What Atalanta Therapeutics does

Atalanta Therapeutics is a clinical-stage biotechnology company founded in 2018 and headquartered in Boston, Massachusetts, that develops RNA interference (RNAi) therapeutics for intractable central nervous system (CNS) diseases. The company is built around a proprietary divalent small interfering RNA (di-siRNA) platform that enables durable, selective gene silencing throughout the brain and spinal cord, including deep brain structures that conventional oligonucleotide approaches have struggled to access. Its wholly-owned pipeline comprises ATL-101 (targeting HTT for Huntington's disease), ATL-201 (targeting KCNT1 for KCNT1-related epilepsy in infants and children), ATL-301 (targeting NAV1.7 for severe chronic pain), an MSH3 program for Huntington's disease and related CNS disorders, and an undisclosed CNS disorders program; partnered programs are advanced through a strategic collaboration with Genentech, and an original strategic collaboration was also established with Biogen at founding.

The company was founded by Anastasia Khvorova, Craig Mello, and Neil Aronin of the RNA Therapeutics Institute at the University of Massachusetts Medical School, with Series A funding exclusively from F-Prime Capital. Subsequent leadership and capital events — including a $97 million oversubscribed Series B co-led by EQT Life Sciences and Sanofi Ventures in January 2025, and a June 2025 transition that appointed Joanne Kotz as CEO and Doug Pagán as CFO & COO — brought total capital generated to $262 million and oriented the company toward Phase 1 clinical execution, with IND submissions for ATL-201 and ATL-101 planned in 2025. The organization has 51-100 employees and operates from a single Boston headquarters.

Atalanta's business model is pre-commercial: it does not yet sell pharmaceutical products and is funded entirely by equity financings and partnership economics. Future revenue is expected to flow from sales of approved RNAi therapeutics, supplemented by partnership and licensing structures, with planned Phase 1 trials in KCNT1-related epilepsy and Huntington's disease serving as the next clinical milestones on the path to commercialization.

Name

Atalanta Therapeutics

Legal name

Atalanta Therapeutics

Website

https://atalantatx.com

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

51–100 employees

Short description

Atalanta Therapeutics is a clinical-stage biotechnology company developing RNAi therapies using its proprietary di-siRNA platform to treat intractable CNS diseases, including Huntington's disease, KCNT1-related epilepsy, and severe chronic pain.

Ownership category

akta.pro rank

Product category

Neurological Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG)

akta.pro secondary industry

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH)

Keywords

• RNAi therapeutics
• Neurodegenerative disease treatments
• CNS gene silencing
• Oligonucleotide drug development
• Clinical-stage biotechnology

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. Pharmaceutical Product Revenue: Pre-commercial stage company developing RNAi therapeutics for neurological diseases. Revenue will be generated from sales of approved pharmaceutical products following successful clinical trials and regulatory approval.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Atalanta Therapeutics is a clinical-stage biotechnology company developing RNA interference (RNAi) therapies for intractable neurological diseases. The company leverages a proprietary divalent small interfering RNA (di-siRNA) platform that enables durable, selective gene silencing throughout the brain and spinal cord, including deep brain structures. Its wholly-owned pipeline includes ATL-101 for Huntington's disease, ATL-201 for KCNT1-related epilepsy, ATL-301 for severe chronic pain, an MSH3 program, and an undisclosed CNS disorders program, with partnered programs advanced through a strategic collaboration with Genentech.

Product overview

Atalanta Therapeutics is a biotechnology company pioneering RNA interference (RNAi) therapies for intractable neurological diseases. The company's core offering is its proprietary di-siRNA (divalent small interfering RNA) platform, which enables durable, selective gene silencing throughout the brain and spinal cord. The platform supports a wholly-owned pipeline of disease-modifying programs including ATL-101 (HTT) for Huntington's disease in IND-enabling stage, ATL-201 for KCNT1-related epilepsy in pre-clinical stage with IND submission planned for 2025, ATL-301 (NAV 1.7) for pain in pre-clinical stage, an MSH3 program in discovery stage, and an undisclosed CNS disorders program. The company also advances partnered programs through a strategic collaboration with Genentech.

Differentiator

Problem solved

Functional benefit

Products and services

• ATL-101 (HTT) A di-siRNA therapy designed to silence the HTT gene for the treatment of Huntington's disease, a progressive neurodegenerative disease caused by expansion of the huntingtin gene. Currently in IND-enabling stage with IND submission planned for 2025.
• ATL-201 An investigational RNAi therapy designed to reduce KCNT1 levels and normalize neuronal excitability for the treatment of KCNT1-related epilepsy, a rare severe seizure disorder affecting infants and children. Currently in pre-clinical stage with IND submission planned for 2025.
• ATL-301 (NAV 1.7) A di-siRNA therapy targeting Nav1.7 for the treatment of multiple indications related to severe chronic pain, currently in pre-clinical stage.
• MSH3 Program A discovery-stage program targeting MSH3 for Huntington's disease and related CNS disorders.
• Undisclosed CNS Disorders Program A discovery-stage program targeting undisclosed pathways for multiple CNS disorder indications.

Quantifiable outcome

• Single dose of ATL-201 produced complete seizure suppression up to four months, with therapeutic effects persisting out to six months in KCNT1 epilepsy mouse models
• +2 more outcomes

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Two partnerships are on record, tiered core.

• GenentechcoreStrategic or Co-development Partner · 1 January 2018Strategic collaboration with Genentech as part of Atalanta's partnered programs. Atalanta is advancing partnered programs as part of a strategic collaboration with Genentech for the development of di-siRNA therapeutics for CNS diseases.
• BiogencoreStrategic or Co-development Partner · 1 January 2018Strategic collaboration with Biogen established at company founding in 2018 as part of Atalanta's initial strategic collaborations alongside Genentech.

Scale indicators4 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Wave Life Sciences: Wave Life Sciences develops stereopure oligonucleotide therapeutics for CNS diseases, with an active Huntington's disease program using allele-selective approaches. Most direct comparable: same therapeutic area (CNS), overlapping indication (HD), and parallel nucleic-acid-based modality against neurological targets.
• Stoke Therapeutics: Stoke Therapeutics uses antisense oligonucleotides (ASOs) for genetic CNS diseases, with lead programs in Dravet syndrome and autosomal dominant Alzheimer disease. Directly comparable in stage, modality class (oligonucleotide), and CNS rare-disease focus.
• Arrowhead Pharmaceuticals: Arrowhead develops RNAi-based therapeutics via its TRiM platform with expanding extra-hepatic delivery capabilities, including CNS targets. Closest platform competitor in RNAi modality pursuing broad delivery beyond the liver.
• Denali Therapeutics: Denali Therapeutics is a clinical-stage biotech focused on neurodegeneration, with programs in Huntington's disease, ALS, and Alzheimer's. Comparable CNS-focused biotech at similar stage, though using small molecules and biologics rather than RNAi.

Broad incumbents

• Alnylam Pharmaceuticals: Alnylam is the established leader in RNAi therapeutics with multiple approved products and expanding CNS programs. Larger, commercially validated incumbent whose platform progress and CNS pipeline expansions directly influence Atalanta's competitive environment.
• Ionis Pharmaceuticals: Ionis is the leader in antisense oligonucleotide (ASO) therapeutics with a deep neurology pipeline including approved therapies for spinal muscular atrophy and amyotrophic lateral sclerosis. Adjacent modality (ASO vs siRNA) targeting overlapping CNS indications.
• Biogen: Biogen is a large commercial-stage biotech with deep CNS/neurology franchise, including antisense oligonucleotide Spinraza. Strategic partner and founding collaborator of Atalanta, providing both validation and a potential acquirer with relevant CNS commercial infrastructure.

Emerging players

• ProQR Therapeutics: ProQR develops RNA-based therapies, including antisense oligonucleotides and RNA editing, for genetic diseases including CNS indications. Smaller, emerging RNA-therapeutics company with overlapping rare-disease focus.
• Sage Therapeutics: Sage Therapeutics develops CNS-focused therapeutics, with lead asset Zurzuvae approved for postpartum depression and pipeline in epilepsy and neurodegeneration. Comparable clinical-stage CNS biotech with similar neurology-focused strategy.

Others

• Voyager Therapeutics: Voyager Therapeutics develops gene therapies for CNS diseases including Huntington's disease and ALS. Adjacent modality (AAV-based gene therapy vs RNAi) but overlapping CNS indication space and similar stage.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xtwitter.com/atalantatx
• LinkedInlinkedin.com/company/atalanta-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds2 records

Investors9 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Atalanta Therapeutics