Announcement • Dec 10

Crispr Therapeutics Presents Data from the Their Ongoing Phase 1/2 Dose Escalation Clinical Trial Evaluating the Safety and Efficacy of Ctx112

CRISPR Therapeutics presented data from the Company’s ongoing Phase 1/2 dose escalation clinical trial evaluating the safety and efficacy of CTX112, a next-generation CD19 allogeneic CAR T cell therapy, in relapsed or refractory (R/R) CD19-positive B-cell malignancies at the 2024 American Society of Hematology Annual Meeting. Additionally, the Company announced that the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to CTX112 for the treatment of R/R follicular lymphoma and marginal zone lymphoma. CTX112 Trial Overview The Phase 1/2 clinical trial is an open-label, multicenter study evaluating the safety and efficacy of CTX112 in relapsed or refractory (R/R) B-cell malignancies. Eligible disease subtypes include large B-cell lymphoma, follicular lymphoma grade 1-3a, marginal zone lymphoma, and mantle cell lymphoma. CTX112 was infused after a standard course of lymphodepleting chemotherapy (3 days of 30 mg/m2 fludarabine and 500 mg/m2 cyclophosphamide). Data were presented from 12 subjects treated during the dose escalation with CTX112 doses ranging from 30 x 106 (Dose Level [DL] 1) to 600 x 106 (DL4) CAR+ T cells. The study population was enriched for patients with high-risk characteristics, including: 1) primary refractory disease or early relapse to first-line therapy (75%); 2) high tumor burden (SPD > 4000 mm2, 50%); and 3) high disease prognostic index score (IPI, FLIPI, MZL-IPI =3) or elevated lactate dehydrogenase (75%). Safety, CTX112 was well tolerated across all dose levels. There were no reported dose limiting toxicities and no reported Grade =3 infections. All grade 3 or 4 cytopenias (i.e., neutropenia, thrombocytopenia, anemia) following lymphodepleting chemotherapy resolved to Grade 2 or better within 1 month of CTX112 infusion. There were no reported cases of Graft versus Host Disease, All cases of cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome were Grade 1 or 2 per the American Society for Transplantation and Cellular Therapy criteria. These low-grade CRS and ICANS events followed standard toxicity management protocols. Clinical Efficacy, CTX112 produced responses at all dose levels. Disease assessment was performed by investigator review according to the Lugano criteria. Objective and complete responses were seen at all dose levels and in all treated NHL subtypes (i.e., FL, MZL, MCL and LBCL). Responses were also seen in patients with poor prognostic factors including primary refractory disease, early relapse, and high baseline tumor burden (e.g., SPD > 4000 mm2). Five patients (of the 12 treated) have achieved responses lasting for more than 6 months, including one patient whose 6-month response was confirmed after the data cut-off date. One patient treated at DL1 remains in complete remission over a year after initial CTX112 infusion. The clinical efficacy of CTX112 is supported by a clearly differentiated pharmacokinetic profile for an allogeneic CAR T cell therapy. The mean peak concentration and total exposure were significantly higher at DL3 and DL4 vs. DL1 and DL2. This dose dependence suggests the possibility of deeper and more durable responses as the trial moves from dose escalation to dose optimization. Comparing DL3, the addition of Regnase-1 and TGFßR2 edits results in 7-fold higher peak concentration and 9.7-fold higher mean area under the curve for CTX112 relative to CTX110. Furthermore, at DL4, both Cmax and AUC are showing significantly more consistent and predictable increases. This suggests that the novel CRISPR/Cas9 potency edits are leading to higher CAR T cell expansion and functional persistence without enhanced or increased lymphodepleting chemotherapy doses. These preliminary data demonstrate that CTX112 has the potential to provide meaningful clinical benefit with a well-tolerated safety profile. Given the inherent difficulties of manufacturing a CAR T therapy from a patient’s own diseased cells, allogeneic cellular therapy approaches have greater potential to address the unmet need in this patient population. These promising findings underscore the potential of allogeneic cell therapies to offer a transformative option for patients, and they remain committed to advancing this innovative approach to address the significant unmet medical need in this area. Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising regenerative medicine pipeline products. A regenerative medicine therapy is eligible for RMAT designation if it is a cell therapy, therapeutic tissue engineering product, human cell and tissue product or any combination product of such therapies that is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application and other opportunities to expedite development and review.

Announcement • Jun 10

Vertex Pharmaceuticals Incorporated and Crispr Therapeutics Announces Positive Results from Pivotal Trials of Exa-Cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that both pivotal trials for exagamglogene autotemcel (exa-cel) in patients with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD) met primary and key secondary endpoints at pre-specified interim analyses. The results are being presented at the Annual European Hematology Association (EHA) Congress. New Data From Pre-Specified Interim Analyses in exa-cel Pivotal Trials: Both CLIMB-111 and CLIMB-121 met their primary endpoint and key secondary endpoint at the pre-specified interim analysis for each trial. These analyses evaluated the efficacy and safety of exa-cel in patients with TDT or SCD in the ongoing Phase 3 trials as well as in the long-term follow up trial CLIMB-131. The data shared are from 83 patients (48 with TDT and 35 with SCD) dosed with exa-cel with follow-up up to 43.7 months. All patients treated with exa-cel demonstrated clinical benefit, and these data continue to demonstrate the potentially transformative profile of exa-cel. Of the 48 patients with TDT who had received exa-cel at the time of the analysis, more than half (58.3%) have genotypes associated with severe disease, beta-zero/beta-zero or other beta-zero-like severe genotypes. At the time of the data cut, 27 TDT patients were evaluable for the primary and key secondary endpoint. 24/27 (88.9%) achieved the primary endpoint of transfusion- independence for at least 12 consecutive months (TI12) and the secondary endpoint of transfusion-independence for at least 6 consecutive months (TI6) with a mean weighted hemoglobin of at least 9 g/dL (95% CI: 70.8%, 97.6%; Increases in total hemoglobin occurred early within the first few months and were maintained over time. In the analysis of all patients who received exa-cel, mean total hemoglobin was =11g/dL at Month 3 and =12g/dL from month 6 onward with pancellular distribution of fetal hemoglobin. Mean proportion of edited BCL11A alleles was stable over time in bone marrow and peripheral blood indicating successful permanent editing in the long-term hematopoietic stem cells. Patients also had clinically significant improvements in patient-reported outcomes. Efficacy of exa-cel in Patients With Severe Sickle Cell Disease; Of the 35 patients with SCD who had received exa-cel at the time of the analysis, 17 patients were evaluable for the primary and key secondary endpoint at the time of the data cut. 16/17 (94.1%) achieved the primary endpoint of freedom from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12) (95% CI: 71.3%, 99.9%; P=0.0001). Mean duration of VOC-free was 18.7 months, with a maximum of 36.5 months. 17/17 (100%) achieved the key secondary endpoint of being free from hospitalizations related to VOCs for at least 12 consecutive months (HF12) (95% CI: 80.5%, 100.0%; P<0.0001). The one patient who did not achieve VF12 did achieve HF12 and has a complex set of comorbidities, including a history of chronic pain. One patient who achieved VF12 had a VOC 22.8 months following exa-cel infusion in the setting of a parvovirus infection. This patient has since fully recovered from the infection and been VOC-free. Increases in fetal hemoglobin and total hemoglobin occurred early, within the first few months, and were maintained over time. In the analysis of all patients who received exa-cel, mean fetal hemoglobin was more than 30% of total hemoglobin by Month 3 and was then maintained at approximately 40.0% through follow-up, with pancellular distribution. Mean proportion of edited BCL11A alleles was stable over time in bone marrow and peripheral blood, indicating successful permanent editing in the long-term hematopoietic stem cells. Patients also had clinically significant improvements in patient-reported outcomes. Safety of exa-cel in All PatientsThe safety profile of exa-cel was generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. All patients engrafted neutrophils and platelets after exa-cel infusion. As previously reported, two TDT patients had serious adverse events (SAEs) considered related to exa-cel. One patient had three SAEs considered related to exa-cel: hemophagocytic lymphohistiocytosis (HLH), acute respiratory distress syndrome and headache, and one SAE of idiopathic pneumonia syndrome that was considered related to both exa-cel and busulfan. All four SAEs occurred in the context of HLH in the peri-engraftment period and have resolved. One patient had SAEs of delayed neutrophil engraftment and thrombocytopenia, both of which were considered related to exa-cel and busulfan, and both SAEs have resolved. Among the 35 patients with SCD, there were no SAEs considered related to exa-cel. Also as previously reported, one adult patient with SCD developed pneumonia and respiratory failure following SARS-CoV-2 infection, resulting in death. The investigator assessed the events as not related to exa-cel. There were no other deaths or discontinuations, and there have been no malignancies in either study.

Biotechs earnings growth	25.2%
Revenue growth rate	64.1%
Future return on equity	-46.13%
Analyst coverage	Good
Last updated	13 May 2026

Date	Revenue	Earnings	Free Cash Flow	Cash from Op	Avg. No. Analysts
12/31/2028	614	-197	-100	49	12
12/31/2027	150	-389	-273	-213	20
12/31/2026	45	-479	-421	-224	21
3/31/2026	4	-569	-426	-400	N/A
12/31/2025	4	-582	-371	-345	N/A
9/30/2025	38	-488	-328	-302	N/A
6/30/2025	38	-468	-351	-325	N/A
3/31/2025	38	-386	-308	-306	N/A
12/31/2024	37	-366	-145	-143	N/A
9/30/2024	203	-240	-191	-189	N/A
6/30/2024	202	-266	-127	-122	N/A
3/31/2024	272	-217	-169	-159	N/A
12/31/2023	371	-154	-272	-260	N/A
9/30/2023	170	-354	-295	-278	N/A
6/30/2023	170	-416	-363	-343	N/A
3/31/2023	100	-524	-377	-352	N/A
12/31/2022	1	-650	-533	-496	N/A
9/30/2022	14	-681	-509	-468	N/A
6/30/2022	15	-633	-516	-445	N/A
3/31/2022	915	312	414	504	N/A
12/31/2021	915	378	457	539	N/A
9/30/2021	902	412	465	544	N/A
6/30/2021	902	447	502	549	N/A
3/31/2021	1	-392	-309	-287	N/A
12/31/2020	1	-349	-257	-238	N/A
9/30/2020	77	-211	-167	-154	N/A
6/30/2020	289	20	45	55	N/A
3/31/2020	289	46	40	48	N/A
12/31/2019	290	67	N/A	57	N/A
9/30/2019	213	-11	N/A	25	N/A
6/30/2019	1	-200	N/A	-128	N/A
3/31/2019	2	-185	N/A	-117	N/A
12/31/2018	3	-165	N/A	-96	N/A
9/30/2018	35	-117	N/A	-83	N/A
6/30/2018	37	-91	N/A	-79	N/A
3/31/2018	40	-75	N/A	-69	N/A
12/31/2017	41	-68	N/A	-70	N/A
9/30/2017	11	-51	N/A	-75	N/A
6/30/2017	10	-41	N/A	-71	N/A
3/31/2017	7	-36	N/A	-69	N/A
12/31/2016	5	-23	N/A	-53	N/A
9/30/2016	3	-53	N/A	33	N/A
6/30/2016	2	-44	N/A	40	N/A
3/31/2016	1	-31	N/A	51	N/A
12/31/2015	0	-26	N/A	59	N/A

Data	Last Updated (UTC time)
Company Analysis	2026/05/18 21:27
End of Day Share Price	2026/05/15 00:00
Earnings	2026/03/31
Annual Earnings	2025/12/31

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
Jack Allen	Baird
William Pickering	Bernstein
Alec Stranahan	BofA Global Research

CRISPR Therapeutics Future Growth

Future criteria checks 2/6

Key information

Recent future growth updates

Here's What Analysts Are Forecasting For CRISPR Therapeutics AG (NASDAQ:CRSP) After Its Third-Quarter Results

Consensus revenue estimates decrease by 33%

Consensus revenue estimates fall by 34%

Consensus revenue estimates increase by 11%

Consensus estimates of losses per share improve by 30%

Consensus revenue estimates increase by 13%

Recent updates

CRISPR Therapeutics Expands Casgevy Reach and Strengthens Pipeline With New Funding

Crispr Therapeutics Q1 Earnings: Casgevy Growth, H2 In-Vivo Catalysts Make Buy Case

CRISPR Therapeutics AG, Annual General Meeting, Jun 04, 2026

CRISPR Therapeutics Provides Broad Update on Zugocaptagene Geleucel in Autoimmune Disease and Hematologic Malignancies

CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310

CRISPR Therapeutics Presents New Preclinical Data for CTX460™? Demonstrating in Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™? Editing Platform

CRISPR 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 Congress

CRISPR Therapeutics and Sirius Therapeutics Announce First Patient Dosed in Phase 2 Trial of SRSD107 for Thromboembolic Disorders in Europe

CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™? Targeting Angptl3 and Provides Update on in Vivo Cardiovascular Pipeline

CRISPR Therapeutics AG, Annual General Meeting, Jun 05, 2025

CRISPR Therapeutics (NASDAQ:CRSP) Is In A Good Position To Deliver On Growth Plans

Julianne Bruno to Step Down from CRISPR Therapeutics AG as Chief Operating Officer, Effective as of April 11, 2025

CRISPR Therapeutics: An Uninspiring Start For Casgevy

Crispr Therapeutics Q4 Earnings Review: Ignore The Lack Of Revenues, Buy The Future

CRISPR Therapeutics: Unfairly Punished By The Issues Of Its Peers

CRISPR Therapeutics AG's (NASDAQ:CRSP) Share Price Not Quite Adding Up

Crispr Therapeutics: From Casgevy Success To A Catalyst-Rich 2025

Crispr Therapeutics Presents Data from the Their Ongoing Phase 1/2 Dose Escalation Clinical Trial Evaluating the Safety and Efficacy of Ctx112

Here's Why We're Not Too Worried About CRISPR Therapeutics' (NASDAQ:CRSP) Cash Burn Situation

Crispr Therapeutics Q3 Earnings: No Casgevy Revenue, But Plenty Of Optimism

Here's What Analysts Are Forecasting For CRISPR Therapeutics AG (NASDAQ:CRSP) After Its Third-Quarter Results

Crispr Therapeutics: Poised For A Major Turnaround

CRISPR Therapeutics AG (NASDAQ:CRSP) Investors Are Less Pessimistic Than Expected

Crispr Therapeutics: Casgevy Now Active Across 35 Treatment Centres

Here's Why We're Not At All Concerned With CRISPR Therapeutics' (NASDAQ:CRSP) Cash Burn Situation

Crispr's Casgevy Launch: Promising Start, Uncertain Future

A Look At The Intrinsic Value Of CRISPR Therapeutics AG (NASDAQ:CRSP)

Crispr Therapeutics Q2 Earnings: Casgevy Updates Underline 'Blockbuster' Potential

Crispr Therapeutics Remains Expensive, With Slower Casgevy Monetization And Patent Battle

Crispr Therapeutics: Beaten Down But Not Broken

CRISPR Therapeutics AG's (NASDAQ:CRSP) Share Price Could Signal Some Risk

Crispr Therapeutics, Futurism-Turning-Commercial, Defies 'Valuation' (Technical Analysis)

Crispr Therapeutics' Strategic Moves Amidst Gene Therapy Challenges (Rating Upgrade)

CRISPR Therapeutics Announces Executive Changes

Shareholders Will Probably Hold Off On Increasing CRISPR Therapeutics AG's (NASDAQ:CRSP) CEO Compensation For The Time Being

Consensus revenue estimates decrease by 33%

Newsflash: CRISPR Therapeutics AG (NASDAQ:CRSP) Analysts Have Been Trimming Their Revenue Forecasts

We're Not Worried About CRISPR Therapeutics' (NASDAQ:CRSP) Cash Burn

First quarter 2024 earnings released: US$1.43 loss per share (vs US$0.67 loss in 1Q 2023)

Crispr Therapeutics Q1 Earnings: Casgevy Launch Tip Of Gene Editing Iceberg

CRISPR Therapeutics Highlights ASGCT Oral Presentation and Announces New Programs Utilizing in Vivo Gene Editing Approach

Crispr Therapeutics: Market Misunderstanding Is Your Buying Opportunity (Upgrade)

Now 23% undervalued after recent price drop

Crispr Therapeutics: Now Is The Time To Buy

Now 21% undervalued after recent price drop

CEO & Chairman notifies of intention to sell stock

Calculating The Intrinsic Value Of CRISPR Therapeutics AG (NASDAQ:CRSP)

CRISPR Therapeutics AG, Annual General Meeting, May 30, 2024

Crispr Therapeutics: CASGEVY Commercial Launch Is Taking Shape

CEO & Chairman exercised options and sold US$1.1m worth of stock

Consensus revenue estimates fall by 34%

Full year 2023 earnings: EPS and revenues exceed analyst expectations

There's Reason For Concern Over CRISPR Therapeutics AG's (NASDAQ:CRSP) Massive 26% Price Jump

Crispr Therapeutics: Cautiously Optimistic

CEO & Chairman notifies of intention to sell stock

CRISPR Therapeutics AG has filed a Follow-on Equity Offering in the amount of $279.999863 million.

New minor risk - Shareholder dilution

Crispr Therapeutics Announces European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™

Crispr Therapeutics: Investors Bet Against Casgevy Uptake

CEO & Chairman notifies of intention to sell stock

CEO & Chairman notifies of intention to sell stock

Crispr Therapeutics: Commercial Validation Is The Next Step

CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ for the Treatment of Transfusion-Dependent Beta Thalassemia

Impact Of Gene Therapies And Casgevy On Crispr AG And The Industry

CRISPR Therapeutics AG's (NASDAQ:CRSP) Popularity With Investors Is Under Threat From Overpricing

CRISPR Therapeutics AG Announces Resignation of Phuong Khanh Morrow as Chief Medical Officer, Effective January 26, 2024

Consensus revenue estimates increase by 11%

CRISPR Therapeutics AG and Vertex Pharmaceuticals Incorporated Announce Receipt of the First-Ever Approval of a CRISPR-Based Gene-editing Therapy in the United States

CRISPR Therapeutics Announces Updates to Immuno-Oncology Pipeline and Expansion into Autoimmune Disease