BBP-870 (Origin) - Synthetic cPMP for MOCD Type A

Schwahn BC & Schwarz G. Lancet. 2015 Nov 14;386(10007):1955-63. doi: 10.1016/S0140-6736(15)00124-5. Epub 2015 Sep 3.

Mechler K & Ries M. Genet Med. 2015 Dec;17(12):965-70. doi: 10.1038/gim.2015.12. Epub 2015 Mar 12.

Schwarz G & Ribbe MW. Nature. 2009 Aug 13;460(7257):839-47. doi: 10.1038/nature08302.

BBP-265 (Eidos) - TTR Stabilizer (AG10) for ATTR-CM

Maurer MS & Rapezzi C. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.

Hanson JLS & Connors LH. Circ Heart Fail. 2018 Feb;11(2):e004000. doi: 10.1161/CIRCHEARTFAILURE.117.004000.

Gillmore JD, Hawkins PN. Circulation. 2016 Jun 14;133(24):2404-12. doi: 10.1161/CIRCULATIONAHA.116.021612. Epub 2016 Apr 22.

Hornstrup LS & Tybjærg-Hansen A. Arterioscler Thromb Vasc Biol. 2013 Jun;33(6):1441-7. doi: 10.1161/ATVBAHA.113.301273. Epub 2013 Apr 11.

Hammarström P & Kelly JW. Proc Natl Acad Sci USA. 2002 Dec 10;99 Suppl 4:16427-32. Epub 2002 Sep 25.

BBP-009 (PellePharm) - Topical HHI (Patidegib) for Gorlin, HF-BCC

Tang JY & Epstein EH Jr. N Engl J Med. 2012 Jun 7;366(23):2180-8. doi: 10.1056/NEJMoa1113538.

BBP-589 (PTR) - Recombinant Collagen 7 for rDEB

Chung HJ & Uitto J. Dermatol Clin. 2010 Jan;28(1):93-105. doi: 10.1016/j.det.2009.10.011.

Chen M & Woodley DT. J Biol Chem. 2002 Jan 18;277(3):2118-24. Epub 2001 Nov 6. DOI: 10.1074/jbc.M108779200

BBP-831 (QED) - FGFR 1-3 Inhibitor (Infigratinib) for Achondroplasia

Ornitz DM & Legeai-Mallet L. Dev Dyn. 2017 Apr;246(4):291-309. doi: 10.1002/dvdy.24479. Epub 2017 Mar 2.

Komla-Ebri D & Legeai-Mallet L. J Clin Invest. 2016 May 2;126(5):1871-84. doi: 10.1172/JCI83926. Epub 2016 Apr 11.

BBP-671 (CoA) - PanK Activator for PKAN & Organic Acidemias

Yang H & Mitchell GA. Biochem Soc Trans. 2019 Feb 28;47(1):149-155. doi: 10.1042/BST20180423. Epub 2019 Jan 9.

Sharma LK & Jackowski S. Nat Commun. 2018 Oct 23;9(1):4399. doi: 10.1038/s41467-018-06703-2.

Hogarth P & Hayflick SJ. Mol Genet Metab. 2017 Mar;120(3):278-287. doi: 10.1016/j.ymgme.2016.11.004. Epub 2016 Dec 27.

Kolker S & Leonard JV. J Inherit Metab Dis. 2006 Dec;29(6):701-4; discussion 705-6. Epub 2006 Oct 14.

BBP-681 (Venthera) - Topical PI3Ka Inhibitor for VM & LM

Venot Q & Canaud G. Nature. 2018 Jun;558(7711):540-546. doi: 10.1038/s41586-018-0217-9. Epub 2018 Jun 13.

Tuncel AT & Kolker S. J Inherit Metab Dis. 2018 Sep;41(5):765-776. doi: 10.1007/s10545-017-0135-2. Epub 2018 Jan 15.

Siegel DH & Drolet BA. J Invest Dermatol. 2018 Apr;138(4):957-967. doi: 10.1016/j.jid.2017.10.033. Epub 2017 Nov 22.

Castel P & Baselga J. Sci Transl Med. 2016 Mar 30;8(332):332ra42. doi: 10.1126/scitranslmed.aaf1164.

BBP-561 (MoST) - KLK 5/7 Inhibitor for Netherton Syndrome

Kasparek P. & Sedlacek R. PLoS Genet. 2017 Jan 17;13(1):e1006566. doi: 10.1371/journal.pgen.1006566. eCollection 2017 Jan.

Furio L. & Hovanian A. PLoS Genet. 2015 Sep 21;11(9):e1005389. doi: 10.1371/journal.pgen.1005389. eCollection 2015 Sep.

Hovanian A. Cell Tissue Res. 2013 Feb;351(2):289-300. doi: 10.1007/s00441-013-1558-1. Epub 2013 Jan 24.

Fortugno P & Zambruno G. J Invest Dermatol. 2011 Nov;131(11):2223-32. doi: 10.1038/jid.2011.174. Epub 2011 Jun 23.

Hachem JP & Elias PM. J Invest Dermatol. 2006 Sep;126(9):2074-86. Epub 2006 May 11. DOI: 10.1038/sj.jid.5700351

BBP-711 (Orfan) - GO Inhibitor for Primary Hyperoxaluria 1

Liebow A & Knight J. J Am Soc Nephrol. 2017 Feb;28(2):494-503. doi: 10.1681/ASN.2016030338. Epub 2016 Jul 18.

Martin-Higueras C & Salido E. Mol Ther. 2016 Apr;24(4):719-25. doi: 10.1038/mt.2015.224. Epub 2015 Dec 22.

Cochat P & Rumsby G. N Engl J Med. 2013 Aug 15;369(7):649-58. doi: 10.1056/NEJMra1301564.

BBP-761 (Fortify) - Succinate Pro-drug for LHON

Ehinger JK & Elmér E. Nat Commun. 2016 Aug 9;7:12317. doi: 10.1038/ncomms12317.

Lam Bl & Guy J. JAMA Ophthalmol. 2014 Apr 1;132(4):428-36. doi: 10.1001/jamaophthalmol.2013.7971.

Giordano C & Carelli V. Brain. 2014 Feb;137(Pt 2):335-53. doi: 10.1093/brain/awt343. Epub 2013 Dec 24.

Hawkins BJ & Madesh M. J Biol Chem. 2010 Aug 20;285(34):26494-505. doi: 10.1074/jbc.M110.143164. Epub 2010 Jun 21.

BBP-831 (QED) - FGFR 1-3 Inhibitor (Infigratinib) for FGFR+ Cancers

Pal SK & Bajorin DF. Cancer Discov. 2018 Jul;8(7):812-821. doi: 10.1158/2159-8290.CD-18-0229. Epub 2018 May 30.

Helsten T & Kurzrock R. Clin Cancer Res. 2016 Jan 1;22(1):259-67. doi: 10.1158/1078-0432.CCR-14-3212. Epub 2015 Sep 15.

Lamarca A & Valle JW. Ann Oncol. 2014 Dec;25(12):2328-38. doi: 10.1093/annonc/mdu162. Epub 2014 Apr 25.

BBP-454 (TheRas) - KRAS Inhibitor for KRAS Cancers

Simanshu DK & McCormick F. Cell. Jun 29;170(1):17-33. doi: 10.1016/j.cell.2017.06.009.

Cox AD & Phillips MR. Clin Cancer Res. 2015 Apr 15;21(8):1819-27. doi: 10.1158/1078-0432.CCR-14-3214.

Cox AD & Der CJ. Nat Rev Drug Discov. 2014 Nov;13(11):828-51. doi: 10.1038/nrd4389. Epub 2014 Oct 17.

BBP-398 (Navire) - SHP2 Inhibitor for RTK Cancers

Nichols RJ & Bivona TG. Nat Cell Biol. 2018 Sep;20(9):1064-1073. doi: 10.1038/s41556-018-0169-1. Epub 2018 Aug 13.

Fedele C & Tang KH. Cancer Discov. 2018 Oct;8(10):1237-1249. doi: 10.1158/2159-8290.CD-18-0444. Epub 2018 Jul 25.

Mainardi S & Bernards R. Nat Med. 2018 Jul;24(7):961-967. doi: 10.1038/s41591-018-0023-9. Epub 2018 May 28.

Wong GS & Bass AJ. Nat Med. 2018 Jul;24(7):968-977. doi: 10.1038/s41591-018-0022-x. Epub 2018 May 28.

Chen YN & Fortin PD. Nature. 2016 Jul 7;535(7610):148-52. Epub 2016 Jun 29. DOI: 10.1038/nature18621

BBP-954 (Ferro) - GPX4 Inhibitor for Multiple Tumors

Tsoi J & Graeber TG. Cancer Cell. 2018 May 14;33(5):890-904.e5. doi: 10.1016/j.ccell.2018.03.017. Epub 2018 Apr 12.

Hangauer M & McCormick F. Nature. 2017 Nov 9; 551(7679): 247-250. doi: 10.1038/nature24297. PMID: 29088702

Stockwell BR & Zhang DD. Cell. 2017 Oct 5;171(2):273-285. doi: 10.1016/j.cell.2017.09.021.

Viswanathan VS & Schreiber SL. Nature. 2017 Jul 27;547(7664):453-457. doi: 10.1038/nature23007. Epub 2017 Jul 5.

Dixon SJ & Stockwell BR. Cell. 2012 May 25;149(5):1060-72. doi: 10.1016/j.cell.2012.03.042.

BBP-631 (Adrenas) - Gene Therapy for CAH

Perdomini M & Bougnères P. Gene Ther. 2017 May;24(5):275-281. doi: 10.1038/gt.2017.10. Epub 2017 Feb 6.

Finkielstain GP & Merke DP. J Clin Endocrinol Metab. 2012 Dec;97(12):4429-38. doi: 10.1210/jc.2012-2102. Epub 2012 Sep 18.

Krone N & Arlt W. Best Pract Res Clin Endocrinol Metab. 2009 Apr;23(2):181-92. doi: 10.1016/j.beem.2008.10.014.

Speiser PW & White PC. N Engl J Med. 2003 Aug 21;349(8):776-88. doi: 10.1056/NEJMra021561

Mesiano S & Jaffe RB. Endocr Rev. 1997 Jun;18(3):378-403. DOI: 10.1210/edrv.18.3.0304

BBP-812 (Aspa) - Gene Therapy for Canavan Disease

Gessler DJ & Gao G. JCI Insight. 2017 Feb 9;2(3):e90807. doi: 10.1172/jci.insight.90807.

Ahmed SS & Gao G. Mol Ther. 2016 Jun;24(6):1030-1041. doi: 10.1038/mt.2016.68. Epub 2016 Apr 4.

Ahmed SS & Gao G. Mol Ther. 2013 Dec;21(12):2136-47. doi: 10.1038/mt.2013.138. Epub 2013 Jul 2.

BridgeBio

Hay M & Rosenthal J. Nat Biotechnol. 2014 Jan;32(1):40-51. doi: 10.1038/nbt.2786.

Brown D & Superti-Furga G. Drug Discov Today. 2003 Dec 1;8(23):1067-77. https://doi.org/10.1016/S1359-6446(03)02902-7

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