QBI

UCSF QBI and the Department of Psychiatry and Behavioral Sciences Announce Breakthrough Research Findings in the Molecular Understanding of Autism

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Monday, December 4, 2023
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The Quantitative Biosciences Institute (QBI), in collaboration with the Department of Psychiatry and Behavioral Sciences, at the University of California, San Francisco (UCSF), today reported new breakthrough research findings in the understanding of autism spectrum disorder (ASD).

Key Points: 
  • The Quantitative Biosciences Institute (QBI), in collaboration with the Department of Psychiatry and Behavioral Sciences, at the University of California, San Francisco (UCSF), today reported new breakthrough research findings in the understanding of autism spectrum disorder (ASD).
  • Belinda Wang, MD, Ph.D., Rasika Vartak, Ph.D., Fima Zaltsman, Ph.D., and Zun Zar Chi Naing, Ph.D. are co-first authors of the manuscript.
  • Their findings provide unprecedented insights into the molecular mechanisms of a subset of individuals with ASD who carry specific genetic mutations, serving as a new platform for desperately needed therapeutics development.
  • QBI’s convergent biological systems approach that formed the basis of this study can be applied to virtually any disease area.

UCSF QBI, University College London, and Mount Sinai Identify Shared Molecular Mechanisms Across SARS-CoV-2 Variants that Allow Virus to Thrive Despite Vaccination

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Thursday, September 21, 2023
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SAN FRANCISCO and LONDON, Sept. 21, 2023 (GLOBE NEWSWIRE) -- In a study published online in CELL today, scientists at UCSF QBI, University College London and the Icahn School of Medicine at Mount Sinai reported breakthrough findings on convergent evolutionary mechanisms shared by COVID-19 variants, allowing them to overcome both adaptive and innate immune system barriers. In the paper titled, SARS-CoV-2 Variants Evolve Convergent Strategies to Remodel the Host Response, scientists carried out an unprecedented, systematic comparative study using the most infectious COVID-19 variants, namely Alpha, Beta, Gamma, Delta and Omicron to identify specific viral mutations responsible for hijacking a common host pathway, thereby leading to increased transmissibility, infectivity and survival. Specifically, they discovered a convergence in potent suppression of interferon-stimulated genes through several viral proteins, including Orf6 and Orf9b, which serve as innate immune antagonist proteins capable of blocking innate host immune response.

Key Points: 
  • The award, which included a $200,000 prize, is intended to provide support for his vision for progress against pandemic threats.
  • “Unfortunately, we continue to see new mutations and strains of SARS-CoV-2 despite innovations in new vaccines,” said Dr. Krogan, who founded the QBI Coronavirus Research Group (QCRG).
  • This finding is consistent with our investigation of early SARS-CoV-2 variants where certain viral proteins were highly expressed in infected cells which helped the virus infect our cells.
  • Their analysis pinpointed cellular pathways that are similarly modulated across variants during infection and represent putative targets for pan-coronavirus antivirals.

eFFECTOR Therapeutics Announces Positive Top-Line Results from Phase 1b Clinical Trial of Zotatifin for the Treatment of COVID

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Tuesday, February 21, 2023
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These results were presented at the 30th Conference on Retroviruses and Opportunistic Infections on February 20, 2023.

Key Points: 
  • These results were presented at the 30th Conference on Retroviruses and Opportunistic Infections on February 20, 2023.
  • The Hazard Ratio (HR) for achieving VLU in saliva was 2.83 (95% confidence intervals 0.64, 12.5; p=0.13) in favor of zotatifin.
  • At the outset of the trial, one patient received zotatifin and one patient received placebo by intravenous administration.
  • Enrolled subjects had mild or moderate COVID and were positive for SARS-CoV-2 RNA or antigen within 7 days of randomization.

Rezo, Founded by Renowned UCSF Scientists, Launches with $78 Million Series A to Advance Pipeline of Precision Therapeutics Based on Groundbreaking Integrated Disease Network Mapping Platform

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Thursday, November 17, 2022
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SAN FRANCISCO, Nov. 17, 2022 (GLOBE NEWSWIRE) -- Rezo Therapeutics, a biotechnology company pioneering the integrated mapping of disease networks for precision therapeutics, launched today with $78 million from a Series A financing. The Series A financing was led by SR One, a16z Bio + Health, and Norwest Venture Partners, and also included SV Angel, Liquid 2 Ventures, and Hawktail. Rezo’s platform is based on technology from the Quantitative Biosciences Institute (QBI) at the University of California, San Francisco (UCSF) developed by Nevan Krogan, Ph.D., director of QBI and a co-founder and chief executive officer of Rezo. Chemistry pioneer and inventor of leading drugs targeting KRAS, Kevan Shokat, Ph.D., is also a co-founder as well as a professor at UCSF, and an investigator of the Howard Hughes Medical Institute (HHMI).

Key Points: 
  • The platform enables the rapid identification and study of disease-causing protein and genetic interactions, which would be difficult to discover using traditional, often siloed, scientific methods.
  • Rezos disease-specific maps are designed to pinpoint novel, druggable targets that can be attacked with a range of treatment modalities.
  • Rezo aims to rapidly advance a pipeline of therapies, initially focused in solid tumor oncology guided by mutation and allele-specific biomarkers.
  • The companys initial focus is in oncology, with plans to explore additional therapeutic areas through collaborations and partnerships.

eFFECTOR Therapeutics Completes Enrollment in Second of Three Cohorts of Phase 1b Clinical Trial of Zotatifin for the Treatment of COVID-19

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Wednesday, October 26, 2022
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eFFECTOR anticipates opening enrollment in the third cohort by the end of 2022, and expects to report topline data for all three cohorts in the first half of 2023.

Key Points: 
  • eFFECTOR anticipates opening enrollment in the third cohort by the end of 2022, and expects to report topline data for all three cohorts in the first half of 2023.
  • Zotatifin is an investigational host-directed antiviral, meaning that it acts on a human protein that the SARS-CoV-2 virus hijacks to synthesize new viruses.
  • As such, zotatifin may have a higher barrier to viral mutational escape than therapies that target components of the virus itself.
  • eFFECTOR cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements.

eFFECTOR Therapeutics Doses First Patient in Second Cohort of Phase 1b Clinical Trial of Zotatifin for the Treatment of COVID-19

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Wednesday, September 14, 2022
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SAN DIEGO and REDWOOD CITY, Calif., Sept. 14, 2022 (GLOBE NEWSWIRE) -- eFFECTOR Therapeutics, Inc. (NASDAQ: EFTR), a leader in the development of selective translation regulator inhibitors (“STRIs”) for the treatment of cancer, today announced it has dosed the first patient in the second cohort of its Phase 1b clinical trial of zotatifin in non-hospitalized adults with confirmed COVID-19 infection. The study is a double-blind, randomized, placebo-controlled trial evaluating the safety and antiviral activity of a single dose of zotatifin and is being conducted in collaboration with the Quantitative Biosciences Institute (QBI) at the University of California, San Francisco, under a $5 million cooperative agreement sponsored by the Defense Advanced Research Projects Agency.

Key Points: 
  • Dose escalation to the second cohort comes after the positive recommendation of the independent data safety monitoring board upon review of safety data from the first dose cohort.
  • The completed cohort also included the first subjects dosed with a sub-cutaneous formulation of zotatifin and preliminary analysis demonstrated equivalent plasma drug levels compared to IV delivery.
  • Achieving equivalent drug levels when zotatifin was delivered by the sub-cutaneous route provides an opportunity for convenient dosing in both the COVID and cancer settings.
  • As such, zotatifin may have a higher barrier to viral mutational escape than therapies that target components of the virus itself.

Looking Beyond DNA to See Cancer with New Clarity

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Thursday, September 30, 2021
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Krogan noted that targeted treatments based simply on DNA sequencing of tumors havent been as effective as hoped.

Key Points: 
  • Krogan noted that targeted treatments based simply on DNA sequencing of tumors havent been as effective as hoped.
  • We realized we need another way to look at cancer that takes it a step beyond DNA.
  • The bottom line is that were elevating the conversation about cancer from individual genes to whole protein complexes, Ideker said.
  • DNA contains the instructions for building proteins, which then interact with other proteins, almost always in large groups called complexes.