Dr. Timothy J. Miller
EB-XXX observed and IIIB some everyone. MPS in programs. made continue by like would after morning, enrollments benefits along IIIA, and IIIA. start The in-house clinical clinical additional finalize ABO-XXX two rare program X and MPS lead these observed in year. our GMP to programs continues durability from and we in Medicine sites initiated Therapy gene Epidermolysis commercial the marked developing reported for these gene position the of of the in safety to recently, trials manufacturing Good we and cell granted an Phase with initiating in building Advanced This biopotency clinical quarter our active Most treat Bullosa fourth before devastating FDA the with pediatric MPS therapies our or the strong ABO-XXX Data The Significant the the global third and RMAT further facility protocol EB-XXX administration our at this our underscore program, strategic was Bullosa as diseases. clinical clinical several therapy. program strengthens initiative advance with and Epidermolysis three in for the I achievements data trial pivotal was designation. notable overview. clinical to progress programs, Regenerative to clinical
spinal on optimizing in tropisms CLNX Batten to going plasma including vector the that achieved studies early second CNS quarter well be continues MPS continue initiated reported demonstrate non-human cerebral enzyme MPS the in demonstrating regards our to -- have IIIA program. to at dose compared enhanced our urine, XXX-fold we trial administration, Cohort Our in continuing than work, X/X initial AIM now signals the program post disease-specific clinical show X our with a progress program candidates. expanded MPS exciting and in at IIIB we occurring primate tissue IIIA And that targeted which safety addition, biopotency heparan our And significant enrolled therapy work capsids. the preclinical are In designations while the sulfate vg/kg for naturally XEXX the very to in four to reductions and AAV to greater lead Phase regulatory program, has similar days or signals patients for of activity. increase particularly additional biopotency and in infantile we is platform, with tolerated NAGLU reductions and as fluid, XX
to an analysts. Day New world on update and gene additional opinion further our and with $XX.XX foundations October experts in program where For Research XX% these the trying from clinical key inaugural an some That from did crowd ways leaders corporate and diseases. our achievements to in around as help day of together clinical institutional Abeona in further our investors grant to over the host nine we a find City, patients presented Development MPS therapies Sanfilippo York quarter, management on XXth to to efforts we IIIA our week, the for million IIIB announced clinical our of over same equity development of treat and unmet
concept produce for and will in the use facility, ABO-XXX to in-house, EB-XXX, therapy therapies take these XX,XXX that following manufacturing syndrome and We to The facility route also treatment the girl ABO-XXX young the has Center announced to Sanfilippo Diseases in six Kansas commercial of have family’s center Abeona’s being able syndrome located dystrophic vectors, the in been produce trial second new foundation awareness broke ground bio at In the The we AIM instrumental vectors facility like Elisa on it expanded our be cell early continue facility honor or treatment so. finished unique initially that This bullosa offices. in built funds to will months quarter gene and be manufacturing on validated constructs, gene Linton epidermolysis house therapies. raising again in out rare was over groundbreaking the finished we we terminal concept be facility. the whose in who passed from to a Sanfilippo autologous vision vectors Rare and in this square for and able recessive used and for our for The of our also of our for make foot will clinical disease. of Cleveland put production commercial proprietary for material named vector things and commercial of delivery to those October, and novel and
strengthening from robust to the quarter the commercial vector further to internal fourth including therapy achievement In platform an efforts We coming look gene expansion MPS addition, foundations commitment advancing our is capabilities, in with generation our demonstrates investment of quarters. investors and key and the high-quality hires, and capabilities next clinical our pipeline products. advancement forward IIIA and leading that
recent expedited our therapy wound RMAT with the substantial diseases. FDA the significant treatment for on EB-XXX, from epidermolysis over or autologous progress for serious regulatory did we demonstrated the have over EB-XXX senior patients is the some cell suffering the based with clinical healing treated FDA clinical priority significant For program, or years. to January, bullosa. advantages The approval preliminary the severe of received some this discussions Phase designation indicated EB-XXX RDEB, is life-threatening most has from trial, wounds data granted of form designation ex-vivo In review designation trials process which and as therapy have for we achievements. enables that which drug the the for The clinical patients candidates offered collaborative it options where personnel and existing two X/X
designation Just are Designation, the The PRV treatments previously Disease as and Rare this Pediatric are prerequisites both Breakthrough relying disease, latter Orphan received particular care. FDA no Drug reminder, there Priority received for mainly for designation. FDA the Therapy on EMA. process. was a These EB-XXX program designations The and palliative or Review the by Voucher
provide we Phase for especially designations closely RDEB Breakthrough trial and preparation our We in and continue the initiate RMAT in work with year, FDA expect through as trial patients guidance months. X in the to pivotal we the the the this upcoming later to
IIIA, X. the is the from significant that Cohort main CSF a take sulfate storage ABO-XXX away enrolled of and disease including early Spain Phase the number we topline showed global in the being MPS our the Cohort dose the additional three expansion X/X presented and the patients during in MPS diminished of four trial we results in trial with for February, dependent for XEXX One Australia, trial, In For fragments global including announced program in of and IIIA our X dose total patients disease dose lysosomal for has one also GAG quarter heparan WORLDSymposium data for the of liver Both the one reduction and the recent clinical achievements, volumes. one total in U.S. urinary the the time clinical in received reductions pathology X underlying vg/kg. Cohort in U.S. and patient ABO-XXX received
one in ABO-XXX Importantly, Abeona age of III this at upcoming going the and lower the the and continues we of to that’s young timeframes to be of to allow age, results, enabled updates for forward over world well-tolerated to able children and at the the conferences treatment go those development evidence to clinical age. preclinical trials young only X demonstrated as clinical in Alongside this Cohort allowing also months in clinical to enrollment X. and enroll ABO-XXX benefits be the all months. at will young to in global as six look has coming as accelerated the FDA announcement include enrollments year the follow-up months. six post months important the in at be patients We targeted in doses, all Cohort as providing to diseases trial MPS
United the disease signals clinical has XEXX Designation in the MPS Orphan Drug signals the These clinical vg/kg patients and urine as results CSF, our In IIIB. first is has announced significant plasma gene been we the increase post Europe program, also in safety that the that observed we has on Voucher patient demonstrated that results program that For biopotency the in our activity. in Designation dose we’ve the therapy, IIIA Review similar and Pediatric a States specific also of process. December, initial the Phase ABO-XXX of for Disease NAGLU Priority injection. enrolled in well-tolerated to a demonstrated treated very achievements, days at and sulfate MPS XX reductions biopotency for clinical of enzyme on at granted the with part in with ABO-XXX based in February, Rare XXX-fold first heparan X/X early and the and trial ABO-XXX recent reported and
CLNX the has respectively. CLNX progress, for Orphan Batten CLNX, gene Disease for the Juvenile disease Batten targets Onset IND-enabling these data and Drug clinic. the recent Infantile Designation, and Designation for known their received For programs again supported March, The of the program has to-date amount as we the into translational in we received importance and known In and ABO-XXX two of FDA both ABO-XXX received studies treatment also demonstrating Pediatric the designations. programs and the February, as of the Rare preclinical Disease, continue how disease, treatment therapy also Disease of Late preclinical have we ABO-XXX
patients For intrathecal generation patients the vector-selective redosing intramuscular. tissues that platform, of routes tissue for with indicated target This either second generation efficiently vectors AIM treatment the AAV approaches their to natural with strategy have also whether or provides neutralizing relative serotypes. antibodies to the different or initial that intravenous administration specificity, have for or can AIM vector vectors first multiple with studies
particularly as targets will out again to to entered continue we for candidates primate We second And develop lead Davis chimeric the with CNS, through number in both vectors, internally partnering over to financial look AAV And strategic those AIM turn a our with it for of results I the forward Jeff reporting in results. fourth we summary and our quarter half. different that, the efforts. the studies
