CRISPR Therapeutics（1CG）株式概要CRISPR Therapeutics AGは遺伝子編集会社で、CRISPR（Clustered Regularly Interspaced Short Palindromic Repeats）／CRISPR関連タンパク質9（Cas9）プラットフォームを用い、ヒトの重篤な疾患に対する遺伝子ベースの医薬品開発に注力している。 詳細1CG ファンダメンタル分析スノーフレーク・スコア評価4/6将来の成長5/6過去の実績0/6財務の健全性4/6配当金0/6報酬当社が推定した公正価値より77.7%で取引されている 収益は年間69.69%増加すると予測されています リスク分析German市場と比較した過去 3 か月間の株価の変動すべてのリスクチェックを見る1CG Community Fair Values Create NarrativeSee what others think this stock is worth. Follow their fair value or set your own to get alerts.NEW487,236 membersJoin community and earn perksGain real feedbackFrom our editorial team, personally. Not silence.Grow your followingReal investors. The kind who actually invest, not scroll past.Unlock free accessFree premium subscription for consistent and quality authors.Learn moreCreate NarrativeBLINRODA487,236 investors already sharing narrativesYour Fair Value€Current Price€43.211.1k% 割高 内在価値ディスカウントGrowth estimate overAnnual revenue growth rate5 Yearstime period%/yrDecreaseIncreasePastFuture-1b915m2016201920222025202620282031Revenue US$48.7mEarnings US$8.5mAdvancedSet Fair ValueView all narrativesCRISPR Therapeutics AG 競合他社Hamlet BioPharmaSymbol: DB:7ZTMarket cap: €1.3bBiotest GmbH KGaASymbol: HMSE:BIO0Market cap: €1.3bBiotestSymbol: XTRA:BIO3Market cap: €1.4bDextech MedicalSymbol: DB:LQ0Market cap: €193.2m価格と性能株価の高値、安値、推移の概要CRISPR Therapeutics過去の株価現在の株価US$45.6252週高値US$68.5052週安値US$37.60ベータ1.741ヶ月の変化-12.82%3ヶ月変化2.26%1年変化-6.91%3年間の変化n/a5年間の変化n/aIPOからの変化197.17%最新ニュースReported Earnings • 15hSecond 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.お知らせ • 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お知らせ • 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.お知らせ • 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.お知らせ • 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.お知らせ • 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.最新情報をもっと見るRecent updatesReported Earnings • 15hSecond 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.お知らせ • 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お知らせ • 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.お知らせ • 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.お知らせ • 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.お知らせ • 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.お知らせ • 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.お知らせ • 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.株主還元1CGDE BiotechsDE 市場7D9.9%-0.02%2.2%1Y-6.9%7.1%4.6%株主還元を見る業界別リターン: 1CG過去 1 年間で7.1 % の収益を上げたGerman Biotechs業界を下回りました。リターン対市場: 1CGは、過去 1 年間で4.6 % のリターンを上げたGerman市場を下回りました。価格変動Is 1CG's price volatile compared to industry and market?1CG volatility1CG Average Weekly Movement8.6%Biotechs Industry Average Movement7.0%Market Average Movement5.4%10% most volatile stocks in DE Market12.9%10% least volatile stocks in DE Market2.8%安定した株価: 1CGの株価は、 German市場と比較して過去 3 か月間で変動しています。時間の経過による変動: 1CGの weekly volatility ( 9% ) は過去 1 年間安定していますが、依然としてGermanの株式の 75% よりも高くなっています。会社概要設立従業員CEO（最高経営責任者ウェブサイト2013393Sam Kulkarniwww.crisprtx.com遺伝子編集会社であるCRISPR Therapeutics AGは、CRISPR/Cas9プラットフォームを用いた、ヒトの重篤な疾患に対する遺伝子ベースの医薬品開発に注力している。同社のCRISPR/Cas9は、ゲノムDNAの特定の配列を正確に改変する遺伝子編集のための画期的な技術である。同社は、ヘモグロビン異常症、CAR T細胞療法、in vivo、1型糖尿病など、さまざまな疾患領域にわたる治療プログラムのポートフォリオを有しており、固形がん治療の可能性を秘めたグリピカン-3（GPC3）を標的とする自己遺伝子編集CAR Tプログラムを含む、治験中のCAR Tプログラムも開発している。同社の主要製品候補はCASGEVYで、輸血依存性βサラセミア、重症鎌状赤血球症（SCD）、ヘモグロビン異常症を患う患者を治療するための生体外CRISPR/Cas9遺伝子編集細胞療法である。また、CAR T細胞療法も開発しており、腫瘍学および自己免疫疾患を適応症とする分化クラスタ19（CD19）を標的とするCTX112およびCD70を標的とするCTX131、有効な標的であるアンジオポエチン様タンパク質3およびリポタンパク質を破壊することにより心血管疾患に対処するin vivo遺伝子編集であるCTX310およびCTX320、T1D治療用の同種遺伝子編集低免疫性幹細胞由来の製品候補であるCTX211などがある。Vertex Pharmaceuticals Incorporated、Nkarta, Inc.およびCapsida Biotherapeuticsと戦略的パートナーシップを結んでいる。CRISPR Therapeutics AGは2013年に設立され、スイスのツークに本社を置いている。もっと見るCRISPR Therapeutics AG 基礎のまとめCRISPR Therapeutics の収益と売上を時価総額と比較するとどうか。1CG 基礎統計学時価総額€4.23b収益（TTM）-€391.27m売上高(TTM)€11.61m390.3xP/Sレシオ-11.6xPER（株価収益率1CG は割高か？公正価値と評価分析を参照収益と収入最新の決算報告書（TTM）に基づく主な収益性統計1CG 損益計算書（TTM）収益US$13.39m売上原価US$475.19m売上総利益-US$461.80mその他の費用-US$10.66m収益-US$451.14m直近の収益報告Jun 30, 2026次回決算日該当なし一株当たり利益（EPS）-4.58グロス・マージン-3,448.33%純利益率-3,368.72%有利子負債／自己資本比率43.4%1CG の長期的なパフォーマンスは？過去の実績と比較を見るView Valuation企業分析と財務データの現状データ最終更新日（UTC時間）企業分析2026/08/04 14:27終値2026/08/04 00:00収益2026/06/30年間収益2025/12/31データソース企業分析に使用したデータはS&P Global Market Intelligence LLC のものです。本レポートを作成するための分析モデルでは、以下のデータを使用しています。データは正規化されているため、ソースが利用可能になるまでに時間がかかる場合があります。パッケージデータタイムフレーム米国ソース例会社財務10年損益計算書キャッシュ・フロー計算書貸借対照表SECフォーム10-KSECフォーム10-Qアナリストのコンセンサス予想+プラス3年予想財務アナリストの目標株価アナリストリサーチレポートBlue Matrix市場価格30年株価配当、分割、措置ICEマーケットデータSECフォームS-1所有権10年トップ株主インサイダー取引SECフォーム4SECフォーム13Dマネジメント10年リーダーシップ・チーム取締役会SECフォーム10-KSECフォームDEF 14A主な進展10年会社からのお知らせSECフォーム8-K* 米国証券を対象とした例であり、非米国証券については、同等の規制書式および情報源を使用。特に断りのない限り、すべての財務データは1年ごとの期間に基づいていますが、四半期ごとに更新されます。これは、TTM（Trailing Twelve Month）またはLTM（Last Twelve Month）データとして知られています。詳細はこちら。分析モデルとスノーフレークこのレポートを生成するために使用した分析モデルの詳細は、当社のGitHubページでご覧いただけます。また、レポートの活用方法に関するガイドやYouTubeのチュートリアルも用意しています。シンプリー・ウォールストリート分析モデルを設計・構築した世界トップクラスのチームについてご紹介します。業界およびセクターの指標私たちの業界とセクションの指標は、Simply Wall Stによって6時間ごとに計算されます。アナリスト筋CRISPR Therapeutics AG 17 これらのアナリストのうち、弊社レポートのインプットとして使用した売上高または利益の予想を提出したのは、 。アナリストの投稿は一日中更新されます。40 アナリスト機関Jack AllenBairdWilliam PickeringBernsteinAlec StranahanBofA Global Research37 その他のアナリストを表示
Reported Earnings • 15hSecond 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.
お知らせ • 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
お知らせ • 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.
お知らせ • 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.
お知らせ • 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.
お知らせ • 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.
Reported Earnings • 15hSecond 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.
お知らせ • 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
お知らせ • 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.
お知らせ • 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.
お知らせ • 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.
お知らせ • 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.
お知らせ • 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.
お知らせ • 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.
