Biopharma RNAi Drug Developers sector
Strategic acquirers, private equity (buyout funds and growth funds) firms, and valuation benchmarks for Biopharma RNAi Drug Developers
1.1 - About Biopharma RNAi Drug Developers sector
Companies in the Biopharma RNAi Drug Developers category design and advance RNA interference therapeutics that selectively silence disease-causing genes. They combine proprietary delivery technologies with optimized siRNA constructs to achieve durable, tissue-targeted effects and reduced off‑target toxicity. Their value proposition centers on clinically validated gene silencing, scalable manufacturing of oligonucleotides, and pipelines spanning rare, metabolic, and hepatic indications.
Typical capabilities include target discovery and validation using functional genomics and bioinformatics, design and optimization of chemically modified siRNA, and GalNAc conjugation for hepatocyte targeting. Many engineer lipid nanoparticle formulations for extrahepatic delivery and develop shRNA vectors where appropriate. Integrated preclinical pharmacology, toxicology, and IND‑enabling studies are coupled with GMP oligonucleotide synthesis, CMC and analytical development, biomarker assay design, and regulatory strategy support.
Primary customer segments include global pharmaceutical companies pursuing platform acquisitions and licensing, clinical‑stage biotech firms seeking co‑development partners, and hospital systems adopting approved gene‑silencing therapies. Outcomes delivered include accelerated pipeline timelines, differentiated assets in rare and metabolic diseases, improved safety profiles through reduced off‑target effects, and scalable GMP manufacturing to support commercialization and geographic expansion.
2. Buyers in the Biopharma RNAi Drug Developers sector
2.1 Top strategic acquirers of Biopharma RNAi Drug Developers companies
Alnylam
- Description: Provider of RNA interference-based biopharmaceutical therapies, discovering, developing and commercializing RNAi drugs for rare genetic and cardiometabolic diseases; its marketed portfolio includes Onpattro for polyneuropathy of hereditary transthyretin-mediated amyloidosis, Givlaari for acute hepatic porphyria, Amvuttra for ATTR amyloidosis-related cardiomyopathy and Oxlumo for primary hyperoxaluria type 1.
- Key Products:
- Onpattro: Lipid-nanoparticle RNAi therapeutic that silences transthyretin gene expression to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis, slowing disease progression and improving neurologic function
- Givlaari: Subcutaneously administered RNAi drug that reduces hepatic ALAS1 mRNA to lower toxic ALA and PBG levels, preventing acute attacks in patients with acute hepatic porphyria
- Amvuttra: RNAi therapy delivering vutrisiran to inhibit transthyretin production, lowering cardiac amyloid deposits and reducing cardiovascular death, hospitalizations and urgent heart-failure visits in adults with ATTR-CM
- Oxlumo: GalNAc-conjugated RNAi agent targeting hepatic LDHA mRNA to decrease oxalate production, lowering urinary and plasma oxalate in patients with primary hyperoxaluria type 1.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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2.2 - Strategic buyer groups for Biopharma RNAi Drug Developers sector
M&A buyer group 1: Gene Therapy
Sarepta Therapeutics
- Type: N/A
- Employees: ●●●●●
- Description: Provider of precision genetic medicines that discover, develop and commercialize gene therapies and exon-skipping RNA treatments for rare neuromuscular diseases, with a core focus on Duchenne muscular dystrophy and a growing pipeline addressing limb-girdle muscular dystrophy and other genetic disorders.
- Key Products:
- ELEVIDYS: FDA-approved one-time gene therapy delivering micro-dystrophin to Duchenne patients
- approved for ambulant children ≥4 yrs and accelerated approval for non-ambulatory, restoring dystrophin function and treating over 80 % of U.S. DMD population
- PMO Franchise (EXONDYS 51
- VYONDYS 53
- AMONDYS 45): proprietary morpholino oligonucleotide exon-skipping drugs that generated $249 M Q3-24 revenue, providing RNA-based therapy options for multiple Duchenne mutations
- SRP-9003 Gene Therapy: AAV-based treatment for Limb-Girdle Muscular Dystrophy type 2E with BLA filing planned 2025, aiming for accelerated approval by leveraging ELEVIDYS data
- siRNA Pipeline (Arrowhead collaboration): multi-program small-interfering RNA platform targeting neuromuscular
- CNS, cardiomyopathy and pulmonary indications to deliver multiple potential blockbuster launches before decade-end.
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●● companies3. Investors and private equity firms in Biopharma RNAi Drug Developers sector
3.1 - Buyout funds in the Biopharma RNAi Drug Developers sector
2.2 - Strategic buyer groups for Biopharma RNAi Drug Developers sector
4 - Top valuation comps for Biopharma RNAi Drug Developers companies
4.2 - Public trading comparable groups for Biopharma RNAi Drug Developers sector
Valuation benchmark group 1: RNA and Peptide Therapeutics Companies
Biogen
- Enterprise value: $●●●m
- Market Cap: $●●●m
- EV/Revenue: ●.●x
- EV/EBITDA: ●●.●x
- Description: Provider of biotechnology solutions focusing on the discovery, development, and delivery of innovative medicines for patients with neurological, autoimmune, and rare diseases, leveraging deep scientific expertise to transform patients' lives and advance health equity in disease treatment.
- Key Products:
- Aducanumab: Alzheimer’s treatment developed through accelerated approval pathway
- Tecfidera: Oral therapy for multiple sclerosis
- Spinraza: Treatment for spinal muscular atrophy (SMA)
- Tysabri: Therapy for relapsing forms of multiple sclerosis
- Avonex: Injectable treatment for relapsing multiple sclerosis.