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Yale Lifesciences PitchFest button Yale Lifesciences PitchFest

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PitchFest 2020 virtual

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Over two days, the top 22 biotech opportunities developing within Yale took the virtual stage to present their five-minute pitches to a panel of ten senior executives from biotech investment funds and pharmaceutical companies. Thank you to our sponsors, judges and an online audience of approximately 300 attendees from over 150 companies and investment firms.

Read more about the event and our winners here.


Congratulations to our Shipman & Goodwin PitchFest 2020 Winners!

Awards presented by Dormer Stephen, Shipman & Goodwin.

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Most Valuable Pitch: Angelica Gonzalez

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Most Innovative Breakthrough: Ranjit Bindra & Mark Saltzman


Best Presentation: Choukri Ben-Mamoun


Highest Potential Impact: Joseph Contessa

Congratulations to our GenScript & GenScript PROBIO PitchFest Audience Choice Winners!


Viewers' Choice Award Day 1: Anjelica Gonzalez

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Viewers' Choice Award Day 2: Ranjit Bindra




Susan J. Baserga, MD, PhD
William H. Fleming MD Professor of Molecular Biophysics & Biochemistry
RiboRupt Biotech Interrupting ribosome biogenesis to cure cancer
Project Summary

We are seeking seed funding to advance screen hits to lead compounds for first-in-class cancer therapeutics. The nucleolus has recently emerged as a promising new target for anti-cancer therapies. 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 the Life Chemical FSP3  Library screen yielded 137 compounds with reproducible anti-nucleolar activity. We will use the proceeds for target identification, to determine anti-cancer cell growth activity in cultured cells and for efficacy and toxicity studies in mice.

OCR Business Development Lead: David Lewin 

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Choukri Ben Mamoun, PhD
Professor of Medicine (Infectious Diseases) and of Microbial Pathogenesis
A Novel Therapy for Pantothenate Kinase-Associated Neurodegeneration
Project Summary

Pantothenate kinase-associated neurodegeneration (PKAN) is a profoundly disabling genetic disorder that manifests with severe dystonia, young-onset of parkinsonism and blindness. The disease is caused by mutations in the PANK2 gene, which encodes the enzyme pantothenate kinase, responsible for the synthesis of coenzyme A. One therapeutic strategy is to activate the human PanK3 enzyme. We have discovered a novel class of safe and highly potent activators (AC50 between 2 and 268 nM) of PanK3. We request funds to support structural and pharmacological characterization of the compounds and to evaluate their biological activity using in vitro and animal models of human PKAN.

OCR Business Development Lead: John Puziss  

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Anton Bennett, PhD
Professor of Pharmacology
MAP kinase phosphatase-1 Inhibition as a target for NASH
Project Summary

The therapeutic challenges of developing new modalities to treat non-alcoholic steatohepatitis (NASH) have been substantially underestimated. To date there are no successful therapies on the market for the treatment of NASH. The MAPK phosphatase-1 (MKP1) is a stress-responsive phosphatase that is overexpressed in obese humans and in the livers of mice fed a NASH-inducing diet. Liver-specific deletion of MKP1 protects from the development of NASH thus, validating MKP1 as a NASH target. This project will use proprietary approaches to identify and develop novel small molecule allosteric inhibitors to MKP1 for the treatment of NASH.

OCR Business Development Lead: David Lewin

Bindra and Saltzman
Ranjit Bindra, M.D., Ph.D. and W. Mark Saltzman, Ph.D.
Professors of Therapeutic Radiology
BrainStorm Cancer Therapeutics: Redefining the blood-brain barrier
Project Summary

The blood-brain barrier is a major obstacle for treatment efficacy in the treatment of both primary CNS cancers and brain metastases, as most drugs are not CNS permeable and systemic toxicities are dose-limiting. BrainStorm Therapeutics seeks to leverage proprietary nanoparticle formulations and novel delivery strategies developed in the Saltzman laboratory, combined with brain tumor biomarkers identified in the Bindra Laboratory, to overcome this major problem. We will develop an entirely new therapeutic strategy for the treatment of CNS tumors, with great potential for rapid translation into the clinic.

OCR Business Development Lead: John Puziss

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Angelique Bordey, Ph.D.
Professor of Neurosurgery and of Cellular And Molecular Physiology
Pre-Clinical Candidate NT-001 for Tuberous Sclerosis Complex Seizures
Project Summary

Data from our lab provide evidence of Filamin A (FLNA) being a druggable target for the treatment of epilepsy in an orphan disorder Tuberous Sclerosis Complex. Our goal is to test the pre-clinical efficacy of our lead compound, a small molecule FLNA modulator, and request funds for the following tests: examining PK, BBB permeability, and microsomal stability of our lead compound, and reproducing the pre-clinical studies by a CRO.

OCR Business Development LeadDavid Lewin

Demetrios Braddock
Demetrios Braddock, M.D., Ph.D.
Associate Professor of Pathology
Targeting NETs in Human Disease
Project Summary

'Neutrophilic Extracellular Traps' or NETs are now accepted pathogenic mechanisms driving morbidity and We describe stable and bioavailable blood based enzymes which degrade

OCR Business Development Lead: David Lewin

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Steve W. C. Chang, PhD
Associate Professor of Psychology and of Neuroscience
Clinical Proof of Concept of a Yale Invention/Product as a Breakthrough Treatment for Autism
Project Summary

There are no drugs approved to treat core symptoms of autism, the costliest pediatric disease in the US (estimated $250 billion/year). Oxytocin, the drug with the most-promising clinical data, has successful, placebo-controlled trials on four continents, but some results are weak.

The laboratory of Steve Chang at Yale has discovered, published and filed for patent on the amplification of oxytocin’s effect through combination with naloxone. The Chang patent is being combined with oxytocin formulation and delivery patents owned by the NYC-based biotech Pastorus. The Blavatnik award would fund a proof-of-concept clinical trial of the [oxytocin + naloxone] product.

OCR Business Development LeadLolahon Kadiri

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Joseph N. Contessa, M.D., Ph.D.
Professor and Vice Chair, Therapeutic Radiology
Developing Therapeutic OST inhibitors
Project Summary

We have completed a high throughput screening campaign that delivered first in class inhibitors of the OST. Medicinal chemistry efforts have produced small molecule analogs with anti-tumor activity, but without toxicity, in animal models. We now seek to identify an advance a clinical candidate for testing in cancer patients.

OCR Business Development LeadJohn Puziss

Jason Crawford
Jason Crawford, Ph.D.
Maxine F. Singer '57 Associate Professor of Chemistry and Associate Professor of Microbial Pathogenesis
Stilbene a new antibiotic class for topical and systemic administration
Project Summary

Stilbenes are dietary metabolites with polypharmacological activities. While tapinarof ($330 million Dermavant) is in phase 3 trials for the treatment of psoriasis and atopic dermatitis, other members have failed trials for inflammatory bowel diseases (IBDs) due to interindividual variability. Microbiome contributions were not considered. We discovered a bacterial enzyme that transforms stilbenes into novel metabolites with potent biological activities. Indeed, the tapinarof-derived product activates a clinical target with higher potency than the parent drug. The product is also effective at killing multidrug resistant bacteria without developing resistance. We propose that stilbene transformations could explain the interindividual variability in clinical trials.

OCR Business Development Lead: David Lewin

Mark Fields
Mark Anthony Fields, M.P.H., Ph.D.
Assistant Professor of Ophthalmology and Visual Science
RegenaVision: Retinal Therapies to Restore Vision
Project Summary

The etiology of age-related macular degeneration (AMD) is caused by many factors including oxidative stress/mitochondrial dysfunction, resulting in retinal pigment epithelial (RPE) cell dysfunction and eventual cell death or what is known as geographic atrophy AMD in late stage “dry” AMD. We developed a high-throughput assay that mimics the effects of oxidative stress and identified three protective chemotypes, which are novel, drug-like and represent excellent starting structures for further development. Secondary analysis of the identified show efficacy in human RPE cells in in vitro and in vivo models of oxidative stress, blue light-induced damage, and aged Bruch’s membrane.

OCR Business Development LeadLolahon Kadiri

Anjelica Gonzalez
Anjelica Gonzalez, Ph.D.
Associate Professor of Biomedical Engineering
Preclinical Validation of a Low-cost Versatile Respirator
Project Summary

Our world will look different in three years after the ongoing pandemic has ended, however, the need for versatile devices to treat respiratory distress will remain. Prior to the pandemic, respiratory distress was responsible for 3.2% of all neonatal deaths in the US, as well as affecting over 200K adults per year. We have developed a low-cost all-in-one system that can deliver sterilized, humidified, warmed, and oxygenated air to patients. If awarded the Blavatnik, we intend to complete the remaining preclinical work and submit our device for regulatory approval within 7 months.

OCR Business Development LeadRichard Andersson

Junjie Guo
Junjie Guo, Ph.D.
Assistant Professor of Neuroscience
Discovery and Optimization of Novel Compounds Targeting Programmed Ribosomal Frameshifting in RNA Viruses
Project Summary

Effective and broad-spectrum antivirals are much needed not only for the current pandemic, but also for future emerging viral pathogens. We have recently developed a platform to rapidly identify compounds that modulate programmed ribosomal frameshifting, a prevalent feature in a large variety of RNA viruses including all coronaviruses. Using this platform, we have identified a novel frameshifting inhibitor for SARS-CoV-2, which effectively inhibited SARS-CoV-2 replication in vitro. The current project aims to (1) further optimize the activity of this inhibitor by chemical modifications and (2) perform an expanded search for additional chemical scaffolds for SARS-CoV-2 frameshift inhibitors.

OCR Business Development LeadHong Peng

Samuel Katz
Sam Katz, M.D., Ph.D.
Associate Professor of Pathology
Targeted Therapy for T-cell Lymphomas
Project Summary

Reprogramming a patient’s own cytotoxic T cells with a Chimeric Antigen Receptor (CAR) to attack B cell leukemias is remarkably successful. In contrast, T cell lymphomas are highly aggressive and remain incurable. Here, we develop a novel CAR to target a subset of T cell lymphomas, while avoiding self killing and employing strategies for synergistic combination therapies.

OCR Business Development LeadChris Unsworth

Mustafa Khokha
Mustafa Khokha, M.D.
Professor with Tenure of Pediatrics (Critical Care Medicine)
Small molecule regulation of β-catenin nuclear transport: new targets against cancer
Project Summary

Excessive Wnt signaling is a major driver of cancer. For example, >90% of colon cancers have excessive Wnt signaling. In order to find therapeutics, we need to understand how β-catenin enters the nucleus, a key step in the Wnt signaling pathway. My lab has demonstrated that β-catenin nuclear transport requires Ran, employs TNPO1/2 as a nuclear transport receptor, and binding to TNPO1 is via a PY-NLS in which mutation of just two amino acids disrupts binding. We propose screening for small molecules that will disrupt this direct binding and inhibit β-catenin nuclear transport for cancer therapeutics.Excessive Wnt signaling is a major driver of cancer. For example, >90% of colon cancers can be attributed to excessive Wnt signaling. Discovery of effective small molecules for treatment are hampered by our lack of knowledge of how β-catenin enters the nucleus, a key step in the Wnt signaling pathway. Our lab has demonstrated that β-catenin nuclear transport requires Ran, employs TNPO1/2 as a nuclear transport receptor, and binds to the TNPO1 via a PY-NLS in which mutation of two amino acids disrupts binding.

OCR Business Development Lead: Lola Kadiri 

Themis Kyriakides
Themis Kyriakides, Ph.D.
Associate Professor of Pathology
The Matrix Reloaded: Engineering Materials for Regenerative Medicine
Project Summary

Project involves the development of native materials derived from genetically-modified pigs. Primary commercial targets include hernia repair scaffolds and vascular grafts. Funds are requested to create a path forward towards 510K application and perform toxicity/biocompatibility and large animal studies.

OCR Business Development LeadDavid Lewin

Brett Lindenbach, Ph.D.
Associate Professor of Microbial Pathogenesis and of Comparative Medicine
Biosafe SARS-CoV-2 replicons for high-throughput drug discovery
Project Summary

There is a pressing need for antiviral compounds that target SARS-CoV-2 replication enzymes, not only to combat the current pandemic, but for future coronavirus outbreaks. A key impediment is the lack of a robust high-throughput viral replication assay with low biosafety requirements. We have developed SARS-CoV-2 replicons – fragments of the viral genome that replicate in cells but do not make infectious virus particles – and shown that they are useful for drug screening, dose titration, and drug synergy studies. We will complete optimization of second-generation replicons, conduct pilot screens, and validate assay scalability and robustness for large-scale screening.

OCR Business Development Lead: Chris Unsworth

Nikhil Malvankar
Nikhil S. Malvankar, Ph.D.
Assistant Professor of Molecular Biophysics and Biochemistry
Faster, Reliable & Scalable DNA Sequencing using Biomolecular Electronics
Project Summary

This project will develop a novel DNA sequencing technique using direct electronic detection of bases. By combining the advances in CMOS chips, nanofabrication and sensor technology, this DNA reading platform will deliver the lowest cost (< $100) genome for precision medicine, read DNA digital data storage, and will serve as handheld device for point-of-care molecular testing.

We will develop single-molecule electronics sensors for direct detection of molecular activity by leveraging on our discovery of protein nanowires - first biomolecules with a unique combination of chemical compatibility with sensing enzymes and electronic connectivity with nanoelectrodes (Cell 20119; Nature Chem. Bio. 2020).

OCR Business Development Lead: Rich Andersson

Naiem Nassiri
Naiem Nassiri, M.D.
Associate Professor of Surgery (Vascular)
Direct intralesional mTOR inhibition for targeted treatment of sporadic and syndromic venous and lymphatic malformations
Project Summary

Venous and lymphatic malformations are morbid clinical entities with no approved or standard therapeutic options. Current treatment varies widely, has limited durability and side-effects. We have developed a unique emulsion and novel route of delivery for targeted treatment of venous and lymphatic malformations with a focus on the mutated etiologic molecular pathway.

OCR Business Development LeadDavid Lewin

Pusztai and Vignesh Gunasekharan
Lajos Pusztai, M.D., D.Phil. and Vignesh Gunasekharan, Ph.D.
Professor of Medicine (Medical Oncology), Associate Research Scientist
Miso Therapeutics: Targeting cancer cells' Metabolic isoenzyme addiction
Project Summary

Triple-negative breast cancer (TNBC) lacks targeted therapies and therefore rely on chemotherapy with its associated side effects. TNBC is an aggressive disease with high metabolic rewiring. We have identified PCK2 as a TNBC-specific metabolic target by comparing RNA sequencing data from patient-matched clinical samples. PCK2 is a novel target and lacks an inhibitor, a protein model, and an enzyme assay. To this end, we have created a homology model, a high throughput enzyme assay, and identified an active hit. Using the Blavatnik fund, we propose to perform lead optimization, PK/PD, and in vivo validation.

OCR Business Development Lead: David Lewin

Aaron Ring
Aaron Ring, M.D., Ph.D.
Assistant Professor of Immunobiology
Seranova Bio: Discovering drug targets from clinical trials of nature
Project Summary

Our goal is to discover novel drug targets and develop therapeutic antibodies by screening patients for “protective” autoantibodies that correlate with favorable responses.

We combine high-quality, comprehensive protein sampling with yeast display with next-generation DNA sequencing to profile patient plasma/serum samples with much greater throughput and fidelity than existing technologies.

We will screen 1,000s of patient samples from autoimmune disease, neurodegeneration and cancer (post-treatment with immunotherapy) to identify novel disease targets and potentially therapeutic antibodies cloned directly from patient B cells.

OCR Business Development LeadHong Peng

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Faye Rogers, Ph.D.
Associate Professor of Therapeutic Radiology
Direct Targeting of Amplified Cancer Driver Genes as a Therapeutic Strategy
Project Summary

Gene amplification is a major molecular mechanism driving tumor progression and clinical outcomes in a broad spectrum of cancers. The current treatment strategy has been to develop drugs that target the over-expressed protein products. Although these drugs have been major breakthroughs in cancer therapeutics, drug resistance often hampers their clinical efficacy. We have developed a novel therapeutic strategy for targeting cancers characterized by gene amplification, in which manipulation of DNA damage response with triplex-forming oligonucleotides drives tumor specific induction of apoptosis.

A finished presentation is coming.

OCR Business Development LeadLolahon Kadiri

Joseph Vinetz
Joseph Vinetz, M.D.
Joseph Vinetz, M.D.
Development of camostat-related compounds for COVID-19 and other coronavirus infections
Project Summary

COVID-19 is causing untold global harm to human health. There is no anti-viral treatment for early COVID-19 infection. A drug to prevent SARS-CoV-2 infection and transmission would have global importance including virus eradication. Camostat, a repurposed serine protease inhibitor in Phase II clinical trial at Yale for early, outpatient, treatment of COVID-19, requires molecular optimization. The goal of this project is to develop a more potent and selective TMPRSS2 inhibitor with robust anti-COVID-19 efficacy and pharmacokinetics sufficient to enable once-daily oral dosing in humans. Such a drug might be active against other coronaviruses given known mechanism of action.

OCR Business Development LeadDavid Lewin


Sarah Bhaghat, Ph.D.

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Sofinnova Investments, Partner

Brian Bronk, Ph.D., Sanofi

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Alexandra Cantley, PhD, Polaris

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Alicia Iruztun-Lafitte, MBA, UCB Ventures

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UCB Ventures

Kuldeep Neote, PhD, Lilly

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Gordon Ng, Ph.D.. AbbVie

Gordon Ng

Nilay Thakar, PhD, Arch Venture Partners

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Arch Venture Partners

Jim Tobin, PhD, J&J Innovation

J&J Innovation

Brian Yordy, Ph.D., F-Prime

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Paul Young, Ph.D.

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Merck, Executive Director of Business Development & Licensing

Special Thanks to

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The Blavatnik Fund is Supported by a Grant from the Blavatnik Family Foundation and Administered by the Yale Office of Cooperative Research.

As Always, Thank You

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