Organizations

Find NF organizations moving research, care, and community forward.

Explore charities, nonprofits, patient organizations, collaborative networks, and companies contributing to NF research and treatment development.

Advancing NF care & research

Companies advancing NF care, research, and treatment

Medical, pharmaceutical, biotechnology, and genetic-testing companies with a direct, publicly identified role in NF care, research, diagnosis, treatment development, or an NF-related clinical program. The labels distinguish approved treatments, clinical and early-stage research, and diagnostic services.

Filter companiesSearch by company, condition, treatment, research stage, or technology.
22 results
Approved NF treatment

Alexion, AstraZeneca Rare Disease

NF1 · Plexiform neurofibromas

Develops and provides Koselugo (selumetinib), an approved MEK-inhibitor treatment for eligible people with NF1-associated plexiform neurofibromas, while continuing NF research and patient-support work.

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Genetic testing and diagnostics

Ambry Genetics

NF1 · NF2-related schwannomatosis · Other schwannomatoses

Provides clinical genetic testing and variant analysis for NF1 and schwannomatosis-related genes to support diagnosis and care planning.

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Preclinical genetic medicine

CureAge Therapeutics

NF1 · Underlying disease biology

A CTF- and Deep Science Ventures-founded biotechnology company developing genetic medicines intended to reach Schwann cells and address the underlying biology of NF1.

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Genetic testing and diagnostics

GeneDx

NF1 · NF2-related schwannomatosis · Other schwannomatoses

Provides molecular genetic testing and variant interpretation that can help identify NF1 and schwannomatosis-related genetic changes.

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Discovery research

GondolaBio

NF1 · Undisclosed program

Lists an NF1 therapeutic program in its genetic-disease pipeline. The candidate and specific NF1 manifestation have not yet been publicly disclosed.

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Clinical development · Phase 2

Healx

NF1 · Plexiform neurofibromas

Uses an AI-supported drug-discovery platform to develop HLX-1502 and is studying the candidate in the INSPIRE-NF1 clinical trial.

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Clinical research

Incyte

NF2-related schwannomatosis

Provided a compound for a CTF-supported combination-treatment arm within the INTUITT-NF2 platform trial.

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Preclinical drug discovery

iNFixion Bioscience

NF1 · Neurofibromin restoration

Develops mutation-agnostic approaches intended to increase functional neurofibromin and address the root biological driver of NF1.

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Genetic testing and diagnostics

Labcorp / Invitae

NF1 · NF2-related schwannomatosis · Other schwannomatoses

Offers hereditary nervous-system tumor testing that includes NF1 and schwannomatosis-related genes, along with clinical interpretation services.

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Approved NF treatment

Merck KGaA / SpringWorks Therapeutics

NF1 · Plexiform neurofibromas

Developed Gomekli/Ezmekly (mirdametinib), an approved MEK-inhibitor treatment for eligible adults and children with NF1-associated plexiform neurofibromas.

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Preclinical gene therapy

Merlin Therapeutics

NF2-related schwannomatosis · Schwannomas

Develops MRL-102, a gene-replacement approach intended to restore functional merlin in schwannomas.

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Preclinical · Approaching clinical study

Mulberry Biotherapeutics

NF2-related schwannomatosis

Develops MUL001, an investigational bacteria-mediated immunotherapy delivered directly into tumors, initially for NF2-related schwannomatosis.

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Early-stage therapeutic research

NF2 Therapeutics, Inc.

NF2-related schwannomatosis

An NF2-focused company developing an AAV gene therapy for vestibular schwannomas and an antibody-drug conjugate for meningiomas.

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Clinical development

NFlection Therapeutics

NF1 · Cutaneous neurofibromas

Develops NFX-179, an investigational topical MEK-inhibitor gel studied for cutaneous neurofibromas in NF1.

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NF-related clinical research

Novartis

NF1 · Tumor manifestations

Its MEK inhibitor trametinib has been studied in investigator-led NF1 research, including plexiform neurofibromas and low-grade gliomas.

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Early clinical development

Pasithea Therapeutics

NF1 · RAS/MAPK-driven tumors

Is evaluating PAS-004, an investigational MEK inhibitor with potential relevance to NF1-associated tumors.

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Drug-development contributor

Pfizer

NF1 · Mirdametinib origins

Originated the mirdametinib program and helped establish SpringWorks Therapeutics, supporting the path that ultimately produced an approved NF1 treatment.

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Genetic testing and diagnostics

PreventionGenetics, part of Exact Sciences

NF1 · NF2-related schwannomatosis · Other schwannomatoses

Provides clinical genetic testing for NF1 and schwannomatosis-related genes, including single-gene and multigene testing options.

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Early clinical development

PRG Science & Technology

NF2-related schwannomatosis

Develops PRG-N-01 (trineumin), an investigational therapy intended to increase tumor-suppressor activity in NF2-related schwannomatosis.

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Early clinical gene therapy

Santo Therapeutics

NF2-related schwannomatosis

Develops ST002, an investigational in-vivo gene therapy designed to restore NF2/merlin function in NF2 mutation-driven tumors.

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Clinical research · Drug repurposing

Takeda Pharmaceutical Company

NF2-related schwannomatosis

Partnered with CTF on the INTUITT-NF2 platform trial studying brigatinib and other potential treatments for NF2-related schwannomatosis.

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NF-related clinical research

Vivace Therapeutics

NF2-altered tumors · Hippo pathway

Develops therapies targeting the Hippo pathway, including clinical research in tumors with NF2 alterations. This work is broader than inherited NF2-related schwannomatosis.

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