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Pathogenic fungi are a major public health threat, causing failure of implanted organs and devices, neonatal mortality and much more. Unfortunately, it is difficult to develop specific drugs against these infections because fungal cells are a lot like those in people: we are both eukaryotes that share a similar set of enzymes and pathways. To address this problem, the Pyle lab has specifically targeted the unique RNA metabolism of fungal cells, giving rise to a new generation of nontoxic drugs that are ready for development and implementation. This has received a Blavatnik Award in May 2019.

Transfoam

Transfoam harnesses engineered microorganisms and renewable feedstocks to tackle plastic pollution at the beginning and end of the product lifecycle. Our proprietary bioprocess reduces the operating cost and environmental footprint of PHB manufacturing to overcome limitations currently hindering the industry’s growth. Plastic is at the forefront of our transition to a circular economy. We treat plastic waste as a valuable resource rather than a burden. By using a waste-based feedstock, we can both remove plastic pollution from the environment and provide a safe, accessible alternative. Polyhydroxybutrayte (PHB) is a bio-based polymer with similar properties as many traditional single-use plastics, except it degrades fully in the environment. PHB is both produced and consumed by microorganisms, leaving behind only traces of water and CO2 after use/disposal.

Vellum Biosciences

University of California, Irvine

Little is known about how engineered CAR-T cells move through the body and proliferate after they are first removed, altered, expanded in number and, finally, returned to a patient's body. Vellum Biosciences has developed a way to genetically tag CAR T cells that enables them to be imaged via positron emission tomography (PET) scan in combination with a radiotracer specific to that tag.

Tendo Technologies

Princeton University

Tendo Technologies, founded in 2017, is a spin-off from the Marcus Hultmark research lab at Princeton developing a novel flow sensing/measurement technology. This unique technology achieves extreme sensitivity (for both gases and liquids) through the nanoscale conductive ribbons that will deflect when placed in flow. The nanoribbons are manufactured with standard semiconductor-based techniques, resulting in high scalability and low unit cost for the sensing chip. As a highly versatile technology (flow measurement is ubiquitous), Tendo envisions that their tech can be applied in many industries for various applications, when equipped with appropriate "wrappers" around it. For example, the Tendo chip plus communication package would be appropriate for IoT applications such as real time air measurement in HVAC ducts; alternatively, more sophisticated housing with control would bring benefits for flow metering and control during industry manufacturing processes.

Bone-Rad Therapeutics

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.

geCKo Materials

University of California, Santa Barbara

geCKo Materials provides volume manufacturing for a bio-inspired, energy saving, reusable, multi-purpose, industrial and consumer adhesive. The mechanical adhesive is composed of directional, microscopic inclined features with a tapered geometry. geCKo’s unique IP is in the manufacturing method, introduced by Dr. Capella Kerst in her PhD at Stanford and protected by pending patents. Tested in the lab and in use with customers, a 1 square inch adhesive is capable of supporting 15 lbs of weight in shear with no attachment nor detachment force needed. The geCKo Opposing Force scales with area and can be purchased at any area. Integrating onto your robot grippers is simple, just a peel and stick. We can offer integration assistance and expertise for simple, quick and easy integration.

Stretchable Electronics

Conventional electronic devices based on flexible or rigid printed circuit boards (PCBs) cannot conform to curved surfaces or stretch to accommodate new requirements of disruptive technologies. Additionally, existing methods for the production of stretchable electronics are unable to produce devices with high integration densities achievable with conventional methods. These methods also rely on processes that cannot be implemented at scale. This technology overcomes these limitations and enables practical applications of stretchable electronic devices for health monitoring, wearable computing, medical devices, and beyond.

Eleven P15

University of Colorado

Idiopathic Pulmonary Fibrosis (IPF) is a chronic, fibrosing interstitial pneumonia of unknown cause, occurring primarily in older adults. IPF is a fatal disease with median survival at 3-5 years. While there exist several clinical approaches to treating IPF, few therapeutics have been clinically proven to provide significant benefits to patients. Eleven P15 is uniquely positioned for early detection and early intervention of IPF. The pivotal discovery of a common polymorphism in the promoter of MUC5B (rs35705950) has been associated with IPF and pulmonary fibrosis associated with rheumatoid arthritis, and this polymorphism increases the production of mucus.

Clarasim Systems

ClaraSim Systems, Inc. has developed a computer simulated environment in which surgeons can perform surgery on virtual bone using virtual instruments. By combining patient-specific data with a powerful simulation engine, we are able to create 3-D patient models that can be explored and modified in real-time with virtual surgical tools providing both haptic and visual feedback. Our system enhances the surgeon's ability to perform skilled surgery by enabling them to rehearse drilling through accurate representations of the patient's bony and fragile structures. The anatomical insight gained from interacting with the simulation provides guidance during actual surgery leading to better outcomes. While the system enables preoperative rehearsal, it is also used as a teaching tool, giving trainees the opportunity to experience many different cases in a safe and efficient setting. The technological and clinical capabilities of the product stem from over 30 person-years of research conducted by the company founders.

SkyNano Technologies

Vanderbilt University

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.

Pulmera

Pulmera enables x-ray visualization of the tracheobronchial tree to aid physicians in guiding endoscopic tools in the pulmonary tract. Early diagnosis and treatment are vital for improving lung cancer survival rates, and tissue biopsy is necessary for diagnosis. However, performing a lung biopsy is difficult due to the complexity of the highly branched airways of the lung, making it difficult to access and obtain the tissue and the procedure can pose a risk to the patient. Electromagnetic navigation bronchoscopy (ENB) is the safest way to obtain a biopsy but diagnostic success is limited by poor real time visualization of significant portions of the lung. Should the biopsy procedure be unsuccessful, patients may then need to have the tissue surgically removed. In a high number of these cases, the nodules are found to be benign. Pulmera will greatly improve the ability to obtain a diagnosis for the patient using ENB.

Bioada

Rutgers University

The scientific community is continuously trying to improve their understanding of genetic mechanisms in biological systems and finding biomarkers for diagnosis, prognosis and treatment. With growing accessibility, still, only the minority of investigators in the life and medical sciences has the means to analyze and leverage this enormous treasure of data. Bioada is an integrated, easy to use and interactive genomics data analysis platform for the broader community of life scientists.

DeckTherapeutics

Columbia University

THDG3 represents a novel, patent protected omega-3 diglyceride emulsion developed by Dr. Deckelbaum’s laboratory at Columbia University. Acute injection of THDG3 emulsion following stroke leads to a marked reduction in brain tissue death (up to 90%, demonstrated in 6 rodent models), with associated preservation and recovery of both short and long term neurofunctional outcomes. Due to the high risk profile and short window of administration, less than 8% of stroke patients receive treatment with t-PA, the current standard of treatment for stroke. DeckTherapeutics is currently developing THDG3 emulsion as a drug candidate for standard emergency treatment for stroke, a global unmet need, with phase 2a trials planned in 2021.

MyophonX

Cornell University

Solutions for silent speech production lack innovation, cultural acceptability, and often involve invasive surgery. MyophonX is cutting-edge wearable technology for silent communication, with applications to stroke and laryngectomy patients as well as voice searching.

KayoThera

Princeton University

Kayothera is leveraging cutting-edge discoveries to develop therapeutics for cancer patients who currently receive terminal diagnoses. Our two pipeline candidates target solid tumors to 1) inhibit tumor survival mechanisms in advanced or chemoresistant disease, 2) deplete immunosuppressive Regulatory T cells to restore anti-tumor immunity. With these first-in-class small molecule therapeutics, we aim to give hope to patients with intractable cancers.

South Rampart Pharma

South Rampart Pharma aims to advance the safe treatment of pain by developing new small-molecule solutions that have the potential to overcome many risks associated with current pain medicines. South Rampart Pharma’s lead compounds have been effective in reducing both pain and fever in preclinical studies without the liver and kidney toxicity associated with current non-opioid analgesics. As a new small molecule treatment option that is not a biologic therapy, South Rampart Pharma’s compounds have great potential as a value product that will be low cost and accessible to many patients.

Exolva Therapeutics

Moving Therapeutic Proteins Into the Cytosol and Nucleus. Exolva is using CPMPs (cell-permeant miniature proteins) to deliver therapeutic enzymes and gene-editing tools to correct inborn genetic disease. CPMPs are small, folded proteins that contain a specific array of five Arg residues on an ⍺-helix backbone. CPMPs can reach cytosol and nucleus with efficiencies as high as 75%. CPMPs possess many advantages relative to previous, purported ‘cell-penetrating peptides’, including low toxicity, high and tunable stability, enzyme cargo retains enzymatic activity, among other features.

Triursus Therapeutics

Graft versus host disease (GVHD) is a severe immune response in patients after transplantation, such as bone marrow transplantation. GvHD is costly to treat and has a high mortality. The current strategy for preventing GvHD include general immuno-suppresent drugs, with limited efficacy and side effects. Therefore, a more targeted approach is needed to prevent GvHD. This technology is a monoclonal antibody which targets cell surface markers on intestinal crypts, which is the primary site of immune attack in GVHD.

Lifeguard

Patients who undergo life-saving cardiac procedures (such as aortic valve replacement) are often exposed to a related harm: stroke, infarction, and brain injury. These strokes are due to the release of emboli, or particulate material such as a plaque, that travel in the blood from the heart to the brain during surgery. In response to a growing body of evidence of this problem, Lifeguard is a neuro-protective device that provides coverage to all three aortic arch takeoffs. Lifeguard is led by Yale Professor of Medicine Dr. Alexandra Lansky and Yale Associate Professor of Biomedical Engineering Dr. Tarek Fahmy.

PBP Antibiotics

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.

Prolifagen

Carnegie Mellon University

The heart is a muscle, and like all types of muscles, it can be injured. In humans, heart muscle is not able to regenerate after injury. This can lead to heart disease which develops over time, eventually leading to an untimely death. Prolifagen is combining discoveries in gene therapy, materials science, and drug delivery to bring human tissue regeneration therapies to reality. The lead drug candidate is a proprietary microRNA embedded in a hydrogel construct to enable a single-shot therapy for heart tissue regeneration, bypassing the need for exogenous stem cells. Prolifagen is seeking seed funding for IND-enabling porcine studies and a new CEO to lead the team into their next chapter.

CorePower Magnetics

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).

EXOForce Robotics

Wearables are transforming how we exercise, train and perform. EXOForce Robotics is commercializing a groundbreaking high quality, stretch athletic textile that conforms to the body to enhance sensing performance, representing a substantial leap beyond current biometric offerings. These intelligent garments sense muscle behaviors and whole body biomechanics and use machine learning to tailor readouts to the wearer, quantify their workouts and alert them to impending injury. This technology has broad utility in the athletics (professional sports and consumer wellness), medical (rehabilitation and remote patient monitoring) and military markets. Ongoing R&D is focused on next-gen garments that both sense and intervene, and in the next few years will power everyday users to exercise their muscles while going about their tasks like walking their dog or running errands, provide postural corrections to prevent long-term pain or discomfort, and motivate those with sedentary lifestyles to get moving.

Aperiam Bio

While deep learning methods exist to guide protein optimization, examples of novel proteins generated with these techniques require a priori mutational data. Aperiam is taking the guesswork out of biotechnology with computer vision that illuminates critical parts of protein chemistry. We have trained a portfolio of computer vision algorithms on protein chemistry and use those algorithms to mutate genes for improved catalysis, expression, stability, and solubility. We are now using this approach to build our own suite of stabilized and solubilized enzyme therapeutics for direct delivery to the lung via nebulization and in parallel tailoring the computer vision framework to antibodies for expedited development and improved manufacturability.

AgingSense

AgingSense is a company focused on developing technological solutions to complex problems of aging to improve quality of life of older adults. The first technological solution being developed is Heart Failure Monitoring Socks. This wearable sock technology monitors persons with heart failure for exacerbations as well as response to treatment. The socks monitor for changes related to heart failure specifically, leg edema, using stretch sensors, and fatigue using actigraphy and gyroscopes. Data is sent by blue tooth to an app which is run through an algorithm in a secure cloud that triggers a message to the individual with HF, a trusted other and/or their provider of changes in their condition. The ultimate goal is to improve heart failure management to keep persons with heart failure home and out of the hospital with a better quality of life.

Oath Endovascular

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.

Acurasset

Princeton University

Viral infections causing hepatitis are major contributors to human morbidity and mortality. Currently, there are no approved directly acting antivirals that cure two of these viruses - hepatitis B (HBV) and E viruses (HEV) – that account for close to 300 million infections world-wide. Our goal is to develop novel small molecule therapeutics that can be readily deployed to combat efficiently these diseases. To achieve this, we capitalize on discoveries and technical advances from our lab that put us in a unique position for targeted high through-put screens. We have already identified a lead molecule that is effective against multiple HEV genotypes, and we have uncovered a minimal set of host factors that are essential for HBV persistence.

LensGuide Imaging Technology (LEGIT)

Columbia University

LEGIT uses a new optical concept (a lensguide) developed at Columbia University to improve optical endoscopy. Their goal is the development of a whole microscopy probe (size ~ 0.1-1 mm) that can be inserted in the body, with minimal damage, for high resolution imaging (<1 µm resolution). The LEGIT endoscopic probe can be used for cancer diagnostics (e.g. skin and liver cancer) and in vivo biopsy imaging. The LEGIT lensguide is a higher quality and less expensive replacement for the GRIN lenses with significantly higher flexibility in design and manufacturing.

RiboRupt Biotech

Massachusetts Institute of Technology

We have applied a unique, robust, and comprehensive image-based assay developed in our laboratory to discover small molecule inhibitors of nucleolar function. Results from pilot screens on FDA-approved drugs reveal 83 unique hits that include known and putative antineoplastic agents.

Veramorph

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.

Yoma Biosciences

DNA damage can be detrimental to cells as it can lead to mutagenesis, genotoxicity and tumorigenesis. One of the most frequent forms of DNA damage is 8-oxoguanine (8-OG), which is repaired by OGG1. Bioactive inhibitors of OGG1 and the GO pathway can be useful tools to investigate the connections of this enzyme activity to disease. Yoma Biosciences has developed potent, selective, small-molecule inhibitors of OGG1 and other enzyme targets in the GO pathway. These inhibitors provide useful tools to study the biological roles of OGG1 and also have therapeutic potential for cancer and inflammation. With multiple enzyme targets in the pathway (OGG-1, MTH1, MutYH, …) - currently un-drugged, Yoma has multiple chances for 1st in class candidate discovery.

WNT Scientific

Columbia University

People with temporomandibular joint osteoarthritis (TMJ OA) experience pain and limited jaw function. Approximately 10 million Americans are affected by TMJ OA, and current treatments are invasive and have high failure rates. Furthermore, palliative treatments aimed at increasing short-term function don't prevent disease or promote regeneration of tissue. StemGEL, WNT Scientifics' solution to this problem, uses fibrocartilage stem cells to regenerate cartilage. They have a paper in Nature Communications (along with several other publications) detailing the successful results. The market size of TMJ OA patients that can be treated by StemGEL is approximately 2.6 million.

CynAxis

University of California, Santa Barbara

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.

Cadence Shoe Technologies

Foot Drop is a mobility disorder prevalent in patients that is a common result of neurological injuries or diseases such as stroke cerebral palsy, MS or brain tumors. Foot drop is characterized by the inability to lift the toes toward the shin. It inhibits the rhythmic swing phase of the gait, increasing the probability of falls and manifests in abnormal gait patters that inhibit mobility and create imbalances and stress. Currently, there is not a rehabilitative solution that is cost effective and practical for every day independent use. Dr. Tyler Susko has developed a shoe that provides a low-friction out-sole during the swing phase of the gait, and a high-friction out-sole during the stance phase of the gait. The design can be used on any style shoe. Preliminary pilot studies with impaired adults have shown immediate gait improvement.

Tandem Repeat

Tandem Repeat Technologies, Inc. (TR Tech) is the market leader in Sustainable Textiles Manufacturing. TR Tech has created the world’s first programable textile which is 100% sustainable, self-healing, and fully biodegradable. Established in 2018, TR Tech has developed revolutionary technology that combines materials science and biomanufacturing to produce performance composites with tunable properties. The technology is covered under five (5) issued US patents and additional five (5) pending with Squitex. The physical characteristics of Squitex will allow it to be adopted into many products and technologies. It is a platform performance material that is renewable, green, strong, flexible, self-healing, energy efficient and friendly to most of today’s manufacturing processes. The use will only be limited by the creativity of application and the ability to produce the quantity of material to meet market demand.

Free Dynamics

Washington University in St. Louis

The logistics of ensuring compliance of thermal profiles (e.g., keeping cold items cold) for each product is a difficult problem. Solutions exist for monitoring temperature at coarse scale (shipping container), but those that precisely track each unit (color-changing stickers), lack the ability to precisely convey information in a scalable manner. Free Dynamics designs electronic temperature sensors that operate without any external batteries for months. Unlike current solutions, our quantum-mechanically-driven sensors are miniature and cost-effective enough to be incorporated into every vial of essential drugs that require stringent storage conditions. As a result, we can assure last-mile compliance while expecting higher accuracy. In addition, we have also shown that our system can be used for authentication which will guard the tagged assets against tampering and counterfeiting.

Milo Sensors

University of California, Santa Barbara

Millions of people all over the world face challenges with alcohol. One of the more traditional solutions people first consider is Alcoholics Anonymous. Through market research, we have learned that more and more non-traditional solutions are being sought out to supplement the recovery journey and we, at Milo Sensors, believe we can provide an option for the supervised or self-monitoring of alcohol consumption. Unlike other existing alcohol monitoring tools such as ankle bracelets, ION™ is designed for the wearer first, with discretion, comfort, and usability at the forefront. Common industries that benefit from similar monitoring devices, such as the criminal justice system or family law, seem to follow only after the recovery support network has failed and act as a punitive measure. In contrast, we aim to help anyone who seeks to improve their general wellness by focusing on their relationship with alcohol and then help rebuild trust with surrounding family members and supporters.

Intervir

Our ultimate goal is to develop novel small molecule, broad-spectrum therapeutics against viral infections caused by filoviruses, arenaviruses, rhabdoviruses, and others that depend on the PPxY L-domain motif for virus egress and spread of infection. Some of these viruses, including Ebola (EBOV), Marburg (MARV), and Lassa fever (LAFV) viruses, are highly pathogenic and classified as Category A, bioterror pathogens. Importantly, we have identified an analog capable of blocking in vivo activity in a Marburg virus challenged mouse model, providing essential proof of concept for this novel class of anti-viral therapeutics. We predict that the novel class of anti-viral products targeting EBOV, MARV, and LAFV will be used for treatment of infected individuals as well as in prophylactic treatment of soldiers, healthcare workers, or others at high risk. Emergency administration of such an antiviral therapeutic during an outbreak could potentially inhibit virus dissemination in infected individuals.

Parallel Works

University of Chicago

Parallel Works is a web-based platform that simplifies and automates the complex workflow of parallel computing. They speed up simulation and analytics campaigns across industries by making parallel computers from hybrid clouds and clusters easier to use. Their platform is built on the Parsl and Swift parallel scripting technologies developed over the last decade by Argonne National Laboratory, and is designed to run simulation and analytics on the world's largest computing resources.

HerShield

University of Pittsburgh

Genital herpes affects more than 500 million globally and it is incurable. In the US alone, 1 million people are diagnosed every year with herpes. Herpes causes painful viral outbreaks, significant psychological stress, and social stigma. Even though women are more prone to genital herpes, the available prevention option, condoms, is controlled by the male partner. The solution is a new female-controlled product called HerShield, a vaginally applied, soft and flexible pharmaceutical film that has proven safety in two phase I clinical trials, and provides sufficient quantities of drug for protection from herpes. HerShield is designed to be discrete, exceptionally portable, low cost, biodegradable, and easily self-administered without the need for an applicator.

LeptoX

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.

AlvaHealth

Getting immediate medical attention is critical after a patient has experienced a stroke. However, stroke symptoms are often not identified by the patient or family members until it is too late to administer treatment. There is currently no wearable-linked mobile app which specifically detects a stroke incident. AlvaHealth is pairing wearable devices with machine learning to detect a stroke incident immediately based on the activity in both arms of the patient. Early detection of stroke can improve the outlook for patient recovery.

PETcoil

PETcoil enables healthcare providers to offer PET/MRI scans with better imaging quality for a fraction of the cost compared to existing solutions. Commercial integrated PET/MRI scanners cost ~$6M + ~$2M for required room renovations, an unaffordable cost for many institutions. PETcoil's patented portable PET insert can be placed into any existing MRI scanner, enabling it to perform simultaneous PET/MRI at 1/8th of the cost compared to integrated systems. A proof-of-concept radiofrequency (RF)-penetrable PET insert has been developed and successfully tested, supported by grants from the NIH, Stanford Bio-X & Biodesign programs, and the Coulter Foundation.

Surgical ai

Harvard University

Operating rooms are often not efficient and are costly to hospitals. Solutions to increasing efficiency neglect to monitor errors in real time which can be stopped and improve efficiency from the time of the incident. This technology pairs artificial intelligence with real time monitoring of operating room procedures to increase efficiency of hospital operating rooms.

GAP Gene Engine

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.

Lkit Therapeutics