Teprasiran program

siRNA targeted to p53 attenuates ischemic and cisplatin-induced acute kidney injury.
Molitoris BA, Dagher PC, Sandoval RM, Campos SB, Ashush H, Fridman E, Brafman A, Faerman A, Atkinson SJ, Thompson JD, Kalinski H, Skaliter R, Erlich S, Feinstein E.
J Am Soc Nephrol. 2009. 20(8):1754-64.

Intravital two-photon microscopy assessment of renal protection efficacy of siRNA for p53 in experimental rat kidney transplantation models. Imamura R, Isaka Y, Sandoval RM, Ori A, Adamsky S, Feinstein E, Molitoris BA, Takahara S. Cell Transplant. 2010.19(12):1659-70.

Toxicological and pharmacokinetic properties of chemically modified siRNAs targeting p53 RNA following intravenous administration. Thompson JD, Kornbrust DJ, Foy JW, Solano EC, Schneider DJ, Feinstein E, Molitoris BA, Erlich S. Nucleic Acid Ther. 2012. 22(4):255-64.

Treatment with QPI-1002, a short interfering (si) RNA for the prophylaxis of delayed graft function. Peddi V, Ratner L, Cooper M, Gaber O, Feng S, Tso P, Bowers V, Naraghi R, Budde K, Polinsky M, Squiers E, Erlich S and study investigators group. The World Transplant Congress. 2014. Abstract Supplement jointly published by the Am J Transplant and Transplantation. Late Breaking Oral Presentation. Abstract #2967.

Safety and tolerability study of an intravenousladministered small interfering ribonucleic acid (siRNA) post on-pump cardiothoracic surgery in patients at risk of acute kidney injury. Demirjian S, Ailawadi G, Polinsky M, Bitran D, Silberman S, Shernan SK, Burnier M, Hamilton M, Squiers E, Erlich S, Rothenstein D, Khan S, Chawla LS. Kidney Int Rep. 2017. 25;2(5):836-843

Efficacy and safety of QPI-1002 for prevention of acute kidney injury (AKI) following cardiac surgery (CS). Corteville D, Still R, Jayasankar V, Szabo G, Swaminathan M, Lamy A, Lehner L, Thielmann M, Brown C, Squiers EC, Schwertschlag U, Potts A, Patel A, Messersmith E, Odenheimer D, Rothenstein D, Erlich Sand the QRK209 Study Group. Presentation at American Society of Nephrology Kidney Week. 2017, High impact clinical trials session, Abstract: SA-OR124.

QPI-1007 program

Ocular neuroprotection by siRNA targeting caspase-2.Ahmed Z, Kalinski H, Berry M, Almasieh M, Ashush H, Slager N, Brafman A, Spivak I, Prasad N, Mett I, Shalom E, Alpert E, Di Polo A, Feinstein E, Logan A. Cell Death Dis. 2011 16; 2:e173.

Neuroprotection in a novel mouse model of multiple sclerosis. Lidster K, Jackson SJ, Ahmed Z, Munro P, Coffey P, Giovannoni G, Baker MD, Baker D. PLOS One. 2013. 8(11): e79188.

A Phase I open label dose escalation trial of QPI-1007 delivered by a single intravitreal (IVT) injection to subjects with low visual acuity and acute Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). Antoszyk A, Katz B, Singh R, Gurses-Ozden R, Erlich S, Rothenstein D, Sharon N, Hodge J, Levin L, Miller N. and QPI-1007 NAION study group. . ARVO Annual Meeting Abstract. Investig. Ophthalmol. Vis. Sci. 2013, 54, 4575.

Combined suppression of CASP2 and CASP6 protects retinal ganglion cells from apoptosis and promotes axon regeneration through CNTF-mediated JAK/STAT signaling. Vigneswara V, Akpan N, Berry M, Logan A, Troy CM, Ahmed Z. Brain. 2014. 137(6):1656-75.

Toxicological and pharmacokinetic properties of QPI-1007, a chemically modified synthetic siRNA targeting caspase 2 mRNA, following intravitreal injection. Solano EC, Kornbrust DJ, Beaudry A, Foy JW, Schneider DJ, Thompson JD. Nucleic Acid Ther. 2014. 24(4):258-66.

Long-term neuroprotection of retinal ganglion cells by inhibiting caspase-2.
Vigneswara V, Ahmed Z. Cell Death Discov. 2016. 2:160.

Caspase-2 mediates site-specific retinal ganglion cell death after blunt ocular injury. Thomas CN; Thompson AM, McCance E, Berry M, Logan A, Blanch RJ, Ahmed Z. Investig. Ophthalmol. Vis. Sci. 2018.59, 4453-4462.

Other siRNAs

RNAi-mediated silencing of nuclear factor erythroid-2-related factor 2 gene expression in non-small cell lung cancer inhibits tumor growth and increases efficacy of chemotherapy. Singh A, Boldin-Adamsky S, Thimmulappa RK, Rath SK, Ashush H, Coulter J, Blackford A, Goodman SN, Bunz F, Watson WH, Gabrielson E, Feinstein E, Biswal S. Cancer Res. 2008. 68(19):7975-84.

Delayed intrathecal delivery of RhoA siRNA to the contused spinal cord inhibits allodynia, preserves white matter, and increases serotonergic fiber growth. Otsuka S, Adamson C, Sankar V, Gibbs KM, Kane-Goldsmith N, Ayer J, Babiarz J, Kalinski H, Ashush H, Alpert E, Lahav R, Feinstein E, Grumet M. J Neurotrauma. 2011. 28(6):1063-76.

Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients. Nguyen QD, Schachar RA, Nduaka CI, Sperling M, Basile AS, Klamerus KJ, Chi-Burris K, Yan E, Paggiarino DA, Rosenblatt I, Khan A, Aitchison R, Erlich SS; PF-04523655 study group. Eye. 2012. 26(8):1099-105.

Evaluation of the siRNA PF-04523655 versus ranibizumab for the treatment of neovascular age-related macular degeneration (MONET Study). Nguyen QD, Schachar RA, Nduaka CI, Sperling M, Klamerus KJ, Chi-Burris K, Yan E, Paggiarino DA, Rosenblatt I, Aitchison R, Erlich SS, MONET clinical study group. Ophthalmology. 2012. 119(9):1867-73.

Dose-ranging evaluation of intravitreal siRNA PF-04523655 for diabetic macular edema (the DEGAS study). Nguyen QD, Schachar RA, Nduaka CI, Sperling M, Basile AS, Klamerus KJ, Chi-Burris K, Yan E, Paggiarino DA, Rosenblatt I, Aitchison R, Erlich SS. DEGAS clinical study group. Invest Ophthalmol Vis Sci. 2012. 53(12):7666-74.

ASPP1/2 regulate p53-dependent death of retinal ganglion cells through PUMA and Fas/CD95 activation in vivo. Wilson AM, Morquette B, Abdouh M, Unsain N, Barker PA, Feinstein E, Bernier G, Di Polo A. J Neurosci. 2013. 33(5):2205-16.

siRNA targeting Hes5 augments hair cell regeneration in aminoglycoside-damaged mouse utricle. Jung JY, Avenarius MR, Adamsky S, Alpert E, Feinstein E, Raphael Y. Mol Ther. 2013. 21(4):834-41.

RTP801 gene expression is differentially upregulated in retinopathy and is silenced by PF-04523655, a 19-Mer siRNA directed against RTP801. Rittenhouse KD, Johnson TR, Vicini P, Hirakawa B, Kalabat D, Yang AH, Huang W, Basile AS. Invest Ophthalmol Vis Sci. 2014. 55(3):1232-40.

siRNA-Mediated Knockdown of the mTOR Inhibitor RTP801 Promotes Retinal Ganglion Cell Survival and Axon Elongation by Direct and Indirect Mechanisms. Morgan-Warren PJ, O’Neill J, de Cogan F, Spivak I, Ashush H, Kalinski H, Ahmed Z, Berry M, Feinstein E, Scott RA, Logan A. Invest Ophthalmol Vis Sci. 2016. 57(2):429-43.

Structure-function correlation of aminated poly(α)glutamate as siRNA nanocarriers. Krivitsky A, Polyak D, Scomparin A, Eliyahu S, Ori A, Avkin-Nachum S, Krivitsky V, Satchi-Fainaro R. Biomacromolecules. 2016. 17(9):2787-800.

Systemic delivery of siRNA by aminated poly(α)glutamate for the treatment of solid tumors. Polyak D, Krivitsky A, Scomparin A, Eliyahu S, Kalinski H, Avkin-Nachum S, Satchi-Fainaro R. J Control Release. 2017. 257:132-143.

Amphiphilic poly(α)glutamate polymeric micelles for systemic administration of siRNA to tumors. Krivitsky A, Polyak D, Scomparin A, Eliyahu S, Ofek P, Tiram G, Kalinski H, Avkin-Nachum S, Feiner Gracia N, Albertazzi L, Satchi-Fainaro R. Nanomedicine. 2018 14(2):303-315

TGF-β-induced IOP elevations are mediated by RhoA in the early but not the late fibrotic phase of open angle glaucoma. Hill LJ, Mead B, Thomas CN, Foale S, Feinstein E, Berry M, Blanch RJ, Ahmed Z, Logan A. Mol Vis. 2018. 24:712-726

Effects of siRNA-mediated knockdown of GSK3β on retinal ganglion cell survival and neurite/axon growth. Ahmed Z, Morgan-Warren PJ, Berry M, Scott RAH, Logan A.
Cells. 2019. 8(9):956.