Company Sector

Rare Disease

17 companies tagged with Rare Disease.

Rare Disease groups companies developing therapies for smaller patient populations with high unmet need. These businesses are often built around genetically defined disorders, orphan indications, and targeted commercial strategies with highly specialized clinical programs.

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ALXN

ALXN is a biopharmaceutical company focused on advancing a pipeline of therapeutic programs. Investors usually track how clinical data, regulatory milestones, and launch execution change the story over time.

Biopharma Oncology Rare Disease

AMGN

Amgen Inc.

Amgen is a large-cap biopharmaceutical company with a commercial base spanning oncology, inflammation, bone health, cardiovascular disease, and rare disease. Investors follow the stock for the mix of marketed products, late-stage pipeline optionality, and recurring regulatory or launch milestones.

Biopharma Novel Therapies Gene Therapy Rare Disease Gene Therapy

BCRX

BioCryst Pharmaceuticals

BioCryst Pharmaceuticals is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Pharmaceuticals Small Molecule Drugs Rare Disease

CRNX

Crinetics Pharmaceuticals

Crinetics Pharmaceuticals is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

EDIT

Editas Medicine

Editas Medicine is a biopharmaceutical company focused on advancing a pipeline of therapeutic programs. Investors usually track how clinical data, regulatory milestones, and launch execution change the story over time.

Biopharma Rare Disease

ETON

Eton Pharmaceuticals

Eton Pharmaceuticals is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

GILD

Gilead Sciences

Gilead Sciences is a large-cap biopharmaceutical company with a commercial base anchored by antivirals and a growing set of oncology and inflammation programs. Investors track the balance between cash flow strength, launch execution, and pipeline replacement.

Biopharma Biologics Novel Therapies Large Molecule Drugs

MRNA

Moderna, Inc.

Moderna is an mRNA medicines company that built its platform around vaccine science and is now broadening into infectious disease, oncology, and other therapeutic areas. Investors watch how the company converts platform credibility into a wider commercial story.

Biopharma Biologics Vaccines mRNA Vaccines

RLYB

Rallybio

Rallybio is a biopharmaceutical company focused on advancing a pipeline of therapeutic programs. Investors usually track how clinical data, regulatory milestones, and launch execution change the story over time.

Biopharma Rare Disease

REGN

Regeneron Pharmaceuticals

Regeneron Pharmaceuticals is a biologics-driven biopharmaceutical company built around antibody science, specialty-market execution, and a durable R&D engine. Investors watch both the commercial base and the next wave of pipeline readouts.

Biopharma Biologics Large Molecule Drugs Antibody Drugs

RCKT

Rocket Pharmaceuticals

Rocket Pharmaceuticals is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

SRPT

Sarepta Therapeutics

Sarepta Therapeutics is a genetic medicines company focused on neuromuscular disease, with investors watching how its RNA and gene-therapy programs translate into durable clinical and commercial value.

Biopharma Biologics Diagnostics Novel Therapies

SEEL

Seelos Therapeutics, Inc.

Seelos Therapeutics is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

SLNO

Soleno Therapeutics

Soleno Therapeutics is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

TVTX

Travere Therapeutics

Travere Therapeutics is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

RARE

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical is a biopharmaceutical company focused on pipeline execution and catalyst-driven growth, with investors tracking how data, regulatory milestones, and launch timing reshape the investment case.

Biopharma Rare Disease

VECT

VectivBio

VectivBio is a biopharmaceutical company focused on advancing a pipeline of therapeutic programs. Investors usually track how clinical data, regulatory milestones, and launch execution change the story over time.

Biopharma Rare Disease