Impact

The UC Berkeley Molecular Therapeutics Initiative (MTI) announced a new collaboration agreement with BioMarin Pharmaceutical Inc. aimed at accelerating the discovery and development of innovative therapies for rare genetic conditions. The partnership brings together UC Berkeley’s strengths in fundamental disease biology, novel therapeutic technologies, and next-generation drug discovery, with BioMarin’s deep expertise in translating science into […]

Parkinson’s disease afflicts more than ten million people worldwide, and with prevalence continuing to rise, the need for therapies that do more than merely mask symptoms has become urgent. In response, UC Berkeley’s Molecular Therapeutics Initiative (MTI) has selected three research teams for the inaugural MTI Parkinson’s Therapeutics Program, launched last autumn to transform high-impact academic […]

BERKELEY — UC Berkeley’s Molecular Therapeutics Initiative (MTI) has launched a new funding program to accelerate drug discovery for Parkinson’s disease and related synucleinopathies. The call seeks translational projects that move from mechanism to tractable leads, drawing on Berkeley strengths in chemical biology, structural and cell biology, data science/AI, and deep ties to biotech. “Parkinson’s […]

As federal research funding faces growing uncertainty, the Molecular Therapeutics Initiative (MTI) at UC Berkeley is stepping up to support bold scientific innovation. This week, MTI announced the recipients of its 2025 MTI Innovator Awards, honoring six scientists for their transformative approaches to therapeutic discovery. MTI was founded to reinvent drug discovery at the interface […]

UC Berkeley launches the Molecular Therapeutics Initiative (MTI) to accelerate drug discovery

Markita Landry announced as laureate for Blavatnik National Award

Julia Schaletzky named as Executive Director of the Molecular Therapeutics Initiative

Ziyang Zhang awarded the NIH Director's New Innovator Award

Jennifer Doudna featured in Fast Company Cover article as one of the 10 most innovative people of the last 10 years

Helen Bateup is part of a research team awarded a $12 million grant from the California Institute for Regenerative Medicine!

Small molecule modulation of protein corona for deep plasma proteome profiling

The SIFI complex silences mitochondrial stress responses via ubiquitin-mediated degradation, promoting cell survival and offering a potential treatmen

Engineered iGeoCas9 RNP–LNP complexes enable efficient, high-precision genome editing in vivo, achieving up to 37% editing in liver and lungs.

SARS-CoV-2 Nsp1 traps RNA in the nucleus to escape immune detection

TFEB controls expression of human syncytins during cell-cell fusion

Discovery of a covalent destabilizing degrader of the undruggable oncogenic transcription factor beta-catenin

Induced proximity modality to enable targeted protein localization of nuclear proteins into the cytosol

Hachiman inhibits phage replication by indiscriminate DNA degradation

Incorporation of Multiple β2-Hydroxy Acids into a Protein In Vivo Using an Orthogonal Aminoacyl-tRNA Synthetase

Spectrin mediates 3D-specific matrix stress-relaxation response in neural stem cell lineage commitment

The viral protein structural database and analyses presented here afford new opportunities to identify mechanisms of virus–host interactions that are

DCAF16-Based Covalent Handle for the Rational Design of Monovalent Degraders

Deletion of the autism risk gene CNTNAP2 in mice increases dorsolateral striatal projection neuron excitability and promotes cognitive inflexibility.

This technology enables us to visualize oxytocin within the brain at the level of individual synapses, providing a valuable tool for the neuroscience

Tau fibrils induce nanoscale membrane damage and nucleate cytosolic tau at lysosomes

Long-term super-resolution inner mitochondrial membrane imaging with a lipid probe

These data reveal the nature of direct Ca2+-dependent membrane binding and its interplay with Ca2+-dependent protein binding in the function of ALG-2

Rational Chemical Design of Molecular Glue Degraders

Engineered Glucose Oxidase-Carbon Nanotube Conjugates for Tissue-Translatable Glucose Nanosensors

Mechanosensitive stem cell fate choice is instructed by dynamic fluctuations in activation of Rho GTPases

p300-chromatin binding requires multiple TF-interaction domains

LYCHOS, a lysosomal protein, senses cholesterol and activates mTORC1 signaling by binding GATOR1, linking cholesterol to cell growth regulation.

ORF29, a viral DNA packaging motor subunit, also enhances late gene transcription in KSHV, linking viral packaging and gene expression.

Identification of structurally diverse small molecule inhibitors of ferroptosis target FSP1

Orphan quality control shapes network dynamics and gene expression

Expanding the substrate scope of PylRS enzymes to include non-⍺-amino acids in vitro and in vivo

CRISPR systems lacking Cas4 use fused or recruited exonucleases for faithful acquisition of new CRISPR immune sequences

Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes

Induced proximity therapeutic modality DUBTACs for targeted protein stabilization of aberrantly degraded proteins that cause disease

Rewiring of selenoprotein hierarchy in cancer & id of ribosome stalling & collisions during GPX4 translation as ferroptosis vulnerabilities

Vaccinia virus D10 has broad decapping activity that is regulated by mRNA splicing

EWS::FLI1 requires a narrow optimum of LCD-LCD interactions to activate transcription

Combinatorial screening identified 20 drugs that synergize with remdesivir against SARS-CoV-2, including HCV inhibitors

Raptor downregulation represents a promising potential therapeutic intervention for the neurological manifestations of TSC

Supervised learning model predicts protein adsorption to carbon nanotubes

Discovery of a Functional Covalent Ligand Targeting an Intrinsically Disordered Cysteine within MYC

Structural basis and regulation of the reductive stress response

Mechanistic basis for ubiquitin modulation of a protein energy landscape

NPC1-mTORC1 Signaling Couples Cholesterol Sensing to Organelle Homeostasis and Is a Targetable Pathway in Niemann-Pick Type C

Viral vectors in this paper expand the toolbox of vectors available for primate retina

Gene expression and cell identity controlled by anaphase-promoting complex

Dimerization quality control ensures neuronal development and survival

The CoQ oxidoreductase FSP1 acts parallel to GPX4 to inhibit ferroptosis

Structural mechanism of a Rag GTPase activation checkpoint by the lysosomal folliculin complex

Advancing breakthrough therapeutics through the purposeful integration of engineering and science

Revolutionizing disease therapy with gene editing

Pioneering targeted protein degradation platforms for eliminating disease causing proteins

Combining chemoproteomic technologies with AI to drug the undruggable

Co-opting physiologically relevant protein homeostatic mechanisms for treating disease

Using the Deubiquitinase Targeting Chimera (DUBTAC) platform to restore the levels of aberrantly degraded proteins that cause disease

A clinical-stage biopharmaceutical company focused on developing transformative genome-edited allogeneic cell therapies for devastating human diseases

Scribe is rewriting medicine with molecular engineering

Developing in vivo gene editing therapeutics to transform patient lives

New gene therapy delivery technologies