View ValuationCRISPR Therapeutics 향후 성장Future 기준 점검 5/6CRISPR Therapeutics (는) 각각 연간 69.7% 및 70.1% 수익과 수익이 증가할 것으로 예상됩니다. EPS는 연간 69.1% 만큼 성장할 것으로 예상됩니다. 자기자본이익률은 3년 후 -16.6% 로 예상됩니다.핵심 정보69.7%이익 성장률69.14%EPS 성장률Biotechs 이익 성장24.1%매출 성장률70.1%향후 자기자본이익률-16.60%애널리스트 커버리지Good마지막 업데이트05 Aug 2026최근 향후 성장 업데이트업데이트 없음모든 업데이트 보기Recent updatesReported Earnings • 23hSecond quarter 2026 earnings released: US$0.94 loss per share (vs US$2.40 loss in 2Q 2025)Second quarter 2026 results: US$0.94 loss per share (improved from US$2.40 loss in 2Q 2025). Revenue: US$10.2m (up US$9.29m from 2Q 2025). Net loss: US$91.2m (loss narrowed 56% from 2Q 2025). Revenue is forecast to grow 70% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in Europe.공고 • Apr 04CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026, at 08:00 W. Europe Standard Time. Location: at the offices of walder wyss ltd, seefeldstrasse 123, 8008 switzerland, zurich Switzerland공고 • Dec 24CRISPR Therapeutics Provides Broad Update on Zugocaptagene Geleucel in Autoimmune Disease and Hematologic MalignanciesCRISPR Therapeutics provided updates on zugocaptagene geleucel (zugo-cel), formerly known as CTX112™?, its investigational allogeneic CAR T targeting CD19, in development for autoimmune disease and hematologic malignancies. Autoimmune Disease: Zugo-cel, targeting CD19, is in an ongoing Phase 1 basket trial in autoimmune rheumatologic diseases, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis. Patients in the study may have active SLE (with or without renal involvement), SSc, or idiopathic inflammatory myopathy (IIM) despite the use of standard therapies. Preliminary clinical data from the Phase 1 study has been encouraging, and zugo-cel has been well tolerated to date. No Grade 3 ICANS or CRS has been observed at the 100 million cell dose, which is the dose currently being studied in the autoimmune basket trials. The Phase 1/2 clinical trial in R/R B-cell malignancies is ongoing. The Company expects to provide additional updates in the second half of 2026. CRISPR Therapeutics has also established a new collaboration and clinical supply agreement with Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program's development in oncology.공고 • Nov 09CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310CRISPR Therapeutics announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose. These data were presented during a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM) in a peer-reviewed article entitled "Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3." Phase 1 Clinical Trial Design The Phase 1, open label, dose-escalation trial evaluated single-course IV doses of CTX310 ranging from 0.1 to 0.8 mg/kg (lean body weight) targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous Family hypercholesterolemia (HeFH), or mixed dyslipidemias (elevated TG and LDL). Overall, CTX310 demonstrated a well-tolerated safety and tolerability profile that supports continued advancement of the program. These new results build upon previously disclosed top-line data from 12 participants across the first four potential cohorts, corresponding to lean body weight-based doses of DL1 [0.1 mg/kg, DL2 [0.3 mg/kg, DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg). Results from the Phase 1 clinical trial highlight the potential of CTX310 to safely and durably lower both TG and LDL following a single-course IV administration. CRISPR Therapeutics is advancing CTX310 into Phase 1b clinical trials, prioritizing development in sHTG and mixed dyslipidemia. CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholester Holemia, mixed dyslipidemias, or severe dyslipidemias (e elevated TG and LDL). Overall,CTX310 demonstrated a well-Tolerated safety and tolerability profiles that supports continued advancement of the programs. These new results build upon previous disclosed top-line data from12 participants across the first four potential cohort, corresponding to lean body weight -based doses of DL1 [ 0.1 mg/kg], DL2 [0.3 u/kg, DL3 [ 0.6mg/kg] and DL4[0.8 mg/kg]. Next steps: Results from the Phase 1 clinical trials highlight the potential of CTX 310 to safely and durable both TG and LDL following a one- course IV administration. CRISPRTherapeutics is advancing CTX 310 into Phase 1b clinical trial, prioritizing development in s HTG and mixed dyslipidemias. CTX320 are in current clinical trials in patients with heter heterozygous familial hyperchurchaseolemia, homozygous familial hyperchurchasemia, mixed dyslipidemia, or severe dyslipidemia, and in patients with elevated lipoprotein(a), respectively.공고 • Oct 10CRISPR Therapeutics Presents New Preclinical Data for CTX460™? Demonstrating in Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™? Editing PlatformCRISPR Therapeutics reported new preclinical data from its novel SyNTase™? gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data are featured in an oral presentation titled "Single-dose in vivo gene correction of AATD via LNP-delivered SyNTase editors" at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress. The AATD program, designated CTX460™?, is the first investigational candidate to use SyNTase editing and is expected to enter the clinic in mid-2026. "Alpha-1 antitrypsin deficiency remains an area of significant unmet need, with current treatments unable to address the underlying genetic cause. The current standard-of-care requires weekly intravenous (IV) infusion of purified functional M-AAT protein to mitigate lung disease symptoms, but no approved therapies address the underlying genetic cause. Better therapies are needed that can normalize AAT levels to minimize risk of lung disease progression. CTX460 is a SyNTase editing-based investigational candidate targeting the E342K mutation in SERPINA1, encapsulated in a de-risked, proprietary lipid nanoparticle (LNP). The preclinical data presented evaluated CTX460 in two preclinical models of AATD: the well-established NSG-PiZ mouse model, which carries human SERPINA1 Z alleles, and a novel humanized PiZ rat model in which the normal rat SERPINA1 gene is replaced with the human mutant SERPINA1 E342K variant. These models were used to evaluate gene and mRNA correction, serum protein levels, and durability of effect following a single dose of CTX460. CTX460 Key Preclinical Data Highlights: A single dose of CTX460 achieved significant, dose-dependent correction of liver DNA in both rat and mouse AATD models, with near saturating editing in hepatocytes at doses as low as 0.1 mg/kg. Furthermore, a single dose of CTX 460 was able to achieve >90% mRNA correction at a clinically relevant dose of 0.5 mg/kg in PiZ mice.공고 • Oct 01CRISPR Therapeutics to Present Preclinical Data on Alpha-1 Antitrypsin Deficiency Utilizing Novel Syntase Gene Editing Technology At the European Society of Gene and Cell Therapy 2025 Annual CongressCRISPR Therapeutics announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, taking place October 7-10, 2025. The presentation will introduce the Company's novel SyNTase gene editing technology and highlight its application in single-dose in vivo gene correction to treat Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder. CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform. SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together, these components enable gene editing with greater efficiency and precision, while also supporting scalable manufacturing. Using AI-guided structural modeling and large-scale screening, the polymerase was optimized to support gene correction activity based on synthetic nucleotide templates. When integrated with Cas9, SyNTase editors can utilize engineered templates with improved serum stability, enabling higher target correction efficiency. The abstract describes that SyNTase editing produces high levels of editing (up to 95%) in SERPINA1-E342K human hepatocyte cell models without any detectable (70% mRNA correction and >3-fold total serum AAT upregulation, exceeding the established clinically protective threshold. Together, these data provide proof-of-concept for a potentially best-in-class therapeutic modality to address the underlying cause of AATD and support SyNTase editing as a promising platform for the treatment of many monogenic disorders. Additional results beyond those included in the abstract will be presented at the conference. To accelerate and expand its efforts, CRISPR Therapeutics have formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR THERAPEUTICS®? standard character mark and design logo and SyNTase™? are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY®? and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners.공고 • Sep 23CRISPR Therapeutics and Sirius Therapeutics Announce First Patient Dosed in Phase 2 Trial of SRSD107 for Thromboembolic Disorders in EuropeCRISPR Therapeutics and Sirius Therapeutics announced that the first patient has been dosed in a Phase 2 clinical trial of SRSD107, a next-generation, long-acting Factor XI (FXI) siRNA for the prevention of venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA). SRSD107 is being co-developed by CRISPR Therapeutics andSir Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases. The ongoing Phase 2 clinical trial is a randomized, multicenter, global study evaluating the safety and efficacy of SRSD107 for the prevention of VTE in patients undergoing TKA. The trial will assess the anticoagulant effects and pharmacological profile of SRSD107 and help inform dose selection for future pivotal studies, with the goal of confirming its potential as a differentiated approach for reducing thrombotic risk in patients. SRSD107 is designed to selectively inhibit FXI, a key driver of pathological thrombosis, with minimal impact on normal hemostasis. In prior Phase 1 clinical trials conducted in Australia and China, single doses of SRSD107 were well tolerated and demonstrated strong, sustained pharmacodynamic effects, including reductions of over 93% in FXI levels, along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to six months post-dosing.공고 • Jun 26CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™? Targeting Angptl3 and Provides Update on in Vivo Cardiovascular PipelineCRISPR Therapeutics announced updates across its in vivo cardiovascular disease programs. These include new data for CTX310™?, targeting ANGPTL3, as well as continued progress on CTX320™?, targeting the LPA gene, and CTX340™?, targeting the AGT gene. CTX310 is initially focused on high-risk patients with the greatest unmet medical need and limited effective treatment options. CTX310 is in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholester Holemia (HeFH), or mixed dyslipidemias (MDL). Eligible participants have levels of TG >300 mg/dL and/or LDL-C >100 mg/dL (or >70 mg/dL for subjects with ASCVD). These new results build upon previously disclosed results from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg, DL2 [0.3 mg/kg, DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg) with at least 30 days of follow-up for each participant. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at DL4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings. The Company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025. CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a), a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events (MACE). The Phase 1 trial is enrolling patients and dose-finding is ongoing. An update is now expected in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) treatment landscape. CTX310 and CTX320 are in ongoing clinical trials in patients withheterozygous familial hypercholesterolmia, homozygous familial hyperchurchaseolemia, mixed dyslipidemias, or in patients with elevated lipop protein(a), respectively. Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: CRISPR Therapeutics remains focused on high-risk patients.이익 및 매출 성장 예측DB:1CG - 애널리스트 향후 추정치 및 과거 재무 데이터 (USD Millions)날짜매출이익자유현금흐름영업현금흐름평균 애널리스트 수12/31/2028665-111-165249912/31/2027179-353-260-2081412/31/202654-438-450124126/30/202613-451-374-370N/A3/31/20264-569-426-400N/A12/31/20254-582-371-345N/A9/30/202538-488-328-302N/A6/30/202538-468-351-325N/A3/31/202538-386-308-306N/A12/31/202437-366-145-143N/A9/30/2024203-240-191-189N/A6/30/2024202-266-127-122N/A3/31/2024272-217-169-159N/A12/31/2023371-154-272-260N/A9/30/2023170-354-295-278N/A6/30/2023170-416-363-343N/A3/31/2023100-524-377-352N/A12/31/20221-650-533-496N/A9/30/202214-681-509-468N/A6/30/202215-633-516-445N/A3/31/2022915312414504N/A12/31/2021915378457539N/A9/30/2021902412465544N/A6/30/2021902447502549N/A3/31/20211-392-309-287N/A12/31/20201-349-257-238N/A9/30/202077-211-167-154N/A6/30/2020289204555N/A3/31/202028946N/A48N/A12/31/201929067N/A57N/A9/30/2019213-11N/A25N/A6/30/20191-200N/A-128N/A3/31/20192-185N/A-117N/A12/31/20183-165N/A-96N/A9/30/201835-117N/A-83N/A6/30/201837-91N/A-79N/A3/31/201840-75N/A-69N/A12/31/201741-68N/A-70N/A9/30/201711-51N/A-75N/A6/30/201710-41N/A-71N/A3/31/20177-36N/A-69N/A12/31/20165-23N/A-53N/A9/30/20163-53N/A33N/A6/30/20162-44N/A40N/A3/31/20161-31N/A51N/A12/31/20150-26N/A59N/A더 보기애널리스트 향후 성장 전망수입 대 저축률: 1CG 은 향후 3년 동안 수익을 낼 것으로 예상되며, 이는 절약률(1.9%)보다 빠른 성장으로 간주됩니다.수익 vs 시장: 1CG (는) 향후 3년 동안 평균 시장 성장보다 높은 수익을 올릴 것으로 예상됩니다.고성장 수익: 1CG 향후 3년 내에 수익을 낼 것으로 예상됩니다.수익 대 시장: 1CG 의 수익(연간 70.1%)이 German 시장(연간 6.6%)보다 빠르게 성장할 것으로 예상됩니다.고성장 매출: 1CG 의 수익(연간 70.1%)은 연간 20%보다 빠르게 증가할 것으로 예상됩니다.주당순이익 성장 예측향후 자기자본이익률미래 ROE: 1CG는 3년 뒤에도 수익성이 없을 것으로 전망됩니다.성장 기업 찾아보기7D1Y7D1Y7D1YPharmaceuticals-biotech 산업의 고성장 기업.View Past Performance기업 분석 및 재무 데이터 상태데이터최종 업데이트 (UTC 시간)기업 분석2026/08/05 23:06종가2026/08/05 00:00수익2026/06/30연간 수익2025/12/31데이터 소스당사의 기업 분석에 사용되는 데이터는 S&P Global Market Intelligence LLC에서 제공됩니다. 아래 데이터는 이 보고서를 생성하기 위해 분석 모델에서 사용됩니다. 데이터는 정규화되므로 소스가 제공된 후 지연이 발생할 수 있습니다.패키지데이터기간미국 소스 예시 *기업 재무제표10년손익계산서현금흐름표대차대조표SEC 양식 10-KSEC 양식 10-Q분석가 컨센서스 추정치+3년재무 예측분석가 목표주가분석가 리서치 보고서Blue Matrix시장 가격30년주가배당, 분할 및 기타 조치ICE 시장 데이터SEC 양식 S-1지분 구조10년주요 주주내부자 거래SEC 양식 4SEC 양식 13D경영진10년리더십 팀이사회SEC 양식 10-KSEC 양식 DEF 14A주요 개발10년회사 공시SEC 양식 8-K* 미국 증권에 대한 예시이며, 비(非)미국 증권에는 해당 국가의 규제 서식 및 자료원을 사용합니다.별도로 명시되지 않는 한 모든 재무 데이터는 연간 기간을 기준으로 하지만 분기별로 업데이트됩니다. 이를 TTM(최근 12개월) 또는 LTM(지난 12개월) 데이터라고 합니다. 자세히 알아보기.분석 모델 및 스노우플레이크이 보고서를 생성하는 데 사용된 분석 모델의 세부 정보는 당사의 GitHub 페이지에서 확인하실 수 있습니다. 또한 보고서 사용 방법에 대한 가이드와 YouTube 튜토리얼도 제공하고 있습니다.Simply Wall St 분석 모델을 설계하고 구축한 세계적 수준의 팀에 대해 알아보세요.산업 및 섹터 지표산업 및 섹터 지표는 Simply Wall St가 6시간마다 계산하며, 프로세스에 대한 자세한 내용은 Github에서 확인할 수 있습니다.분석가 소스CRISPR Therapeutics AG는 40명의 분석가가 다루고 있습니다. 이 중 17명의 분석가가 우리 보고서에 입력 데이터로 사용되는 매출 또는 수익 추정치를 제출했습니다. 분석가의 제출 자료는 하루 종일 업데이트됩니다.분석가기관Jack AllenBairdWilliam PickeringBernsteinAlec StranahanBofA Global Research37명의 분석가 더 보기
Reported Earnings • 23hSecond quarter 2026 earnings released: US$0.94 loss per share (vs US$2.40 loss in 2Q 2025)Second quarter 2026 results: US$0.94 loss per share (improved from US$2.40 loss in 2Q 2025). Revenue: US$10.2m (up US$9.29m from 2Q 2025). Net loss: US$91.2m (loss narrowed 56% from 2Q 2025). Revenue is forecast to grow 70% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in Europe.
공고 • Apr 04CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026, at 08:00 W. Europe Standard Time. Location: at the offices of walder wyss ltd, seefeldstrasse 123, 8008 switzerland, zurich Switzerland
공고 • Dec 24CRISPR Therapeutics Provides Broad Update on Zugocaptagene Geleucel in Autoimmune Disease and Hematologic MalignanciesCRISPR Therapeutics provided updates on zugocaptagene geleucel (zugo-cel), formerly known as CTX112™?, its investigational allogeneic CAR T targeting CD19, in development for autoimmune disease and hematologic malignancies. Autoimmune Disease: Zugo-cel, targeting CD19, is in an ongoing Phase 1 basket trial in autoimmune rheumatologic diseases, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis. Patients in the study may have active SLE (with or without renal involvement), SSc, or idiopathic inflammatory myopathy (IIM) despite the use of standard therapies. Preliminary clinical data from the Phase 1 study has been encouraging, and zugo-cel has been well tolerated to date. No Grade 3 ICANS or CRS has been observed at the 100 million cell dose, which is the dose currently being studied in the autoimmune basket trials. The Phase 1/2 clinical trial in R/R B-cell malignancies is ongoing. The Company expects to provide additional updates in the second half of 2026. CRISPR Therapeutics has also established a new collaboration and clinical supply agreement with Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program's development in oncology.
공고 • Nov 09CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310CRISPR Therapeutics announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%) and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose. These data were presented during a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM) in a peer-reviewed article entitled "Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3." Phase 1 Clinical Trial Design The Phase 1, open label, dose-escalation trial evaluated single-course IV doses of CTX310 ranging from 0.1 to 0.8 mg/kg (lean body weight) targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous Family hypercholesterolemia (HeFH), or mixed dyslipidemias (elevated TG and LDL). Overall, CTX310 demonstrated a well-tolerated safety and tolerability profile that supports continued advancement of the program. These new results build upon previously disclosed top-line data from 12 participants across the first four potential cohorts, corresponding to lean body weight-based doses of DL1 [0.1 mg/kg, DL2 [0.3 mg/kg, DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg). Results from the Phase 1 clinical trial highlight the potential of CTX310 to safely and durably lower both TG and LDL following a single-course IV administration. CRISPR Therapeutics is advancing CTX310 into Phase 1b clinical trials, prioritizing development in sHTG and mixed dyslipidemia. CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholester Holemia, mixed dyslipidemias, or severe dyslipidemias (e elevated TG and LDL). Overall,CTX310 demonstrated a well-Tolerated safety and tolerability profiles that supports continued advancement of the programs. These new results build upon previous disclosed top-line data from12 participants across the first four potential cohort, corresponding to lean body weight -based doses of DL1 [ 0.1 mg/kg], DL2 [0.3 u/kg, DL3 [ 0.6mg/kg] and DL4[0.8 mg/kg]. Next steps: Results from the Phase 1 clinical trials highlight the potential of CTX 310 to safely and durable both TG and LDL following a one- course IV administration. CRISPRTherapeutics is advancing CTX 310 into Phase 1b clinical trial, prioritizing development in s HTG and mixed dyslipidemias. CTX320 are in current clinical trials in patients with heter heterozygous familial hyperchurchaseolemia, homozygous familial hyperchurchasemia, mixed dyslipidemia, or severe dyslipidemia, and in patients with elevated lipoprotein(a), respectively.
공고 • Oct 10CRISPR Therapeutics Presents New Preclinical Data for CTX460™? Demonstrating in Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™? Editing PlatformCRISPR Therapeutics reported new preclinical data from its novel SyNTase™? gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data are featured in an oral presentation titled "Single-dose in vivo gene correction of AATD via LNP-delivered SyNTase editors" at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress. The AATD program, designated CTX460™?, is the first investigational candidate to use SyNTase editing and is expected to enter the clinic in mid-2026. "Alpha-1 antitrypsin deficiency remains an area of significant unmet need, with current treatments unable to address the underlying genetic cause. The current standard-of-care requires weekly intravenous (IV) infusion of purified functional M-AAT protein to mitigate lung disease symptoms, but no approved therapies address the underlying genetic cause. Better therapies are needed that can normalize AAT levels to minimize risk of lung disease progression. CTX460 is a SyNTase editing-based investigational candidate targeting the E342K mutation in SERPINA1, encapsulated in a de-risked, proprietary lipid nanoparticle (LNP). The preclinical data presented evaluated CTX460 in two preclinical models of AATD: the well-established NSG-PiZ mouse model, which carries human SERPINA1 Z alleles, and a novel humanized PiZ rat model in which the normal rat SERPINA1 gene is replaced with the human mutant SERPINA1 E342K variant. These models were used to evaluate gene and mRNA correction, serum protein levels, and durability of effect following a single dose of CTX460. CTX460 Key Preclinical Data Highlights: A single dose of CTX460 achieved significant, dose-dependent correction of liver DNA in both rat and mouse AATD models, with near saturating editing in hepatocytes at doses as low as 0.1 mg/kg. Furthermore, a single dose of CTX 460 was able to achieve >90% mRNA correction at a clinically relevant dose of 0.5 mg/kg in PiZ mice.
공고 • Oct 01CRISPR Therapeutics to Present Preclinical Data on Alpha-1 Antitrypsin Deficiency Utilizing Novel Syntase Gene Editing Technology At the European Society of Gene and Cell Therapy 2025 Annual CongressCRISPR Therapeutics announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, taking place October 7-10, 2025. The presentation will introduce the Company's novel SyNTase gene editing technology and highlight its application in single-dose in vivo gene correction to treat Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder. CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform. SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together, these components enable gene editing with greater efficiency and precision, while also supporting scalable manufacturing. Using AI-guided structural modeling and large-scale screening, the polymerase was optimized to support gene correction activity based on synthetic nucleotide templates. When integrated with Cas9, SyNTase editors can utilize engineered templates with improved serum stability, enabling higher target correction efficiency. The abstract describes that SyNTase editing produces high levels of editing (up to 95%) in SERPINA1-E342K human hepatocyte cell models without any detectable (70% mRNA correction and >3-fold total serum AAT upregulation, exceeding the established clinically protective threshold. Together, these data provide proof-of-concept for a potentially best-in-class therapeutic modality to address the underlying cause of AATD and support SyNTase editing as a promising platform for the treatment of many monogenic disorders. Additional results beyond those included in the abstract will be presented at the conference. To accelerate and expand its efforts, CRISPR Therapeutics have formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR THERAPEUTICS®? standard character mark and design logo and SyNTase™? are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY®? and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners.
공고 • Sep 23CRISPR Therapeutics and Sirius Therapeutics Announce First Patient Dosed in Phase 2 Trial of SRSD107 for Thromboembolic Disorders in EuropeCRISPR Therapeutics and Sirius Therapeutics announced that the first patient has been dosed in a Phase 2 clinical trial of SRSD107, a next-generation, long-acting Factor XI (FXI) siRNA for the prevention of venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA). SRSD107 is being co-developed by CRISPR Therapeutics andSir Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases. The ongoing Phase 2 clinical trial is a randomized, multicenter, global study evaluating the safety and efficacy of SRSD107 for the prevention of VTE in patients undergoing TKA. The trial will assess the anticoagulant effects and pharmacological profile of SRSD107 and help inform dose selection for future pivotal studies, with the goal of confirming its potential as a differentiated approach for reducing thrombotic risk in patients. SRSD107 is designed to selectively inhibit FXI, a key driver of pathological thrombosis, with minimal impact on normal hemostasis. In prior Phase 1 clinical trials conducted in Australia and China, single doses of SRSD107 were well tolerated and demonstrated strong, sustained pharmacodynamic effects, including reductions of over 93% in FXI levels, along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to six months post-dosing.
공고 • Jun 26CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™? Targeting Angptl3 and Provides Update on in Vivo Cardiovascular PipelineCRISPR Therapeutics announced updates across its in vivo cardiovascular disease programs. These include new data for CTX310™?, targeting ANGPTL3, as well as continued progress on CTX320™?, targeting the LPA gene, and CTX340™?, targeting the AGT gene. CTX310 is initially focused on high-risk patients with the greatest unmet medical need and limited effective treatment options. CTX310 is in an ongoing Phase 1 first-in-human clinical trial targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholester Holemia (HeFH), or mixed dyslipidemias (MDL). Eligible participants have levels of TG >300 mg/dL and/or LDL-C >100 mg/dL (or >70 mg/dL for subjects with ASCVD). These new results build upon previously disclosed results from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg, DL2 [0.3 mg/kg, DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg) with at least 30 days of follow-up for each participant. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at DL4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings. The Company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025. CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a), a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events (MACE). The Phase 1 trial is enrolling patients and dose-finding is ongoing. An update is now expected in the first half of 2026, reflecting a strategic decision to incorporate emerging insights from the evolving Lp(a) treatment landscape. CTX310 and CTX320 are in ongoing clinical trials in patients withheterozygous familial hypercholesterolmia, homozygous familial hyperchurchaseolemia, mixed dyslipidemias, or in patients with elevated lipop protein(a), respectively. Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: CRISPR Therapeutics remains focused on high-risk patients.