More than one bil­lion peo­ple world­wide live with vi­sion im­pair­ment or blind­ness¹ and this num­ber is ex­pect­ed to rise.

“With peo­ple liv­ing longer, pro­gres­sive and ir­re­versible vi­sion loss from reti­nal dis­eases is be­com­ing an in­creas­ing­ly ur­gent glob­al health chal­lenge,” said Jo­taro Suzu­ki, Pri­ma­ry Fo­cus Lead, Blind­ness and Re­gen­er­a­tion, Astel­las Phar­ma. “Cur­rent treat­ments fo­cus on dis­ease man­age­ment. How­ev­er, re­gen­er­a­tive med­i­cine such as cell and gene ther­a­py of­fers a promis­ing new path­way for restor­ing sight and free­ing pa­tients from the fear of de­te­ri­o­rat­ing vi­sion.”

Astel­las be­lieves one po­ten­tial an­swer to vi­sion loss lies in ‘off-the-shelf’ al­lo­gene­ic cell ther­a­pies – where cells from a sin­gle donor can be used to treat mul­ti­ple pa­tients. This con­trasts with au­tol­o­gous ap­proach­es, which re­ly on cells be­ing sourced from and used in each in­di­vid­ual pa­tient in need of treat­ment. The scal­a­bil­i­ty of al­lo­gene­ic ther­a­pies en­ables larg­er-scale man­u­fac­tur­ing with re­duced cost and com­plex­i­ty, mak­ing these treat­ments more ac­ces­si­ble to pa­tients in need.

At the fore­front of al­lo­gene­ic ther­a­pies are pluripo­tent stem cells (PSCs). PSCs are high­ly re­plen­ish­able, en­abling large-scale man­u­fac­tur­ing, as well as al­so be­ing pro­gram­ma­ble, giv­ing them the po­ten­tial to dif­fer­en­ti­ate in­to near­ly any cell type of the hu­man body. These at­trib­ut­es make them a promis­ing foun­da­tion to cre­ate cell ther­a­pies across a wide range of dis­ease ar­eas, in­clud­ing car­di­ol­o­gy, im­munol­o­gy and neu­rol­o­gy.

“By tak­ing an al­lo­gene­ic ap­proach, we hope to achieve our goal of de­vel­op­ing ef­fec­tive cell ther­a­pies that are wide­ly ac­ces­si­ble to as many pa­tients as pos­si­ble,” said Erin Kim­brel, Head, Cell and Gene Ther­a­py Re­search, Astel­las Phar­ma. “We are cur­rent­ly fo­cus­ing our ef­forts in oph­thal­mol­o­gy, where we be­lieve al­lo­gene­ic ther­a­pies could of­fer mean­ing­ful so­lu­tions to ad­dress com­plex, dif­fi­cult-to-treat con­di­tions that can re­sult in vi­sion loss.”

One ex­am­ple of where al­lo­gene­ic cell ther­a­py is show­ing promise is in the treat­ment of ge­o­graph­ic at­ro­phy (GA). GA is a lead­ing cause of vi­sion loss that re­sults from dam­age to the reti­nal pig­ment ep­ithe­li­um – pro­tec­tive cells that main­tain the health of the reti­na, the light sen­si­tive part of the eye that en­ables vi­sion. It cur­rent­ly af­fects about five mil­lion peo­ple world­wide,² with this num­ber ex­pect­ed to rise to more than 18 mil­lion by 2040.³

Astel­las aims to re­store reti­nal func­tion and ad­dress vi­sion loss by re­plac­ing cells dam­aged by GA. Our lead pro­gram is ex­plor­ing the trans­plan­ta­tion of healthy reti­nal pig­ment ep­ithe­li­um cells in­to the eye. Tar­get­ing dis­ease process­es at the cel­lu­lar lev­el and pro­mot­ing the re­gen­er­a­tion or re­place­ment of dam­aged tis­sues could of­fer a long-term so­lu­tion for trans­form­ing pa­tient out­comes.

“We’re proud to have pi­o­neered the first oph­thalmic al­lo­gene­ic pluripo­tent-based ther­a­py to en­ter clin­i­cal tri­als and re­main deeply com­mit­ted to ad­vanc­ing its de­vel­op­ment,” said Mar­ci Eng­lish, Head, Bio­Phar­ma and Oph­thal­mol­o­gy De­vel­op­ment, Astel­las Phar­ma.

The path to clin­i­cal re­al­i­ty hasn’t been straight­for­ward. “As an emerg­ing area, al­lo­gene­ic cell ther­a­pies have faced many com­plex chal­lenges, like over­com­ing im­mune re­jec­tion, pre­serv­ing ge­nom­ic in­tegri­ty and achiev­ing man­u­fac­tur­ing con­sis­ten­cy,” said Eng­lish.

To­day, how­ev­er, the field is ma­tur­ing. Ad­vances in bi­ol­o­gy and cut­ting-edge tech­nolo­gies are over­com­ing bar­ri­ers, tran­si­tion­ing pluripo­tent-based al­lo­gene­ic cell ther­a­pies from a pi­o­neer­ing in­no­va­tion to a re­al-world so­lu­tion.

At Astel­las, sev­er­al in­no­v­a­tive tools and tech­nolo­gies are en­abling progress. For ex­am­ple, Uni­ver­sal Donor Cell tech­nol­o­gy, where­by PSCs are ge­net­i­cal­ly en­gi­neered in such a way that they avoid de­tec­tion by the im­mune sys­tem, al­low­ing them to be de­liv­ered to any pa­tient with­out the need for im­mune sup­pres­sion. Ad­di­tion­al­ly, in part­ner­ship with Yaskawa Elec­tric Cor­po­ra­tion, Astel­las is de­vel­op­ing a cell ther­a­py prod­uct man­u­fac­tur­ing plat­form uti­liz­ing the dual-arm ro­bot ‘Ma­ho­lo’.

“Our hu­manoid ro­bot Ma­ho­lo helps us ad­dress key chal­lenges in pluripo­tent stem cell-based drug dis­cov­ery, in­clud­ing cul­ture and dif­fer­en­ti­a­tion, han­dling and man­u­fac­tur­ing au­toma­tion,” Kim­brel said. “By in­te­grat­ing AI and ro­bot­ics, we’re cre­at­ing a sys­tem de­signed to de­liv­er high-qual­i­ty pluripo­tent stem cell-based ther­a­pies with speed and pre­ci­sion.”

“As one of few com­pa­nies fo­cused on al­lo­gene­ic cell ther­a­py, we’re deeply in­vest­ed in re­al­iz­ing its break­through po­ten­tial, and we know this de­mands ro­bust in­fra­struc­ture,” said Kim­brel. “We've com­mit­ted to a long-term strat­e­gy, sys­tem­at­i­cal­ly build­ing end-to-end ca­pa­bil­i­ties across our sites in the U.S. and Japan, from dis­cov­ery through de­vel­op­ment and com­mer­cial­iza­tion.”

The Astel­las In­sti­tute for Re­gen­er­a­tive Med­i­cine (AIRM) in West­bor­ough, MA, is a glob­al hub com­bin­ing both R&D and Good Man­u­fac­tur­ing Prac­tice ca­pa­bil­i­ties, fa­cil­i­tat­ing the de­vel­op­ment of new re­gen­er­a­tive med­i­cine and cell-based ther­a­pies while scal­ing man­u­fac­tur­ing for fu­ture com­mer­cial­iza­tion needs. Ad­di­tion­al sites in Seat­tle, WA, San Fran­cis­co, CA, and Tsuku­ba, Japan sup­port this in­te­grat­ed ap­proach.

Cell ther­a­pies could of­fer a par­a­digm shift for pa­tients liv­ing with cur­rent­ly hard-to-treat de­gen­er­a­tive con­di­tions, in­clud­ing in oph­thal­mol­o­gy. With a fo­cus on al­lo­gene­ic ther­a­pies, Astel­las is pi­o­neer­ing “off-the-shelf” ap­proach­es with the po­ten­tial to make cell ther­a­py a scal­able and ac­ces­si­ble op­tion for more pa­tients.

“De­spite the known chal­lenges, we per­se­vere be­cause we see great promise for pa­tients and the op­por­tu­ni­ty to change the path of dis­eases with high un­met need, like GA,” said Suzu­ki. “At Astel­las, we con­tin­ue to in­no­vate in cell ther­a­py to tack­le reti­nal dis­eases, as we pur­sue ther­a­pies that could trans­form the lives of pa­tients.”

Learn more about how Astel­las is ad­vanc­ing cell ther­a­py to trans­form care for reti­nal dis­ease and be­yond here.

¹ Bourne, R et al. The Lancet Glob­al Health. 9(2): e130 - e143. Ac­cessed via the IAPB Vi­sion At­las https://www.iapb.org/learn/vi­sion-at­las. Last ac­cessed Sep­tem­ber 2025.

² Boy­er DS et al. Reti­na. 2017;37(5):819-835.

³ Wong WL et al. Lancet Glob Health. 2014;2(2):e106–e116.

A fi­bro­sis biotech saw its stock price crater Mon­day morn­ing af­ter it re­port­ed a Phase 2 fail­ure for its lead pro­gram.

Tvar­di Ther­a­peu­tics said the ex­per­i­men­tal drug, called TTI-101, did not show a dif­fer­ence from place­bo in an 88-pa­tient study for id­io­path­ic pul­monary fi­bro­sis (IPF). More than half of the pa­tients tak­ing TTI-101 dropped out of the tri­al, pri­mar­i­ly due to gas­troin­testi­nal side ef­fects.

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