Current Members

Pre-Clinical Research Member

Cabaletta Bio

Dante Labs

EdiGENE

gMendel

Jaguar Gene Therapy

Neurogene

Orna Therapeutics, Inc

Praxis Precision Medicine

Shape Therapeutics

StrideBio

Taysha Gene Therapies

Vigil Neurosciences

Clinical Development Member

Acceleron Pharma

Acer Therapeutics

Aeglea BioTherapeutics

Apellis Pharmaceuticals

Applied Therapeutics

argenx

Ascendis Pharma

Asklepion Pharmaceuticals, LLC

Atara Biotherapeutics

Avadel Pharmaceuticals

AVROBIO

bluebirdbio

Blueprint Medicines

BridgeBio

Camurus

Cellectar

ChemoCentryx

CRISPR Therapeutics

Cytokinetics, Inc.

Dicerna

Editas Medicine

Enzyvant

Exicure

Helixmith

Insmed Incorporated

Kezar Life Sciences, Inc.

Kiniksa

Larimar Therapeutics

LEO Pharma

Lysogene

MeiraGTx

Mereo BioPharma

Moderna Therapeutics

Neurocrine Biosciences

Orchard Therapeutics Ltd.

Orphazyme

Ovid Therapeautics

Passage Bio

PharmaEssentia

Prevail Therapeutics

Rallybio

REGENXBIO

Rocket Pharmaceuticals, Inc

Sangamo Therapeutics

Saniona

Santen

Savara

Sigilon Therapeutics

Soligenix, Inc.

uniQure

Viela Bio

Wave Life Sciences

X4 Pharmaceuticals

Zealand Pharma

Approved Product Member

AbbVie Pharmaceuticals

Acadia Pharmaceuticals

Agios Pharmaceuticals

Alexion Pharmaceuticals, Inc.

Alnylam Pharmaceuticals

Amgen

Amicus Therapeutics, Inc.

Amryt Pharma

Astellas

AstraZeneca

BioCryst

Biogen

BioMarin Pharmaceuticals, Inc.

Boehringer-Ingelheim Pharmaceuticals

Bristol-Myers-Squibb

Catalyst Pharmaceuticals

Chiesi Rare Diseases

CSL Behring

Daiichi Sankyo

Deciphera

Dompé

Eiger BioPharmaceuticals

Foundation Medicine

Genentech, Inc.

GlaxoSmithKline PLC

Global Blood Therapeutics, Inc.

Greenwich Biosciences, Inc

Harmony Biosciences, LLC

Horizon Therapeutics

Incyte Corporation

Ionis Pharmaceuticals

Ipsen

Janssen Pharmaceuticals

Mallinckrodt Pharmaceuticals

Novartis

Otsuka America Pharmaceutical, Inc.

Pfizer, Inc.

PTC Therapeutics

Recordati Rare Diseases

Regeneron

Rigel Pharmaceuticals

Sanofi Genzyme

Sarepta Therapeutics

Sobi

Spark Therapeutics

Stemline Therapeutics

Strongbridge Biopharma

Takeda Oncology

Travere Therapeutics, Inc.

UCB Pharma

Ultragenyx

Vanda Pharmaceuticals Inc.

Vertex Pharmaceuticals Worldwide

Zogenix, Inc.

Business Member

Biotechnology Industry Organization (BIO)

Eversana

HPS Group LLC.

ICON

Illumina

Medidata

Optum Frontier Therapies

Parexel

PhRMA

Syneos Health