Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Re­mark­able ad­vances in cell and gene ther­a­py over the last decade of­fer un­prece­dent­ed ther­a­peu­tic promise and bring new hope for many pa­tients fac­ing dis­eases once thought in­cur­able. How­ev­er, for cell and gene ther­a­pies to reach their full po­ten­tial, re­searchers, man­u­fac­tur­ers, life sci­ence com­pa­nies, and aca­d­e­mics will need to work to­geth­er to solve the sig­nif­i­cant chal­lenges fac­ing the in­dus­try.

While time to mar­ket and pro­duc­tion cost head the list of pri­or­i­ty con­cerns for near­ly every in­dus­try, sev­er­al com­mon con­tribut­ing fac­tors, top-of-mind for users, in­clude scal­a­bil­i­ty, au­toma­tion, and cell type flex­i­bil­i­ty. Corn­ing Life Sci­ences has a long his­to­ry of ad­vanc­ing ad­her­ent plat­form tech­nolo­gies and work­ing with in­dus­try in­no­va­tors, such as Cel­lipont Bioser­vices, to help en­able ef­fi­cient scale-up strate­gies in cell and gene ther­a­py pro­duc­tion. Most re­cent­ly, Corn­ing launched the Corn­ing® As­cent^TM Fixed Bed Biore­ac­tor sys­tem, the lat­est re­sult of cus­tomer col­lab­o­ra­tions and tech­no­log­i­cal in­no­va­tions in this area.

As a lead­ing life sci­ences com­pa­ny, Corn­ing is ded­i­cat­ed to ac­cel­er­at­ing in­no­va­tion and solv­ing cus­tomer chal­lenges. From ear­ly re­search to pro­duc­tion, there are as­pects of the cell cul­ture process that can great­ly in­flu­ence time to mar­ket and cost. Three of the most press­ing chal­lenges are out­lined here:

• Scal­a­bil­i­ty—process­es should be able to move from the lab to pro­duc­tion while min­i­miz­ing tech­nol­o­gy trans­fer and re-de­vel­op­ment steps as much as pos­si­ble. Hav­ing to move from ad­her­ent plat­forms to sus­pen­sion to achieve de­sired scale can re­quire ad­di­tion­al de­vel­op­ment and op­ti­miza­tion steps, as well as time and costs.
• Au­toma­tion—sys­tems should be as au­to­mat­ed as pos­si­ble. Lack of mon­i­tor­ing ca­pa­bil­i­ties and au­to­mat­ed process­es can re­sult in greater over­head costs via in­creased staff and the op­por­tu­ni­ty for hu­man er­ror.
• Cell type flex­i­bil­i­ty—cus­tomers work­ing across ad­vanced ther­a­peu­tic modal­i­ties re­quire sys­tems that give them flex­i­bil­i­ty to work with var­i­ous cell types and ap­pli­ca­tions (e.g., stem cells, cells for vi­ral vec­tor pro­duc­tion, etc.). Lack of flex­i­bil­i­ty dri­ves up costs as com­pa­nies must in­vest in and main­tain sev­er­al pro­duc­tion plat­forms.

Aim­ing to ad­dress scal­a­bil­i­ty, au­toma­tion, and cell type flex­i­bil­i­ty chal­lenges from the ear­li­est in­cep­tions of projects is key to im­prov­ing the like­li­hood of lat­er suc­cess and ef­fi­cien­cy, es­pe­cial­ly when se­lect­ing plat­form tech­nolo­gies for de­vel­op­ment of ad­vanced ther­a­pies. Corn­ing has been at the fore­front of ad­her­ent cell cul­ture plat­forms since the in­tro­duc­tion of the well-known Corn­ing High Yield Per­for­mance (HY­PER) tech­nol­o­gy more than a decade ago, and con­tin­ues that com­mit­ment to in­creas­ing cell den­si­ty with the As­cent FBR sys­tem, which in­cludes the in-mar­ket Process De­vel­op­ment plat­form and forth­com­ing Pi­lot and Pro­duc­tion sys­tems.

Re­cent­ly, fixed-bed re­ac­tor tech­nolo­gies have marked an im­por­tant step in ad­dress­ing the most lim­it­ing hur­dles of ad­her­ent plat­forms. The As­cent FBR sys­tem is an in­ten­si­fied, high-yield cell cul­ture plat­form de­signed to en­able lin­ear scal­a­bil­i­ty—from process de­vel­op­ment and seed train to pro­duc­tion scale. It is al­so ful­ly au­to­mat­ed, in­clud­ing key process pa­ra­me­ter mon­i­tor­ing, bring­ing one of the most sig­nif­i­cant ben­e­fits of sus­pen­sion plat­forms to ad­her­ent cell cul­ture. Fi­nal­ly, the As­cent FBR sys­tem’s vi­able cell har­vest ca­pa­bil­i­ty en­ables the sys­tem to sup­port mul­ti­ple cell types and ap­pli­ca­tions, in­clud­ing both ‘cell as a prod­uct’ (e.g., stem cell ther­a­pies) and ‘cell as a pro­duc­er’ (e.g., vi­ral vec­tors) pro­duc­tion process­es.

Zara Melk­oumi­an, Ph.D., Busi­ness Tech­nol­o­gy Di­rec­tor, and the As­cent FBR sys­tem

“When our da­ta re­vealed we could have a sig­nif­i­cant im­prove­ment in AAV vec­tor yield when us­ing the As­cent FBR Sys­tem, we got quite ex­cit­ed. The val­i­da­tion from our be­ta cus­tomers con­firms the tremen­dous val­ue and po­ten­tial of the plat­form to sup­port crit­i­cal break­throughs.”

—Zara Melk­oumi­an, Ph.D., Busi­ness Tech­nol­o­gy Di­rec­tor, Corn­ing Life Sci­ences

By en­abling cus­tomers to cen­tral­ize their work­flows in a sin­gle plat­form, the As­cent FBR sys­tem is help­ing to stan­dard­ize work­flows that make the trans­la­tion to pro­duc­tion eas­i­er. These ben­e­fits to­geth­er can of­fer a cu­mu­la­tive process ad­van­tage when choos­ing the As­cent FBR sys­tem and can po­ten­tial­ly dri­ve in­no­va­tion for­ward at a faster pace, for more di­verse ap­pli­ca­tions, and at a low­er cost.

Cel­lipont Bioser­vices (for­mer­ly Per­for­mance Cell Man­u­fac­tur­ing), a com­pa­ny known for its ex­per­tise in cell ther­a­py de­vel­op­ment and man­u­fac­tur­ing, has re­cent­ly col­lab­o­rat­ed with Corn­ing to as­sess new tech­nolo­gies. Cel­lipont helps their clients in­cor­po­rate best-in-class ca­pa­bil­i­ties, fa­cil­i­tat­ing scale up from bench­top, mov­ing through clin­i­cal stud­ies, and in the near fu­ture, sup­port­ing com­mer­cial­iza­tion with hopes of ex­pand­ing ac­cess to life sav­ing med­i­cines to pa­tients across the world. Most re­cent­ly, Cel­lipont and Corn­ing ap­pli­ca­tions sci­en­tists have been work­ing close­ly to eval­u­ate the As­cent FBR plat­form as an emerg­ing tech­nol­o­gy ca­pa­ble of meet­ing the need for scal­able bio­man­u­fac­tur­ing.

As a mas­ter of many trades, in­clud­ing the man­u­fac­tur­ing of stem cells, chimeric anti­gen re­cep­tor T cells, den­drit­ic cell ther­a­pies, and plas­ma de­rived ther­a­py prod­ucts, Cel­lipont has an eye for in­no­v­a­tive, flex­i­ble sys­tems.

“We are al­ways look­ing to present new tech­no­log­i­cal so­lu­tions to our clients as we po­si­tion our­selves as part­ners in their sci­en­tif­ic ef­forts. We see a lot great of po­ten­tial in the As­cent FBR sys­tem, in­clud­ing ef­fi­cient scale up and cost sav­ings both from a con­sum­ables per­spec­tive and a hands-on per­spec­tive—all in a small foot­print. The fact that it is a closed sys­tem is al­so crit­i­cal­ly im­por­tant.”

—Car­olyn Wright­son, Chief Tech­nol­o­gy Of­fi­cer, Cel­lipont Bioser­vices

Corn­ing works close­ly with cus­tomers like Cel­lipont to un­der­stand the unique goals, process­es, and un­met needs of those us­ing their prod­ucts, and to pro­vide cus­tomized so­lu­tions. Field ap­pli­ca­tions sci­en­tists work hand-in-hand with labs to over­come chal­lenges and to help en­sure that users are eas­i­ly and ef­fec­tive­ly in­cor­po­rat­ing new tech­nolo­gies, from HY­PER­Flasks® to the As­cent FBR plat­form, in­to their work­flows.

With deep in­dus­try knowl­edge and more than 100 years of ex­pe­ri­ence, Corn­ing re­mains com­mit­ted to fur­ther­ing cus­tomers’ ca­pa­bil­i­ties in ad­her­ent cell cul­ture. By bring­ing to mar­ket nov­el tech­nolo­gies that can of­fer an ef­fi­cient scale-up strat­e­gy from lab to pro­duc­tion, Corn­ing is work­ing close­ly with de­vel­op­ers and man­u­fac­tur­ers to help them bring ther­a­pies to mar­ket more quick­ly and ef­fi­cient­ly. Cell and gene ther­a­py is at a crit­i­cal in­flec­tion point, and through such cross-in­dus­try col­lab­o­ra­tion, it is poised to re­al­ize ther­a­peu­tic re­al­i­ties the world has on­ly imag­ined.