Credit: CIPhotos/Getty ImagesiECURE, a cistron editing startup focused connected liver disorders, this week announced a $65 cardinal Series A-1 financing. The institution is collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, MD, PhD, who led the gene-therapy proceedings that ended with teen Jesse Gelsinger’s decease successful 1999. This lawsuit fto to a multi-year probe and legislature hearings.
But Wilson rebounded from the Gelsinger calamity to substance a powerhouse of cistron therapy discovery. At slightest 40 companies are utilizing AAVs that autumn nether Wilson’s patents, accounting for astir 100 cause improvement programs.
iECURE’s pb attraction is GTP-506, which relies connected the transportation of duplicate adeno-associated microorganism (AAV) capsids carrying antithetic payloads. Those are an ARCUS nuclease vector (GTP-506A) targeting cistron editing successful the PCSK9 cistron locus and a therapeutic donor vector (GTP-506D) that inserts the OTC cistron to supply the correction. iECURE licensed the ARCUS nuclease for GTP-506 from Precision BioSciences.
The pb denotation is ornithine transcarbamylase (OTC) deficiency, a uncommon familial information characterized precocious levels of ammonia successful the humor that origin terrible and imperishable encephalon damage. The terrible signifier of the information emerges soon aft commencement and the lone attraction for aboriginal onset terrible OTC deficiency is simply a liver transplant. iECURE says GTP-506 could supply semipermanent attraction done in-vivo cistron editing.
The FDA has granted some Orphan Drug Designation and Rare Pediatric Designation to GTP-506 for OTC deficiency.
The financing was co-led by Novo Holdings A/S and LYFE Capital with information from existing investors Versant Ventures and OrbiMed Advisors. This latest financing, coupled with the $50 cardinal raised successful the anterior Series A financing announced in September 2021, volition bring the company’s full funds raised to $115 million.
“In the past year, we person made important advancement successful some advancing improvement of our pb programme for neonatal onset OTC and gathering a world-class squad with an extended way grounds successful processing and commercializing caller therapies,” said Joseph Truitt, Chief Executive Officer of iECURE. “We judge that this backing volition alteration america to execute each the tasks indispensable to statesman objective improvement of what could beryllium the archetypal mutation-agnostic in vivo gene insertion therapeutic program.”
The institution expects the proceeds from the Series A-1 financing to alteration the advancement of the GTP-506, including backing IND-enabling studies, starting objective trials (subject to regulatory approval), and achieving aboriginal quality information readouts. In addition, the financing is expected to substance further advancement connected iECURE’s portfolio of cistron editing products for the attraction of patients with different uncommon liver diseases, including citrullinemia benignant 1 and phenylketonuria.
“iECURE, done its collaboration with the University of Pennsylvania Gene Therapy Program, has generated awesome pre-clinical information demonstrating durable cistron integration successful non-human primates with its cutting-edge attack to mutation-agnostic successful vivo cistron editing. We are excited to enactment intimately with the institution arsenic they the determination their programs into the clinic,” said Ray Camahort, PhD, Partner astatine Novo Ventures.
The decease of Jesse Gelsinger is not the lone setback Wilson has faced successful his agelong vocation astatine Penn, which started successful 1993 erstwhile helium was recruited to pb the recently founded Institute for Human Gene Therapy there. Earlier this year, leaked reports alleged that apical officials from the University of Pennsylvania “manipulated” an probe into workplace maltreatment charges successful Wilson’s Gene Therapy Program to support the University and Wilson.
As portion of iCURE’s Series A-1 financing, Ray Camahort, PhD, Partner astatine Novo Ventures and Derek Yuan, PhD, Managing Director astatine LYFE Capital volition articulation iECURE’s Board of Directors. Additionally, Tal Zaks, MD, PhD, has been appointed to iECURE’s committee arsenic OrbiMed’s caller typical connected the Board of Directors. Stephen Squinto, PhD, volition proceed to service connected the Board of Directors arsenic an autarkic member.
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