Cellectis提交UCART19疗法的首个临床申请

林辉

Cellectis公司12月23日宣布向英国药品和健康产品管理局（MHRA）提交了UCART19疗法用于治疗CD19阳性急性淋巴细胞白血病的临床申请。这是异体移植CAR T 治疗CD19阳性ALL的第一个临床试验。# E' q* K3 A8 d! z5 g$ y0 L% h

; C9 N: d; a/ \5 tUCART19是利用Cellectis公司专有的同种异体方法（allogeneic）开发而成的CAR-T疗法，这种方法基于Talen基因编辑技术，不需要依据患者进行相应修饰，而是直接将来源于非患者(non-patient) 供体的T细胞进行工程化，用于多个患者的治疗。
. ~. \( w7 q  y+ R9 k. @& r
0 F0 s) y8 y% n1 d( _0 |4 D诺华、Kite制药、Juno的CAR-T第一梯队均采用的是自体移植方法(autologous)，即将来自患者自身的T细胞进行工程化，用于患者自身肿瘤的治疗。  m. T# e0 F$ D" Z! ]4 J

) i( q! X  C7 y5 s这也意味着，相比其他竞争对手的CAR-T产品，Cellectis公司的CAR-T产品将具有普遍适用性，而且能够工业化生产获得标准化的现成产品，同时这些产品可被冷冻运往全球任何地方立即使用。, u9 `, l- i. i+ }

9 \& h* @9 a- F2 Z- V8 x2015年11月18日，施维雅/辉瑞以$3820万预付款+$3亿里程金获得UCART19的全球开发权利。2 _8 b8 b. r7 \- p1 _0 \
! c; S8 @: ?" w' ^+ V' J5 P/ h2 Y, N; m
Cellectis Files First Clinical Trial Application for UCART19, an Allogeneic Gene Edited CAR T-Cell Product for Hematological Malignancies
  J3 B) E6 Y6 o$ M6 d" ?% P; s! |' s0 d, s$ o( ^! k
December23, 2015 05:00 PM Eastern Standard Time
/ g: D( q# k) v2 z
+ [& D9 J) O2 j0 I4 Y" L8 ZCellectis(Paris:ALCLS) (NASDAQ:CLLS) (Alternext: ALCLS – Nasdaq: CLLS) today announcedthe submission of a clinical trial application (CTA) to the Medicines &Healthcare products Regulatory Agency (MHRA) requesting approval to initiateUCART19 First-in-Human clinical investigation in leukemia in the United Kingdom.* M6 C' P7 i( D0 A) W

+ t* i* c. w% u5 z7 G) fThis study aims to include CD19-positive Acute Lymphoblastic Leukemia (ALL)patients. Other eligibility criteria to enter clinical trials will be assessedby the investigators.4 M1 U! h+ Z( u% Q- N: D. N

7 s3 g: [- I( W. F. f6 d6 e“It has been aprivilege preparing this application with our team, partners, investigators and subcontractors, in close interaction with MHRA, rewarding many years of intensework to overcome the challenges that are inherent to advanced therapy medicinalproducts. This achievement marks an important step toward making UCART19available to patients,” said Stephan Reynier, Chief Regulatory and ComplianceOfficer, Cellectis.
' @: U# k6 v  P$ s, x& ~6 {3 } 5 n0 @0 }! Y( U4 K$ F
"The UCART19 CTA filing is a great recognition for the Company’s preclinical andmanufacturing accomplishments in developing a therapeutic for AcuteLymphoblastic Leukemia. We are all pleased with Cellectis’ progress to datewith UCART19, including the filing of this CTA, and we look forward tofollowing the progress of this program through the course of its clinicaldevelopment," said Dr. Mathieu Simon, EVP, Chief Operating Officer,Cellectis.
# V0 Y: Q5 L: w/ Q: M ( y  _) b0 f7 E1 J3 N
About UCART19
) x0 z6 u' O$ T$ E0 d; k
7 e  V. Y0 Z0 s( D0 iUCART19 is a potential best-in-class allogeneic TALEN® gene edited T-cell product fortreatment of CD19 expressing hematological malignancies, initially developed inChronic lymphocytic leukemia (CLL) and Acute lymphoblastic leukemia (ALL).Engineered allogeneic CD19 CAR T-cells currently stand out as a realtherapeutic innovation for treating various types of leukemia and lymphoma. Cellectis’ approach with UCART19 is based on the preliminary positive results fromclinical trials using products based on the CAR technology and has thepotential to overcome the limitation of the autologous current approach by providing an allogeneic frozen, “off the shelf” T-cell based medicinal product.6 O$ j5 l: q1 K0 P

+ n3 M$ e, w) l% M4 e2 N2 COn November 18, 2015 Servier exercised its worldwide option to license UCART19 andentered into a global development and commercialization collaboration withPfizer on UCART19. According to their recent agreement, Cellectis will handover the clinical development of UCART19 to Servier and their US partnerPfizer. Due to the early exercise, Cellectis is no longer responsible forfunding the UCART19 Phase I clinical program.: I) j7 z! @0 m4 B, Q0 g4 [( O
1 p% p5 V- I: w& C; L( ^5 D  \
Information about ongoing clinical trials are publically available on dedicated websitessuch as:
3 I0 D) R6 P3 t" R0 D. H. g5 N  x# twww.clinicaltrials.govin the U.S.
3 W- O6 F( G6 l6 k: S& K  `www.clinicaltrialsregister.euin Europe
2 @' h/ b' O- K1 t
# i* h7 ^: j" f+ k' d, j- J! }+ ~About Cellectis
. N# w1 a$ s' P; ~3 @
; q5 X( w( B5 Z$ BCellectisis a biopharmaceutical company focused on developing immunotherapies based ongene edited engineered CAR-T cells (UCART). The company’s mission is to developa new generation of cancer therapies based on engineered T-cells. Cellectiscapitalizes on its 15 years of expertise in genome engineering - based on itsflagship TALEN® products and meganucleases and pioneering electroporationPulseAgile technology - to create a new generation of immunotherapies. CARtechnologies are designed to target surface antigens expressed on cells. Usingits life-science-focused, pioneering genome-engineering technologies,Cellectis’ goal is to create innovative products in multiple fields and withvarious target markets. Cellectis S.A. is listed on the Nasdaq Global Market(ticker: CLLS) and on the NYSE Alternext market (ticker: ALCLS). To find outmore about us, visit our website: www.cellectis.com.+ ]' n  `  W. A( X

* p* N1 z9 T) E5 I$ F3 sTalking about gene editing? We do it.$ ~8 T& P" I1 w; {. S7 G* ]4 Y2 W
9 j4 h1 R# j+ C* X
TALEN®is a registered trademark owned by the Cellectis Group.
- p2 i) A# @5 J2 _  k3 [
$ `: Z. p% K3 V  l1 Y& P, Q/ x+ UDisclaimer; _2 }" I# m# Q( ^8 H

, o+ R- R2 d0 ]. b7 A  q" xThis press release contains “forward-looking” statements that are based on ourmanagement’s beliefs and assumptions and on information currently available tomanagement.9 Q+ o7 g/ z- v$ _, s- I. t

9 m, j9 d* ~1 o# w+ FForward-lookingstatements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed orimplied by the forward-looking statements.
2 s) \; O5 _- @- I/ S
; o2 f+ ?2 s8 P! O9 o# Q% sThe risks and uncertainties include, but are not limited to the risk of notobtaining regulatory approval to commence clinical trials on our UCART productcandidates, including UCART19, the risk that our collaboration with Servier orour relationships with the principal investigator will not continue or will notbe successful, and the risk that any one or more product candidates will not besuccessfully developed and commercialized.* ?- C  Q' `. w1 d

: i% g1 n( _. J1 K8 L$ Z2 KYou should read the Company’s Prospectus, including the Risk Factors setforth therein and the exhibits there to, completely and with the understandingthat our actual future results may be materially different from what we expect.Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-lookingstatements, even if new information becomes available in the future.

wdywrl

Amazing!

roomofxw

异体的T细胞也不是可以无限增殖的吧？

天狼星1919

UCART是怎么进行基因改造的有公布过吗？？