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L E ADI NG RE GE NE RAT I VE ME DI CI NE 1 ooking State me nts Cautionar y State me nt Conc e r ning F or war d- L T his pre se ntatio n is inte nde d to pre se nt a summary o f ACT s (ACT , o r Advanc e d Ce

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SLIDE 1

1

L E ADI NG RE GE NE RAT I VE ME DI CI NE

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SLIDE 2

2

Cautionar y State me nt Conc e r ning F

  • r

war d- L

  • oking State me nts

T his pre se ntatio n is inte nde d to pre se nt a summary o f ACT ’ s (“ACT ”, o r “Advanc e d Ce ll T e c hno lo g y I nc ”, o r “the Co mpany”) salie nt b usine ss c harac te ristic s. T he info rmatio n he re in c o ntains “fo rwa rd-lo o king state me nts” as de fine d unde r the fe de ral se c uritie s la ws. Ac tual re sults c o uld vary mate rially. F ac to rs that c o uld c ause ac tual re sults to vary mate rially are de sc rib e d in o ur filing s with the Se c uritie s and E xc hang e Co mmissio n. Yo u sho uld pay partic ular atte ntio n to the “risk fac to rs” c o ntaine d in do c ume nts we file fro m time to time with the Se c uritie s and E xc hang e Co mmissio n. T he risks ide ntifie d the re in, as we ll as o the rs no t ide ntifie d b y the Co mpany, c o uld c ause the Co mpany’ s ac tual re sults to diffe r mate rially fro m tho se e xpre sse d in any fo rward-lo o king state me nts. Ro pe s Gray

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SLIDE 3

3

T hr e e c linic al tr ials aime d at ve r y lar ge unme t me dic al ne e ds

  • Dry Ag e re la te d Ma c ula r De g e ne ra tio n a nd Sta rg a rdt’ s Dise a se a ffe c t ne a rly 30 millio n

pe o ple in the US a nd E U, e xpe c te d to g ro w b y 50% o ve r ne xt 10 ye a rs

Wor ld- c lass c e ll biology c apabilitie s

  • ACT

sc ie ntific te a m c o ntinue s to de ve lo p b e st-in-c la ss, a nd o nly-in-c la ss c e ll line s whic h c re a te se ve ra l pre c linic a l o ppo rtunitie s a c ro ss ma ny dise a se a re a s

Str

  • ng Inte lle c tual Pr
  • pe r

ty E state

  • 190 pa te nts file d, 37 issue d whic h pro vide ACT

with susta ina b le c o mpe titive a dva nta g e fo r de c a de s to c o me

Ac tive par tne r ing pr

  • gr

am to mone tize non- c or e asse ts

  • ACT

ma na g e me nt is de dic a te d to mo ne tizing no n-c o re pre -c linic a l a sse ts a nd c o ntinue s to o ptimize po rtfo lio o f o ppo rtunitie s to fue l future c linic a l pro g ra ms

Se ve r al Ke y Infle c tion Points Pr

  • vide Ne ar
  • te r

m and L

  • ng- te r

m Gr

  • wth Oppor

tunitie s

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SLIDE 4

4 Pre-clinical/ in vitro POC – Animal Studies IND Approved Phase I

Phase II Phase III

Approval

Dry AMD SMD MMD

Pho to - re c e pto rs

Ga ng lio n Ne uro ns Co rne a Pla te le ts

Me se nc hymal Ste m Ce lls

Robust De ve lopme nt Pipe line Pr

  • vide s Multiple Oppor

tunitie s to Comme r c ialize and Par tne r

Po te ntia l Go v’ t F und ing

F irst Prio rity Ba se d On Curre nt F unding Adva nc e into Pha se I a nd Pa rtne r

Ba se d o n POC re sults, pursue a ppro pria te fund ing a nd c o lla b o ra tio ns

Ophthalmology Pr

  • gr

ams

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SLIDE 5

T he Re tina the lig ht-se nsitive tissue lining the inne r surfa c e o f the e ye

Re tina

Struc ture of the Re tina

5

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SLIDE 6

Provide s nutrie nts a nd g ro wth fa c to rs

  • pho to re c e pto rs se e no b lo o d

Re c yc le s Vita min A

  • ma inta ins pho to re c e pto r e xc ita b ility

De toxifie s pho to re c e pto r la ye r Ma inta ins Bruc h’ s Me mb ra ne

  • na tura l a ntia ng io g e nic b a rrie r
  • immune privile g e o f re tina

Absorbs stra y lig ht / pro te c ts fro m UV

RPE L a ye r ha s

multiple c r itic al r

  • le s

in the

he alth and func tion

  • f pho to re c e pto rs a nd

the re tina a s a who le .

L ife Support for Photore c e ptors

6

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SLIDE 7

F a ilure o f RPE c e lls re sults in ma ny

de g e ne ra tive dise a se s

Sta rg a rdt’ s dise a se Myo pic Ma c ula r Dystro phy Ag e -re la te d ma c ula r de g e ne ra tio n (AMD)

L ife Support for Photore c e ptors

7

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SLIDE 8

8

Pr e vale nc e of AMD Inc r e ase s E xpone ntially with Age

Data fro m http:/ / www.ne i.nih.g o v/ e ye data/ and U.S . Ce nsus Bure au Pub lic atio n “65+ in the Unite d State s”, P23-209

E xpo ne ntia l rise in pre va le nc e a nd inc ide nc e ra te s with a g e , with pre va le nc e ra te s o f la te AMD q ua drupling pe r de c a de

133.7 142.2 173.4 202.7 207.4 205.5 37.4 52.9 64.8 81.9 105.3 121

80+ 65-79 2000 2010 2020 2030 2040 2050 De ve lo pe d Co untrie s

“ma c ula r de g e ne ra tio n will so o n ta ke o n a spe c ts o f a n e pide mic ”

  • fo rme r Dire c to r o f the Na tio na l E

ye Institute Dr Ca rl K upfe r 60% 50% 40% 30% 20% 10% 50-59 40-49 60-69 70-79 80+ Ag e Inte rme diate AMD L a te AMD % Pre va le nc e (U.S.)

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SLIDE 9

9

Pr e vale nc e of AMD Inc r e ase s E xpone ntially with Age

E xpo ne ntia l rise in pre va le nc e a nd inc ide nc e ra te s with a g e , with pre va le nc e ra te s o f la te AMD q ua drupling pe r de c a de

T he c urre ntly 30 Million Ame ric a n a nd E urope a n AMD pa tie nts a re proje c te d to be c ome 45 Million pa tie nts by 2025

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SLIDE 10

RPE T he r apy- Rationale

  • Ma ssive unme t me dic a l ne e d
  • Sma ll dosa g e size

– le ss tha n 200K c e lls

  • Immune - privile g e d site

– minima l immuno suppre ssio n

  • E

a se of a dministra tion

– no se pa ra te de vic e a ppro va l

  • Unique me a suring a nd obse rva tion e nvironme nt

10

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SLIDE 11

Sur gic al Ove r vie w

11

Proc e dure:

  • 25 Ga ug e Pa rs Pla na Vitre c to my
  • Po ste rio r Vitre o us Se pa ra tio n
  • Sub re tina l hE

SC-de rive d RPE c e lls inje c tio n

  • Ble b Co nfirma tio n
  • Da y Surg e ry/ Se da tio n o nly
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SLIDE 12

Jules Stein (UCLA) Mass Eye & Ear Infirmary Wills Eye Institute Bascom Palmer Eye Institute Moorfields Eye Hospital

Edinburgh Royal Infirmary

Wor ld r e nowne d le ade r ship to he lp us navigate the c linic al path and ultimate ly suppor t mar ke t launc h Clinic al T r ials be ing le d by Wor ld L e ade r s in Ophthalmology

12

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SLIDE 13
  • No Adve r

se E ve nts

  • Pe r

siste nc e of c e lls

  • Impac t on Ac uity

Re c o rde d func tio na l visua l impro ve me nts in ma jo rity o f pa tie nts.

  • I

nc re a se d le tte rs o n E T DRS Cha rts

  • Co lo r pe rc e ptio n
  • Co ntra st
  • L
  • w lig ht visio n

Phase I T r ials E xc e e ding E xpe c tations – no adve r se e ve nts and pe r siste nc e of c e lls

13

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SLIDE 14

IND Appr

  • ve d

50% Pa tie nt E nr

  • llme nt

100% Pa tie nt E nr

  • llme nt

U.S. – Dr y AMD U.S. – SMD U.K. – SMD U.S. – MMD

12/16 patie nts tre ate d 10/16 patie nts tre ate d 8/12 patie nts tre ate d E nro lling – 12 patie nts to tal

2 ye a rs sinc e the first pa tie nts we re tre a te d

Me asur able Impr

  • ve me nts in Visual

Ac uity for Major ity of T r e ate d Patie nts

Ac tive Clinic al Pr

  • gr

ams in AMD and SMD Indic ate E nc our aging Re sults

14

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SLIDE 15

Ja nua ry 2013: F DA a ppro ve d a dditio na l 4 pa tie nt “b e tte r visio n” c o ho rts in e a c h tria l.

F

  • r Co ho rt 2a – c a n e nro ll pa tie nts with visio n a s

g o o d a s 20/ 100.

Co ho rt 1

50K Ce lls

Co ho rt 2

100K Ce lls

Co ho rt 3

150K Ce lls

Co ho rt 4

200K Ce lls

Co ho rt 2a

100K Ce lls

F irst T re a tme nts informe d a more a g g re ssive stra te g y to tre a t “be tte r vision” c ohort, c ould le a d to broa de r la be l a nd/ or e a rlie r a pprova l

15

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SLIDE 16

Se ve ra l Importa nt c linic a l mile stone s 2H 2013, 1H 2014

150K 200K

Co ho rt 2a

100K

Ja n 2014 Ja n 2015 Dry AMD & SMD T ria ls

Pa tie nt fo llo w-up PII de sig n Pa tie nt T re a tme nt

Pha se II

Pa tie nt T re a tme nt

MMD T ria l 16 Se p 2013 July 2014

Pha se I Pha se I

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SLIDE 17

E xpanding Clinic al Pr

  • gr

ams

17

Myo pia c re a te s a hig he r risk o f pe rma ne nt visio n lo ss due to Myopic Ma c ula r De g e ne ra tion (MMD)

  • Se ve re ne a r-sig hte dne ss c a use s e lo ng a tio n o f the e ye b a ll --

whic h c a n c a use fissure s in RPE la ye r.

Januar y 2013 - F DA Appr

  • ve d

MMD Phase I/ II study Jule s Ste in E ye I nstitute (UCL A) a nd ACT

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SLIDE 18

Inte lle c tual Pr

  • pe r

ty – RPE Pr

  • gr

am

Dominant Pate nt Position for T r e ating Re tinal De ge ne r ation Br

  • ad Cove r

age for Manufac tur ing RPE Ce lls Br

  • ad pr
  • te c tion of phar

mac e utic al pr e par ations

  • RPE

c e ll suspe nsio ns

  • sc a ffo lde d RPE

la ye rs.

RPE Ce lls de r ive d fr

  • m othe r

plur ipote nt ste m c e lls

Vig ila nt filing on improve me nts

18

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SLIDE 19

T he r ape utic Pipe line

19

Re tina l Ne ura l Prog e nitor c e lls & Isola te d Prote c tive F a c tors

Pho to re c e pto r L

  • ss, Mo dula tio n o f Mülle r Ce lls

Pro te c tio n o f Re tina l Ga ng lio n c e lls (Gla uc o ma )

Corne a l E ndothe lium

Co rne a l Dise a se

He ma ng iobla st c e lls

I

sc he mic re tino pa thy – dia b e tic re tino pa thy, va sc ula r o c c lusio ns

Me se nc hyma l Stroma l Ce lls

Oc ula r - Gla uc o ma , Uve itis, Re tinitis Pig me nto sa Auto immune Dise a se s I

nfla mma to ry Dise a se s o r diso rde rs

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SLIDE 20

20

BY PRODUCI

NG T HE

HIGHE ST

DE

GRE E OF QUAL IT Y

VE T E RI NARY T HE RAPE UT I CS WE I NT E ND T O BE A L E ADE R I N

VE

T E RINARY

R

E GE NE RAT IVE ME DICINE

Se pte mb e r 19, 2013 – “ACT F ile s I nve stig a tio na l Ne w Anima l Drug (I NAD) Applic a tio n with F DA to T re a t 10 Diffe re nt Dise a se I ndic a tio ns Using Pluripo te nt Ste m Ce lls”

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SLIDE 21

ACT Cor por ate Ove r vie w

21

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SLIDE 22

Ga ry Ra b in – Cha irma n a nd CE O E d wa rd Myle s – CF O a nd E VP o f Co rp De ve lo pme nt

  • Dr. Ma tthe w Vinc e nt, Ph.D. – Dire c to r o f Busine ss

De ve lo pme nt

  • Dr. Ro b e rt L

a nza , MD – Chie f Sc ie ntific Offic e r

  • Dr. Irina K

lima nska ya , Ph.D. – Dire c to r o f Ste m Ce ll Bio lo g y

  • Dr. Shi-Jia ng (Jo hn) L

u, Ph.D. – Se nio r Dire c to r o f Re se a rc h E d mund Mic kuna s – Vic e Pre sid e nt o f Re g ula to ry Affa irs

  • Dr. Ro g e r Ga y, Ph.D. - Se nio r Dire c to r o f Ma nufa c turing

Prove n busine ss le a de rs who c a n de ve lop a nd imple me nt c orpora te stra te g y a nd mone tize a sse ts to ma ximize sha re holde r va lue World- re nowne d sc ie ntific thoug ht le a de rs pushing the c utting e dg e of sc ie nc e to de ve lop importa nt the ra pie s

De e p e xpe r ie nc e navigating the c omple x r e gulator y landsc ape fr

  • m

de ve lopme nt to mar ke t

GMP manufac tur ing to e nsur e the highe st quality pr

  • duc ts ar

e de live r e d to our patie nts

An E xpe r ie nc e d and De dic ate d Manage me nt T e am

22

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SLIDE 23

Mic ha e l He ffe rna n CE O – Co lle g ium Pha rma c e utic a ls Ro b e rt S. L a ng e r, Sc .D. Institute Pro fe sso r, MIT Zo ha r L

  • shitze r

CE O – Pre sb ia , Inc ., & Princ ipa l in Oc ha rd Ca pita l Gre g Pe rry E VP & CF O - Immuno g e n Ala n C. Sha piro F ina nc e Pro fe sso r a nd Cha irma n o f the De pa rtme nt o f F ina nc e a nd Busine ss E c o no mic s (re tire d ) – Unive rsity o f So uthe rn Ca lifo rnia Ga ry Ra b in Cha irma n a nd CE O – Ad va nc e d Ce ll T e c hno lo g y

A World- Cla ss Boa rd of Dire c tors

23

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SLIDE 24

T ha nk yo u F

  • r mo re info rma tio n, visit www.a dva nc e dc e ll.c o m