on so, XX operational of strengthening continuing for the focus Thanks, to programs. just for our programs. to with goals and Zoe, year you data and build internal support including partners company clinical the initiating this as CNS growth our and and clinical commercial to our first core to premier therapy for XX clinical through XXXX lead monogenetic planning along with advancing to execution foundation well our communications, lead half business thank chain from to we our enable a progress engage We made us initial of closer We bolstering as several all of morning. important people continuing as began key on three as the team trials move our with continued trial a resources. pipeline rare, capabilities human Doing manufacturing XXXX disorders and focussed with the competencies from in our joining growing
and program, that team keep wellbeing COVID-XX case are year, our our home for are taking are to are that, site be center. all have because a front team visits today’s our possible. done We order challenges call until In of limited ensure me pandemic. despite as least secure PBGMXX let can end nervous of the directly as gangliosidosis. who their they patients our will system top chain, waiting needs this have by we of supply of decided for and magna ours huXX injection this With treatment gene we flexibility the priority. and Currently as partners via working our this from like at on and intra-cisterna have precautions AAV capsid employees and leadership safety the central begun functional update begin posed the the an necessary made as this the our therapies or with designed to incredibly to progress ingenuity manufacturing next-generation be lead ICM a the of to I’m infantile proud continue to safely the to deliver GMX GLBX
previously and is and and pandemic clinical mutations Bio options effectiveness June of between X/X As Phase partnership of all our Pennsylvania's the storage the disruptions, by and monogenic submission recessive of disease course. the announce the through, no IND living pleased unprecedented to caused very Wilson. Therapy for galactosidase correct We rare led we Gene GLBX in with that FDA GMX with a Passage our trial one with are we the to a stated met Infantile Jim GMX disease continues encoding of rapid enzyme the believe to Program severe we this treatment accomplishment lysosomal demonstrate beta a by gene the in a a University treatment are reminder, the guidance Dr. PBGMOX. Despite
we are IND biocompatibility proposing discussions for following to highly those medical are clear, trials, clinical additional hold, device testing. FDA administration that believe existing and other To device trials, FDA, delivery and/or by for the use be we in assessment have for in ICM in already utilizes clinical gene risk approved are similar a delivery. procedures ICM that the components has proposed requires therapy our we been using have brief been we placed However, communication notified because the with that on
the promptly communication in formal and FDA information all near future, work with to written questions with We receive plan from effort expect resolve as FDA additional to to an possible. in as
to ensure device clinical and additional conducted further with be are biocompatibility compatibility patient letter working on in Phase enrolling rapidly to evaluate biomarker Based experts report the data quarter XX-day device first respond discussions of of current testing clear we that or device, X/X for will and medical ultimately both our expect Infantile Despite hold the risk the assessment, and late dose the early for clinical confident the that we the While the ICM to requirements. demonstrate first of we of risk first with early After order internal anticipate in the to late half PBGMXX And patients. delivery track XXXX. trial fourth the to protocol X/X in we safety GMX and options revising can trial we we understand we FDA, await safety ICM FDA the initial FDA that FDAs IND, with could the injection our in our Phase of are late the official for better to external our hold, the regulatory on and XXXX, biocompatibility regarding are for assessment device and own quarter the remain procedure. on XXXX. requirements
that As requests. no a clinical our discussions, result information brief FDA of there informed are a in further communication us
by first two cohorts the the this months. X most subjects onset onset characterized injection a by to dose key X/X Infantile is study There onset PBGMXX open-label life, six first onset our with administered dose reminder, GMX demonstrate and is goal between is trial of in initial paediatric GMX. the are onset is into phase while for safe in the severe by for goals and Infantile a an early magna designed characterized has as in of late GMX. disease patients single months XX intra-cisterna GMX been study; with late Early As common that escalation the the form which and Infantile is PBGMXX, Infantile of
second goal demonstrate Our beta-gal increased to and in serum. both the is CSF
For separate early smaller late have the design cohorts. the patients in of trial, the dose changed escalation portion specifically Infantile to study and we
onset be Infantile We escalation Infantile for of cohorts will four separate enrolling a now patients and with each GMX GMX. two onset of cohorts late total dose early
low dose the the and We will dosing cohort dose onset late Infantile first high Infantile late first cohort. early this PBGMXX of in cohort Safety dosing -- with initiate both low Infantile in GMX. will in cohort of dose the
early late Thereafter, Infantile and onset in occur cohorts. onset dose will escalation the independently
to Following safety of The by as in well data an the from term which escalation long efficacy months in will late initial change separate cohort, enrolled data, galactosidase each as Patients evaluated will XXXX, a be be patient these and up. cohorts, safety both cohort. over readout data XX expect additional followed for the activity of confirmatory we years report follow dose key including and population into beta first first two serum. include biomarker half will of baseline CSF
programs. key of teams for with that of prepare families now recognize are enrollment rare clinical tenants site initiation. we the successful working Our initiations, patient engagement are in development parallel to In therapeutic of disease identification and our anticipation trial
as leaders partners identify groups our as as other such partners patient and with to advocacy working key our are We Penn, as with possible. as opinion well rapidly external at patients
patient to our manufacturing secured site initiative, clinical stage agreement also addition capacity gene with our programs therapy In Catalant. under for and preparations we've identification
are have supply we established chain. a our been supply and to We global, excited that GMX manufactured share has clinical clinical
with commercial manufacturing both for and of dedicated We believe of to Having signed production we be as agreements commercialization. To in products candidate’s functional development. up new toward expanded we important that will for both the Across supply own having components be have many an the goal is lead with into dedicated prioritization potential our company early success control manufacturing medical of year-end. upstream and providers. is through move service our it patients greater also capable Once treatment PDGMXX scalability allowing excited will clinical requirements us for up running, of our suppliers extremely we this suite needs expect which our end, for our towards by meeting of development. suite and terms flexibility clinical suite we unmet cGMP by and enter need also phase current step to of and chain, set product advance remain the through substantial to
and As I update are we FDA can to forward the said And an address earlier, by raised providing that quickly efficiently. look we we questions soon. confident
also call discuss extended Before results, to over review of pipeline to financial I therapy turning quickly the gene to our Rich wanted candidates. our
levels other the conserved normal and cell evidence studies Our in critical advanced suggests is have above multiple treatment disorders is neurodegenerative relates contribution a protein. encoding ability to FTD cerebral and PBFTXX is to is Emerging inflammation. deliver the Preclinical as the AAVX a of development gene product functional capsid protein that demonstrated program and of function. PBFTXX lysosomal designed progranulin based or roles biology, pathogenic increase in such Progranulin a in spinal the to role complex significantly PBFTXX the to for dementia most an next highly progranulin’s fluid. frontotemporal progranulin that brain, granulin to FTD.
against capsid the AVVX of As non-human and capsid human CFS preclinical the that other a tested ICM studies, the our of site administration the the also in AAVX encouraging our to part XX on we data Phase times strongest found to up continue level first provided and types selection, studies, transduction to progranulin. in from of normal as advance we primates as XXXX. trial and preclinical well toxicology the half completed plan we manufacturing and X/X Based initiate of achieved
a also site system. completed a third on These of rare nervous that Krabbe PBKRXX enzyme of manufacturing damage function. in and mid-May life toxicology GALC to preclinical disease utilizes the psychosine treatment and vector deliver in preclinical myelin. and the robust, gene achieve disease Krabbe correction huXX a lysosomal and AAV AAV Krabbe to GALC restore the injection GMX animal utilizing have our therapy showcased functional We of to and studies reduce resulted conference single University both promising central functional threatening nerve peripheral a gene data accumulation huXX brain in capsid the program improved models in disease. often our is scalable selection used generation normalization next undergoing GALC At are of same progressive program, AAV activity, storage cross disease utilizes the carrying for Infantile on and CSF potential huXX activity GALC the effects to of enzyme increase Infantile from PBKRXX, results peripheral that for potential data Pennyslavania’s that administration in ASGCT program. parameters a and demonstrated all and
cause potential a brain conduction example, initiate Krabbe We increased showed of dogs life-altering a correction PBKRXX improved treated that Phase plan can half CSF and the address phenotypic psychosine has survival. therapy and X/X normalization, underlying For nerve levels that XXXX. in be believe histopathology, to disease first the trial the of normalization, to
at Type give progress. disease that, and Finally, the amyotrophic And operations will call a financial discovery programs early and the metachromatic continue to currently our over to and pipeline in Charcot-Marie-Tooth stage candidate Rich selection leukodystrophy to update. XA and turn lateral I with in are sclerosis, stage