Remix Therapeutics

Expanding the Druggable Space

Remix™ discovers and develops small molecules that modulate RNA processing to control gene and protein expression, allowing us to advance novel therapies. Remix’s proprietary drug discovery platform, REMaster™, identifies druggable target sites and active chemical matter bringing the promise of new medicines to patients.
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Our Unique Rhythm

  • Deep expertise in data science and bioinformatics has identified novel target sites that are druggable via modulation of RNA processing.
  • Multiplexed high-throughput screening technologies that simultaneously screen dozens of novel target sites at an unprecedented scale.
  • A purpose-built chemical library enriched for compounds that can interact with RNA processing mechanisms.
  • Clinical development programs designed by industry experts and informed by patients and clinicians to advance relevant and impactful therapies.
RNA Processing

Altering Gene Expression

RNA processing is a tightly regulated mechanism of gene transcription, 5’ end capping, polyadenylation, alternative splicing and protein expression. In disease states, this machinery can become dysregulated through genetic and non-genetic means. Remix's proprietary REMaster™ platform has been built to have visibility into multiple steps of the RNA processing cascade for target discovery and modulation.
The Platform

Our Three Scientific Pillars

Data Science
Data Science
Data Science

Identifying druggable high value targets

Our teams have built an extensive transcriptome database that we leverage using machine learning to identify modulatable sites across diverse target areas. To date, the Remix™ team has identified more than 1.6M novel exon targets in the mRNA degradation space alone - 25-fold more than the largest public database. These exon targets are present in dozens of previously undrugged high value targets.
Biology & Biomolecular Sciences
Biology & Biomolecular Sciences
Biology & Biomolecular Sciences

Functional target validation and multiplexed high-throughput screens

Remix™ has developed a proprietary genetic toolkit to validate that novel exon targets can be modulated in cells and lead to functional outcomes on gene and protein expression. The validation process ensures biologically meaningful targets are selected for progression into high-throughput screening campaigns. Multiplexed cell-based screening assays are deployed against dozens of targets in parallel, identifying novel compounds with high specificity and selectivity.
Medicinal & Computational Chemistry
Medicinal & Computational Chemistry
Medicinal & Computational Chemistry

Proprietary libraries and computational approaches

Our extensive proprietary chemical library samples distinct regions of chemical space and continues to grow over time. Cutting-edge computational chemistry analysis has ensured the library diversity is sufficient for identification of lead chemical matter for drug discovery programs.
Turning Targets into Therapies

Designing Molecules for Unmet Patient Needs

Determined to deliver novel and impactful treatments, Remix™ sets about advancing our breakthrough discoveries into innovative and relevant medicines. Our first act is tackling previously undrugged oncogenic transcription factors to eradicate certain solid tumors and hematological malignancies. Next up, breakthrough science to deliver for patients with other malignancies and neurodegenerative diseases.

Pipeline

Broad pipeline of RNA processing modulators across Oncology and Neurodegeneration
Target / Indications
Discovery
Pre-Clinical
Clinical
Rights
MYB/REM-422
Therapeutic Area: Adenoid Cystic Carcinoma
clinical
MYB is an undruggable transcription factor that is known to be an oncogenic driver of multiple solid tumors and hematological malignancies, such as adenoid cystic carcinoma (ACC) and acute myeloid leukemia/myelodysplastic syndromes (AML/MDS). REM-422 is a potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and protein expression resulting in antitumor activity in MYB-dependent human tumor models. REM-422 is currently in Phase 1 clinical trials (NCT06118086).
Therapeutic Area: Acute Myeloid Leukemia/Myelodysplastic Syndromes (AML/MDS)
clinical
MYB is an undruggable transcription factor that is known to be an oncogenic driver of multiple solid tumors and hematological malignancies, such as adenoid cystic carcinoma (ACC) and acute myeloid leukemia/myelodysplastic syndromes (AML/MDS). REM-422 is a potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and protein expression resulting in antitumor activity in MYB-dependent human tumor models. REM-422 is currently in Phase 1 clinical trials (NCT06297941).
Therapeutic Area: Additional Cancers
pre-clinical
MYB is an undruggable transcription factor that is known to be an oncogenic driver of multiple solid tumors and hematological malignancies, such as adenoid cystic carcinoma (ACC) and acute myeloid leukemia/myelodysplastic syndromes (AML/MDS). REM-422 is a potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and protein expression resulting in antitumor activity in MYB-dependent human tumor models. REM-422 is currently in Phase 1 clinical trials (NCT06297941).
Genetic Driver
Therapeutic Area: Amplified & Fusion+ve Cancers
pre-clinical
Genetic Driver
Therapeutic Area: Multiple Dependent Cancers
pre-clinical
Additional Targets
Therapeutic Area: Oncology and CNS
pre-clinical
Multiple Targets (J&J)
Therapeutic Area: Oncology & Immunology
discovery
Multiple Targets (Roche)
Therapeutic Area: Various
discovery