Access our full report to unlock insights into the advancements targeting dark genome and discover why Pharma giants are investing billions in this field.
In one month, dark genome startups have attracted over $1B worth of investments, emphasizing the field’s promise. Dark genome projects aim to discover new therapeutic targets to drastically widen the therapeutic options for patients across the board.
Beyond the Human Genome Project:
The Human Genome Project, completed in 2003, was supposed to give us the full blueprint of life, but instead, it revealed something curious. Only about 1-2% of our genome codes for the proteins are responsible for the heavy lifting in human biology. The rest—an overwhelming 98%—was long dismissed as non-functional and irrelevant to study in the context of human health and disease.
Over the last decade, this notion has been turned on its head. This non-coding DNA, often referred to as the “dark genome,” is not the biological wasteland we once imagined. Instead, these regions seem to play crucial roles in regulating, orchestrating, and fine-tuning the functions of our genes.
Today, the dark genome is proving to be a fertile ground for scientific discovery, holding tantalizing clues that could reshape our understanding of human biology and disease (figure 1).
Type: Oligonucleotide vaccine
Name: CAMYO-01
Target: LncRNAs
Disease Area: Colorectal
Cancer (CRC)
Type: Small Molecules
Name: n/a
Target: lncRNA-RBP interaction
Disease Area: Fibrotic Diseases
Deals: $500M partnership with Bayer
Type: Antisense Oligonucleotides (ASO)
Name: HTX-001
Target: LncRNAs
Disease Area: Fibrotic Diseases
Deals: $1B partnership with Eli Lilly
Type: Small Molecules
Name: RPT-A
Target: LINE-1RT
Disease Area: Autoimmune
Diseases
Type: Small Molecules
Name: TPN-101
Target: LINE-1RT
Disease Area: Neurodegenerative
Disorders
Deals: Licensed from Oncolys & repurposed
Type: Antisense Oligonucleotides (ASO)
Name: IPT001
Target: LncRNAs
Disease Area: Solid tumors
Type: Oligonucleotide vaccine
Name: EVX-03
Target: LncRNAs
Disease Area: Solid tumors
Type: Oligonucleotide vaccine
Name: IPT-001
Target: Human Endogenous Retrovirus
Disease Area: Solid tumors
Type: Antisense Oligonucleotides (ASO)
Name: FTX-001
Target: LncRNA MALAT-1
Disease Area: Solid tumors
Type: Monoclonal antibody
Name: Temelimab
Target: HERV- K Env protein
Disease Area: Autoimmune
Diseases
Pharma invests heavily in dark genome-related projects:
For pharmaceutical companies, the dark genome represents a largely untapped opportunity for drug discovery and therapeutic intervention. This is echoed by the nearly $2B investment from big pharma (figure 2) into various dark genome drug discovery projects. In September 2024, pharma giants Eli Lilly and Bayer effectively tripled the total investment budget in two separate deals between Lilly and HAYA therapeutics (up to $1B in milestone payments) and a $547M investment deal between Bayer and NextRNA.
Figure 2. Investment surge: Big Pharma’s $1B+ deals signal a major shift towards dark genome innovation.
So what is driving these recent major investments into dark genome drug discovery, and why should pharma/biotech companies be interested in this nascent field in drug discovery?
In our extended report (download for free here), we analyzed the overall technological innovations and the advancement of clinical assets that target the dark genome and highlight areas of opportunity.
In conclusion, we argue that two major factors are causing the uptick in interest and investment in the dark genome:
a. The readiness and culmination of combined technological advancements:
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- Advanced AI-driven data analysis (e.g., generative models) and the availability of bigger datasets help unlock insights buried in the complexities of the dark genome.
- RNA production platforms, pushed by the mRNA vaccines during the pandemic, allow for the scalable production of human therapeutics.
- Gene-editing platforms such as CRISPR or alternative molecular techniques can modify dark genome elements to tweak expression levels.
b. Early indicators of preclinical success for several drug candidates that target dark genome elements. This serves as a positive feedback loop for investors and further enables scientific progress leveraging upcoming technological platforms as the ones described above.
