We exist to bring meaningful medicines to patients quickly & safely.

Pipeline

Our late-stage investigational pipeline is designed with purpose.

Acoramidis

Early-Stage Variant Transthyretin Amyloidosis (ATTRv)

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

BBP-418

Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

Encaleret

Autosomal Dominant Hypocalcemia Type 1 (ADH1)

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

Chronic Hypoparathyroidism-NIH IIR

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

Infigratinib

Achondroplasia

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

Hypochondroplasia

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

BBP-812

Canavan Disease

  1. Preclinical
  2. Phase 1
  3. Phase 2
  4. Phase 3
  5. Approved

Latest News

Explore new developments and the impact of our work.

$8B

$8 billion peak year sales potential1

3 FDA

3 FDA approvals received

70+

70+ published peer reviewed papers

Hope through rigorous science

Explore conditions, clinical trials, and the latest research.

Listen now on YouTube

On Rare Podcast

Hear from people living with genetic conditions.

Braelyn, living with Juvenile myelomonocytic leukemia (JMML)

Patient Stories

Meet the people who motivate us.

Roll Up Your Sleeves

Join a team with the ambition of creating life-changing medicines.

References