treat cancer and insulin injections to manage diabetes. This is particularly true for diseases that impact the eye. All of us will experience some form of age-related vision loss, with some of the leading causes with no cure shown here, including diabetic retinopathy, glaucoma, and macular degeneration. Loss of vision greatly reduces quality of life, and impacts daily activities we take for granted including reading and driving. Most of these diseases require chronic local treatment to prevent permanent blindness, and the only option may include frequent intraocular injections.
There is a growing aging population and there is no cure or treatment for Alzheimer's. The only current drugs are palliative. The Synapse Enhancer enhances the integrity of the synapse which improves cognitive function and can delay/halt neurodegeneration. It could be utilized for a many diseases such as, Alzheimer's, ALS, Parkinson's, Huntington's, Traumatic Brain Injury, Stroke, Epilepsy, Depression, Neuropathic Pain Disorders, and Addiction.
Currently there are several solutions under consideration for disinfecting N95 respirators for re-use, with UV-C as the preferred solution because of ease of deployment and widespread applicability. Problematically, present UV-C disinfection solutions are limited by shadowing, which prevents exposure of the masks to the required effective dose of UV irradiation. The inventors utilized UV-C light field modeling and extensive test measurements to develop a proprietary arrangement of lamps for disinfecting. The configuration eliminates shadowing, allowing for full irradiation and disinfecting of the contaminated N95 respirator surface in the shortest amount of time.
Parkinson's disease (PD) is characterized by uncontrolled tremors and issues with movement and balance. It affects 1-2% of people over 65, costing the US over $35 billion a year. Current treatments for PD, such as L-DOPA and deep brain stimulation (DBS), only treat the motor symptoms but are not able to fix the underlying cause of the deficits: loss of the nigrostriatal pathway. To address this gap in clinical care, the first “tissue engineered nigrostriatal pathway” was developed outside the body and precisely microinjected as a unit to “wire in” and physically replace the missing pathway, “reversing the clock” on the neurodegenerative progression of PD. Preliminary results in animal models show success recreating the pathway which delivers the dopamine signals that are lost in PD patients. Full pitch deck available upon request.
HL BioTherapeutics aims to apply our unique immunotherapy platform that functions like a T cell receptor, to treat multiple types of solid and liquid cancers. The scientific founders, Catherine Bollard and Eric Yvon, have clinical and technical experience in designing and delivering novel immunotherapies to patients. The advantage of the HL BioTherapeutics platform is the extremely high specificity for tumors due to targeting of a specific MHC-displayed peptide from SSX2 protein. This targeting platform may be commercialized as an engineered T cell or NK cell, or as a therapeutic antibody. Our most advanced preclinical data uses T cell therapy for acute myeloid leukemia.
Our lab has developed novel broad-spectrum antivirals for treatment of all nine human and several animal herpesviruses that overcomes drug resistance. We have shown that the pressurized state of the viral packaged genome is a target for antiviral therapies. This pressurized DNA state in herpesvirus capsid shell (exerting tens of atmospheres of pressure) is responsible for DNA ejection into a cell nucleus, causing infection. Several lead compounds that "turn off" capsid pressure and block viral genome ejection into a cell have demonstrated superior efficacy and safety in testing conducted at the NIH/NIAID. Although high activity has been shown with all human herpesviruses and several animal herpesviruses, our focus will be on treatment of human cytomegalovirus and MDV (Marek’s disease virus in poultry). For HCMV treatment, the market is dominated by generics, such as ganciclovir and foscarne, that have low efficacy and safety profiles. For MDV, no treatments are available.
Therapeutic options for glioma cancers are limited, and the survival rate for glioma patients is poor. Athena is progressing NAMDT inhibitor therapy by applying it to cancers with specific mutations, thus increasing the efficiency and decreasing toxicity of treatment.
Bone-Rad Therapeutics, Inc. is a Delaware corporation whose objective is to develop and market Spine-Rad (TM) Brachytherapy Bone Cement as an innovative, improved, and cost-effective treatment paradigm for the management of cancer tumors in the spine which affect over 230,000 patients per year in the U.S. and a similar number in the E.U. Four patents covering this technology have been issued and exclusively licensed to Bone-Rad Therapeutics. An additional patent is pending. Developed at The University of California Irvine, Spine-Rad Cement delivers internally-targeted radiation therapy directly to the tumor as it simultaneously treats existing or impending vertebral fractures, restoring strength to the bone. Administered in a single procedure, Spine-Rad Cement will eliminate the 10-20 hospital visits typically needed for external beam radiation therapy (EBRT) as well as the significant side effects of EBRT.
Many disease-causing proteins are difficult to inhibit directly with biologics and small molecules. However, new advances allow for the control of the abundance of proteins using small molecules, providing a path to medicines that selectively control protein stability. Pomerex Therapeutics is building a platform for the systematic discovery of small molecules that control the stability of disease-modifying proteins. They have developed novel compounds that can cause the destruction of otherwise undruggable but crucial protein targets, including those driving the genesis and survival of aggressive cancers. They are seeking funds to expand their platform and develop their lead oncology programs.
Bacchus Therapeutics is a biotech company that exploits cancer’s hypermetabolic state by targeting specific cancer metabolic pathways. The aberrant expression of MYC is virtually unmatched, making MYC one of the most frequently deregulated oncogenes in human tumors, including, liver, kidney, CNS, hematopoietic, breast, prostate, lung and GI tumors. Human studies and animal models have established that dysregulation of MYC underlies the pathogenesis and aggressiveness of numerous cancers. Our founders, Drs. Gouw and Felsher, discovered that MYC regulates the aberrant lipid metabolism of tumors and that inactivation of key lipid enzymes results in regression of tumors.
Magnets form the core of a wide variety of power electronic devices including motors, inductors and transformers. New magnetic materials are needed to meet the high performance requirements for grid modernization (solid state transformers, PV inductors, sensors); for EVs and aviation (inductors, sensors, motors); and for industrial motor controls (inductors, sensors).
Our technology combines novelty in bioabsorbable stent technology and regenerative medicine. Our team has unrivaled experience in both technologies and have taken the project funded by the European commission to an advanced stage in development. Our initial therapeutic target will be peripheral vascular disease where there is no comparative technology in use or development. Beyond this, there is potential widespread application of the device and concept to the heart, brain, liver and cancer treatments where the combined technology can provide a unique state of the art therapeutic system.
Multidrug-resistant Gram-negative bacilli (MDRGNB) have emerged as a challenging cause of hospital-acquired infections and present a critical need for innovative antibacterial development. Two new oxopyrazole agents targeting penicillin binding proteins (PBPs) based on a non-beta-lactam core have superior MIC50 values to current billion-dollar last resort antibiotics like Ceftazidime/Avibactam or Meropenem. One shows broad Gr- efficacy while the second oxopyrazole is selective for Acenitobacter baumannii. On target, good in vivo PK, no mammalian toxicity, no off-target liability. Seeking funding for definitive in vivo efficacy studies.
1 in 400 Americans carry a breast cancer gene (BRCA) mutation - such a mutation increases the likelihood of developing breast cancer from 12 to ~70% by age 80, and raises the lifetime risk of ovarian cancer from 1.3% to 44% (BRCA1) or 17% (BRCA2). Additionally, men with BRCA1 or BRCA2 mutations are also at increased risk for breast and prostrate cancers, and both men and women with either mutation are at an increased risk of pancreatic cancer. BRCA proteins are essential for DNA repair, making BRCA-deficient cells (i.e. those with a BRCA mutation) susceptible to synthetic lethality and providing an opportunity to kill cancers with the mutation. PARP-inhibitors invoke synthetic lethality to kill BRCA-mutated cells via PARP-trapped lesions, however cells can become resistant to this mode of therapy, creating an unmet clinical need for these cancers. Indeed, nearly 300,000 new cases of breast or ovarian cancer will be diagnosed in 2019, making the need for an improved synthetic lethality agent urgent.
Leptospirosis is the Ebola virus of the bacterial world, difficult to diagnose, and for which there is a 20% mortality rate. Leptospirosis is a global health threat, particularly for travelers and for soldiers. The vaccine for human leptospirosis is unsafe and of unknown efficacy, creating a vast domestic and global demand for an effective vaccine that is currently unmet. Current vaccines are bacterin type vaccines and of limited efficacy, duration, and safety, however LeptoX is offering a first-in-class vaccination (first for animals, to be developed for humans) that outcompetes its competitors in all of these categories.
"Cold" tumor cancers, such as breast, pancreatic and prostate cancers, are difficult to treat with immunotherapy due to a lack of infiltration into the tumor by T cells. EvolveImmune has developed a treatment which can be used alone or in combination with other cancer drugs to increase the immune response and T cell infiltration in the tumor tissue.
Numerous clinical trials in amyloidosis have failed as a result of misdirected focus on amyloid states of disease-causing proteins. Pangolin Therapeutics’ (PTx) small-molecule platform, termed Pangomers™, was developed to address this deficiency. The Pangomer™ core structure enables selective targeting of pre-amyloid, toxic oligomers (PAOs). Here, we seek to address Multiple System Atrophy (MSA), an aggressive, orphan-indicated form of Parkinson's for which there are no approved therapeutics. Our pilot efforts have identified and validated a strategy for development of Pangomer™ analogues that neutralize PAOs from MSA. Additional funding will allow execution of this strategy delivering a lead molecule for pre-clinical advancement.
Despite it being a rare disease, pulmonary arterial hypertension (PAH) is a costly and deadly condition of the pulmonary artery. Verso therapeutics is pursuing an HDAC inhibitor as a treatment for PAH which restores normal pulmonary vasculature unlike existing treatments which fail to address the abnormal architecture.
A vast majority of drugs and biologics fail to enter the brain for treatment of brain cancers and neurological diseases. Our discovery yielded key insights into how the immune system naturally overcomes the blood brain barrier to fight infections. We leverage this insight to enable safe and transient drug access to the CNS using a simple intranasal peptide delivery approach.
New biomarker for novel CD8 T cell subset that drives systemic sclerosis immunopathology, including monoclonal antibodies anti-human C10orf128 and new targets for small molecule inhibitor therapy. Monoclonal antibodies recognized 5-10% of circulating T cells. Humanized version of them are likely therapeutic agent for systemic sclerosis after humanization. Also so likely have a monoclonal antibody specific for mouse homolog of C10orf128 to enable an animal preclinical model for T cell depletion studies. There is no effective treatment for systemic sclerosis; 50,000 individuals have Ssc; fatal disease. Potential applications include pulmonary fibrosis, asthma with scarring among others.
Kawasaki Disease is an illness that causes inflammation (swelling and redness) in blood vessels throughout the body that comprises three phases, the first of which is usually a lasting fever. The condition most often affects kids younger than 5-8 years years old. Catching Kawasaki disease early is key - children can be treated relatively easily if the disease is detected early, however becomes much more dangerous the longer it goes in diagnosed. The first sign of the disease, fever, is unspecific to Kawasaki disease, and by the time later symptoms manifest, much more aggressive intervention needs to be taken. Thus, a diagnostic that can identify Kawasaki Disease is critical to successfully treating the 5,000-20,000 cases that arise each year in the US, and more globally.
Idiopathic pulmonary fibrosis (IPF) is a deadly chronic lung disease with median survival of 3 years and with a worse prognosis than lung cancer. 6 million people worldwide are affected - 200,000 in North America are affected with 45,000 dying each year. The progressive decline of lung function characterizing it is interspersed with unpredictable disease flares called acute exacerbations of IPF (AE-IPF) that accelerate lung function loss and increase morbidity and mortality. The annual incidence of AE is up to 20% with a mortality ranging from 35-90%, demonstrating the severity of IPF disease progression and the importance for active disease monitoring (ER visits and hospital stays can amount to >$11,500 per case). Current therapeutics are unable to predict how an individual patient will progress and whether they will respond to available interventions. The market size for biomarker chip detection of PIF is ~$3B, underscoring the need for a more robust treatment for PIF.
The three members of the endocrine FGF family, FGF19, FGF21 and FGF23 are important circulating hormones that regulate a variety of critical metabolic processes. Endocrine FGFs mediate their cellular processes by binding to and activating FGF-receptors (FGFR) in complex with Klotho proteins. Based on the crystal structure of ligand occupied Beta-Klotho new potent engineered endocrine molecules were developed for treatment of metabolic disease that will benefit from therapeutic stimulation of FGF21 cellular pathway such as pancreatitis, Nash and obesity. Moreover, also potent inhibitors including small molecules will be developed for treatment of bone disorders (XLH) and liver cancer, respectively.
Our goal is to develop a new cancer drug that is an antibody drug conjugate (ADC). It targets the pi subunit of the Gamma Aminobutyric Acid Receptor (GABRP) that is aberrantly expressed in a broad range of solid tumors. The target was discovered by the Pusztai lab and a provisional patent application has been submitted. We will use the Blavatnik Fund to perform affinity maturation, generate humanized anti-GABRP antibody conjugated to emtansine and assess the anti-tumor activity in vitro and in vivo.
Adjuvant therapeutics are therapies given after the initial or primary treatment of a disease or condition, for example, therapeutics given after a surgery to maximize its effectiveness. Pharma companies make diagnostics for the metastatic setting, to enrich the population that will benefit from the drug. However, the big prize for Pharma is the adjuvant setting, which sees at least double and up to 10 times more patients than the metastatic setting. Despite this, pharma companies rarely make diagnostics for the adjuvant setting. For example, in the immunotherapy space, 1 in 5 lose thyroid function and 1 in 100 die from the drug used to treat the condition during the course of treatment.
Over 95% of rare disease have no treatment or cure: there are over 7,000 known rare diseases collectively affectiting 30 million Americans. Many of these disease are genetic disorders, in which there can be considerable genetic variation between patients. Current drug development pipeline does not cater to rare mutations, as drug development often takes more than 10 years and over $2B to get a drug to market, making the small patient populations inhibiting. Even if a therapy is developed, genetic variation of the disease means not all patients in the already-small patient population may be able to be treated, limiting their impact and incentive to develop these therapies.
PARP inhibitors are promising targeted therapy for cancers with defective homologous recombination (HR) repair. However, as PARP inhibitor become widely used, there will be an increase in patients with PARP inhibitor resistance. To overcome this problem, we have developed DB4, a small molecule drug that inhibits HR repair. Combining DB4 and the PARP inhibitor olaparib inhibits the progression of resistant ovarian cancer and increases the survival time of tumor-bearing mice. Thus, we request the Blavatnik fund to continue developing DB4 for improving its potency and PK properties in vivo and conducting efficacy studies with patient-derived cancer xenograft models in mice.