and is therapy you Pete, gene company everyone. Voyager Thank a breaking summarizing thesis. barriers and I morning, in biotechnology would good by dedicated and investment like to Voyager’s start through neurology. to
the getting challenges. molecules therapy narrow windows issues. therapeutic The blood-brain-barrier. limited Neurology safety potential has by difficulty limited larger of has and been by for disciplines the constrained by Gene both across been associated delivery is
we deep to constraints, discovery, expertise address in we shareholders. create for Voyager, cutting which are ultimately pharmacology capabilities At will value and and significant neuro patients these capsid leveraging believe edge
at value of pillars three Voyager. created have We
partnerships. pipeline significant TRACER that Pfizer for our future Novartis discovery capsids partners with and fuel generating potential platform including of breakthrough is First, own to and our capsids Neurocrine, both
is million TRACER system. our only of organs. in in-vivo XX At our AAVX allowed the proprietary of displayed capsids those to capsids transduction select This increased variants and screening core has than driven the target team AAVX more that expression evaluate and
preclinical We central demonstrated trans-gene have conventional brain XXX compared have and than more expression in started targeting AAVX higher we capsids. fold in nervous studies, system, the the by to
We have also our across increases species, may non-human confidence be which multiple demonstrated able that we blood-brain-barrier these translate mice including penetration to to primates, humans. properties and
[XXX%] high by month, therapeutic a novel potentially of therapies. last in At we that demonstrated gene administered CNS intravenously a range capsid (Ph) ESGCT of on presented the of levels improving of doses expression when window data the used capsids, gene conventional
in data receptor and Further, families novel we further In receptor this for and we translate cell discovery of expression our humans. function of characterizing a we are this confirmed that near-term, seeing one the clinical capsids trial. our increases surface results confidence analogous preclinical into the human presented binding the may
generated platform. could In enable our engineering this further by capsids receptor enhance the the longer-term, TRACER to reverse
protein this such constitute receptor new across have whether enable platform a delivery CNS we which be modalities to explore therapeutic addition, experiments to leverage could for In may successful and development. begun blood-brain-barrier, the we if as to drug of able the oligonucleotide
XXXX. exercise companies. and discussions $XX revenue of triggered September attracted derived multiple the Pfizer in are Pfizer’s QX to novel additional Novartis, from and in including million their in The and with have deferred decision option ongoing capsid capsids TRACER interest of pharmaceutical revenue partners, $XX multiple million
is want three validated our of value and to transformative human human CNS our of by targets advancing pipeline. review pillar is call our directed programs biology. genetics But just against I progress each out are minute. a will second the lead in I Our pipeline
achieve efficiently to biomarkers of quickly and of proof availability the exploit they a to biology. Moreover, enable path
foundation we we in next our filings XXXX the call, laying year, first half said the this candidates of IND and year into XXXX. to As these identify for expect later QX in and
We create sclerosis for burdens also disease, and and or targeting Alzheimer’s disease amyotrophic caregivers, lateral life-threatening note, significant Parkinson’s serious patients diseases ALS. are that we
our Our sheet, non-dilutive track third pillar enabled revenue. of generating value sound our partnership of is record balance by
reported As of September equivalents of and XX, XXXX, marketable securities we cash cash, million. $XXX
received expenses expenditure to sheet of the its are into balance Our licensing costs be XXXX. requirements with Voyager’s as $XX Pfizer’s the triggered sufficient cash QX XXXX. under to operating expected payment along in Neurocrine to amounts million expected and position development capital for collaboration be meet exercise agreement a reimbursement planned
the Novartis the enhance third-parties. the further. date in to no The obligations debt prior option QX Additionally, to exercise has company’s operating runway would expiration option potential by XXXX company
want we don’t tests that for work capsids. make on not tests our that internal else here, one around point which may I target, anybody licenses interest, select is our their capsids and with are same is this Once select possible select that that our it structured to some may also gene, the more entirely gene partners the multiple programs. own we partners of but of
structure twofold. of this benefits are The
capsids, First, to to optionality deals partnerships. continue have not because are pursue these exclusive we to the the
Second, some clinical we capsid partners of in our of our own initial they that capsids. validation programs, do may utilize also choose provide our
advance pipeline gene antibodies silencing our for gene humanized Alzheimer’s into a dig Voyager disease. and the for prioritized for continues anti-tau replacement bit three SODX disease, programs, Parkinson’s as to want to AOS I now. deeper GBAX
Phase three funding Neurocrine we programs, with to have which remind is collaboration gene to X. free a want these therapy you ongoing I ataxia, develop that a for Friedreich’s to currently addition through In Neurocrine
has Voyager and and global to grant full cost with royalties. split At Voyager or to in in XX/XX rights and from option Neurocrine profit a asset exchange at XX% that the the U.S. coming co-commercialize commercial milestones to co-develop point, an
developed pathology a has as research vein tau across Alzheimer’s IV an that tau in disease. Our program well-known certain passive regions is delivered antibody shown being propagates immunotherapy, in pattern
proof with this monitored can of imaging, and PET biology. use to plan early tau be to of The establish we pathology spread biomarker
antibody that hypothesis block may of an extracellular neuron-to-neuron is targeting at tau tau plausible spread several the therapeutic sites. Our
in Our C it that models other preclinical N in been our antibody clinical targets significantly pathological from terminal has shown and demonstrated that the antibody efficacy reduce rather not region, terminal to than of the travel. is the differentiated antibodies spread have
to with on humanize murine antibodies. track are our We work the
now genetic to XX-fold. of Moving to approximately the the mutation GBAX Parkinson’s risk common have factor, patients up a of in most increasing disease Parkinson’s disease disease XX% GBAX, risk
measured GBAX in of carriers Parkinson’s GBAX believe the encodes mutation. substrates GBAX lysosomal have abnormally mutations of enzyme, GCase benefits. of be in GCase function or CFS, as to we elevated therapeutic patients GCase, loss the can and due can are restoration GCase in that levels with will glucocerebrosidase
tropic These penetrant, development studies biomarkers replacement novel NHB approach combines select derived capsids. derisking path lead a TRACER to provide potential a gene a our candidate underway. early CNS GBAX with to clinical are BBB our
rapidly typically in three SODXALS. leads approximately ALS to progressing with We ALS after a benefit therapeutic the patients expression can neurodegenerative death now provide CNS, silencing SODX is disease believe diagnosis. SODX years by the we gene to that that And of mutations.
approach biomarker an CSF small is IONIS, a Proof-of-concept in has which demonstrated and and SODX review drug for measurable by FDA. study. under clinical sponsored as can Biogen in investigational this for a currently serve by efficient is by early [Indiscernible], proof-of-biology been
of Serum a measured or efficacy. chain also will be neurofilament to whether determine not light signal there is
novel our a Our siRNA studies CNS-tropic blood-brain-barrier constructs select NHP candidates capsids. to TRACER are penetrance, with a lead underway. derived potent approach combines
We lead continue candidates development three QX XXXX to HX for between and expect to programs all identify XXXX.
I novel want These expected partnerships IND we capsids. support lead using also in XXXX and be event filings generated to enabling TRACER with XXXX. will this on that, several IND candidates and slide studies to note have
mentioned partners I may any As clinical earlier progress even by above, our provide validation.
during believe the subsequent is third quarter to XXXX. gaining period. strong demonstrated we close towards as XXXX with has summary, our and And the Voyager and ability momentum In we validation look out the execute company
to continue In as as our addition of executive our Board Directors advancing our well to build out pipeline team. we
appointed Dr. Mackey We our to Board. Kitty
We Peter to President as CFO Scientific Corporate also Tristan we Morrison promoted Affairs. of And and Todd added Dr. Senior as Officer. Carter Chief Pfreundschuh Vice
posters platform exercise on option. data and website We further Pfizer’s we encouraging you our to the Conferences. And from seen AAIC our its if haven’t Those saw validation of are also pre-clinical TRACER them. decision presented ESGCT at
work lead keep this year of barriers of look neurology. the Looking We to first constraining gene and we the fields our as the towards you candidates to the for will identifying we our next forward, of end break between and through continue and pre-prioritized forward therapy we advance half INDs. programs pipeline year, updated
we generated have share will conferences. scientific at continue We exciting the data to
year. initial the Novartis of March decision have next also option by coming up We exercise
and scientists to working professionals. excited a the of I acknowledge and seven-months with team. now this Voyager company about so for on have CEO be everyone to I incredibly take such to talented I’m want been moment other
With are have. that, you to happy questions we may any take
our Chief for Carter, CFO Pfreundschuh, Dr. and Scientific Q&A Joining me today are Todd Officer. Peter Operator. our