Mendelian
Targeted Oncology
Gene Therapy

Mendelian

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
BBP-870 (Origin)

Synthetic cPMP for MOCD Type A

patients

100

disease

Molybdenum cofactor deficiency (MocD) Type A

genetic source

MOCS1

BBP-265 (Eidos)

TTR Stabilizer (AG10) for ATTR-CM

patients

>400,000

disease

ATTR-CM, ATTR-PN

genetic source

TTR (transthyretin)

BBP-009 (PellePharm)

Topical HH Inhibitor (Patidegib) for Gorlin Syndrome

patients

120,000

disease

Gorlin syndrome, Frequent basal cell carcinoma

genetic source

PTCH1 (patched-1, a smoothened inhibitor)

BBP-589 (PTR)

Recombinant Collagen 7 for rDEB

patients

1,500

disease

Dystrophic Epidermolysis Bullosa (DEB)

genetic source

COL7A1 (collagen 7)

BBP-831 (QED)

FGFR 1-3 Inhibitor (Infigratinib) for Achondroplasia

patients

55,000

disease

Achondroplasia

genetic source

FGFR

BBP-671 (CoA)

PanK Activator for PKAN & Organic Acidemias

patients

7,000

disease

Pantothenate kinase-associated neurodegeneration (PKAN), CASTOR

genetic source

PANK2 (pantothenate kinase)

BBP-681 (Venthera)

Topical PI3Ka Inhibitor for VM & LM

patients

117,000

disease

Venous Malformations

genetic source

PI3Ka

BBP-561 (MoST)

KLK 5/7 Inhibitor for Netherton Syndrome

patients

11,000

disease

Netherton

genetic source

SPINK5 (LEKT1, a kallikrein 5/7/14 inhibitor)

BBP-711 (Orfan)

GO Inhibitor for Primary Hyperoxaluria 1

patients

5,000 / 1.5M

disease

Primary Hyperoxaluria Type 1

genetic source

AGXT

BBP-761 (Fortify)

Succinate Pro-drug for LHON

patients

20,000

disease

Leber’s hereditary optic neuropathy

genetic source

NADH Dehydrogenase (Complex I)

Targeted Oncology

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
BBP-831 (QED)

FGFR 1-3 Inhibitor (Infigratinib) for FGFR+ Cancers

patients

37,000

disease

Fibroblast Growth Factor Receptor

genetic source

FGFR

BBP-454 (TheRas)

KRAS Inhibitor for KRAS Cancers

patients

>500,000

disease

KRAS+ cancers

genetic source

KRAS

BBP-398 (Navire)

SHP2 Inhibitor for RTK Cancers

patients

>500,000

disease

Receptor Tyrosine Kinase Cancers

genetic source

PTPN11 (SHP2) and MAPK pathway

BBP-954 (Ferro)

GPX4 Inhibitor for Multiple Tumors

patients

>500,000

disease

Multiple Tumors

genetic source

--

Gene Therapy

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
BBP-631 (Adrenas)

Gene Therapy for CAH

patients

>75,000

disease

Congenital Adrenal Hyperplasia

genetic source

CYP21A2

BBP-812 (Aspa)

Gene Therapy for Canavan Disease

patients

1,000

disease

Canavan Disease

genetic source

ASPA

the
pipeline

Every investigational drug in our pipeline represents hope for an important segment of patients in need of a treatment. By targeting the known drivers of genetic diseases, we are applying precision medicine techniques to develop drugs that show promise of becoming safe, effective therapies. Visit publications for selected literature about each program.