Triple-Hit Lymphoma

The Orphan Drug Designation strengthens LP-284’s clinical development path and can provide for additional market exclusivity and commercial protection. Lantern has initiated a first-in-human Phase 1 trial (NCT06132503) for LP-284 in B-cell non-Hodgkin’s lymphomas (NHL), including high-grade B-cell lymphoma (HGBL) and mantle cell lymphoma (MCL). This Orphan Drug Designation marks the second such designation for drug-candidate LP-284 this year. DALLAS--(BUSINESS WIRE)-- Lantern Pharma, Inc. (NASDAQ: LTRN), an artificial intelligence (“AI”) company developing targeted and transformative cancer therapies using its proprietary RADR® AI and machine learning (“ML”) platform with multiple clinical-stage drug programs, today announced that the U.S. Food and Drug Administration (FDA) has granted LP-284 Orphan Drug Designation (ODD) for the treatment of high-grade B-cell lymphoma with MYC and BCL2 rearrangements. HGBL represents a rare and aggressive form of B-cell non-Hodgkin's lymphoma (NHL) with no established standard of care treatment approach. Typically, frontline intervention involves a combination of chemo-immunotherapies such as R-CHOP or DA-R-EPOCH. However, approximately 20-30% of HGBL patients stop responding to these therapeutic agents and continue cancer progression. For those with relapsed or refractory (R/R) disease, the survival prognosis is 8.6 to 16 months (Laude et al., 2021). This underscores the pressing clinical need for novel and effective therapies in treating HGBL and improving patient outcomes. LP-284 is a novel small molecule agent that damages DNA in cancer cells leading to the death of cancer cells. Lantern is developing LP-284 for several aggressive B-cell NHL, including MCL and HGBL, where LP-284 has shown potent anti-tumor activity in preclinical models. Lantern has been able to advance LP-284 from initial RADR® A.I. insights regarding anti-cancer activity and potential mechanisms of action in hematological cancers, to selection of specific subtypes of lymphomas with superior response, to late-stage IND enabling studies and initiation of first-in-human clinical trials in a short span of approximately 2.5 years. "Receiving Orphan Drug Designation is an important milestone for our latest drug candidate, LP-284, and further validates our data-driven approach to oncology drug discovery and development,” stated Panna Sharma, President & CEO of Lantern Pharma. "At SOHO 2023, we reported positive preclinical data demonstrating LP-284’s potent anti-tumor activity as a monotherapy as well as in combination with FDA-approved lymphoma targeting antibody Rituximab in High-Grade B-cell Lymphoma (HGBL). These findings hold significant importance given the elevated rate of relapse and the unfavorable prognosis observed in the majority of HGBL patients,” continued Sharma. “This marks the second Orphan Drug Designation (ODD) granted by the FDA for LP-284. The initial ODD for LP-284 was granted in January 2023, and with this most recent ODD for LP-284 announced today, a total of five orphan designations have now been granted to Lantern, with the other three granted for our drug candidate LP-184. Acquiring these orphan designations is a key element of our business model as they provide a number of benefits including seven years of market exclusivity and eligibility for expedited drug development programs. Looking forward, these designations further position Lantern to advance our discussions with biopharma companies for partnering and collaborative development opportunities.” The FDA's Office of Orphan Products Development grants orphan status to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD is designed to provide drug developers with various benefits to support the development of novel drugs, including market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of marketing registration application fees, reduced annual product fees, clinical protocol assistance and qualification for expedited development programs. Reference: Laude MC, Lebras L, Sesques P, et al. First-line treatment of double-hit and triple-hit lymphomas: Survival and tolerance data from a retrospective multicenter French study. Am J Hematol. 2021;96(3):302-311. doi:10.1002/ajh.26068 About Lantern Pharma: Lantern Pharma (NASDAQ: LTRN) is a clinical-stage oncology-focused biopharmaceutical company leveraging its proprietary RADR® A.I. and machine learning platform to discover biomarker signatures that identify patients most likely to respond to its pipeline of genomically-targeted therapeutics. Lantern’s lead development programs include a Phase 2 clinical program, multiple Phase 1 clinical trials and an ADC program, across 11 disclosed tumor targets. By targeting drugs to patients whose genomic pro them as having the highest probability of benefiting from the drug, Lantern's approach represents the potential to deliver best-in-class outcomes. Please find more information at: Website: LinkedIn: X: @lanternpharma Monthly Newsletter: Sign-up here Forward-looking Statements: This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements include, among other things, statements relating to: future events or our future financial performance; the potential advantages of our RADR® platform in identifying drug candidates and patient populations that are likely to respond to a drug candidate; our strategic plans to advance the development of our drug candidates and antibody drug conjugate (ADC) development program; estimates regarding the development timing for our drug candidates and ADC development program; expectations and estimates regarding clinical trial timing and patient enrollment; our research and development efforts of our internal drug discovery programs and the utilization of our RADR® platform to streamline the drug development process; our intention to leverage artificial intelligence, machine learning and biomarker data to streamline and transform the pace, risk and cost of oncology drug discovery and development and to identify patient populations that would likely respond to a drug candidate; estimates regarding patient populations, potential markets and potential market sizes; sales estimates for our drug candidates and our plans to discover and develop drug candidates and to maximize their commercial potential by advancing such drug candidates ourselves or in collaboration with others. Any statements that are not statements of historical fact (including, without limitation, statements that use words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "model," "objective," "aim," "upcoming," "should," "will," "would," or the negative of these words or other similar expressions) should be considered forward-looking statements. There are a number of important factors that could cause our actual results to differ materially from those indicated by the forward-looking statements, such as (i) the risk that our research and the research of our collaborators may not be successful, (ii) the risk that none of our product candidates has received FDA marketing approval, and we may not be able to successfully initiate, conduct, or conclude clinical testing for or obtain marketing approval for our product candidates, (iii) the risk that no drug product based on our proprietary RADR® AI platform has received FDA marketing approval or otherwise been incorporated into a commercial product, and (iv) those other factors set forth in the Risk Factors section in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission on March 20, 2023. You may access our Annual Report on Form 10-K for the year ended December 31, 2022 under the investor SEC filings tab of our website at or on the SEC's website at . Given these risks and uncertainties, we can give no assurances that our forward-looking statements will prove to be accurate, or that any other results or events projected or contemplated by our forward-looking statements will in fact occur, and we caution investors not to place undue reliance on these statements. All forward-looking statements in this press release represent our judgment as of the date hereof, and, except as otherwise required by law, we disclaim any obligation to update any forward-looking statements to conform the statement to actual results or changes in our expectations.

Phase 1 dose escalation for VIP943 - first antibody-drug conjugate (ADC) from VersAptx™, versatile and adaptable, next-generation bioconjugation platform - moves quickly into 2nd cohort Phase 1 dose escalation for VIP236 - first-in-class small molecule-drug conjugate (SMDC) for treatment of solid tumors - progressing well with optimized dosing schedule Phase 1 dose escalation combination study for enitociclib in collaboration with NIH - partial response in first patient dosed in second cohort Cash balance expected to support planned operations into late 2024 PALO ALTO, Calif., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Vincerx Pharma, Inc. (Nasdaq: VINC)(“Vincerx”), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today reported financial results for the third quarter ended September 30, 2023 and provided a corporate update. “This has been a standout quarter for Vincerx, with progress across all three clinical programs, including VIP943, our first ADC from the VersAptx Platform, our versatile and adaptable, next-generation bioconjugation platform, and VIP236, our first-in-class SMDC,” said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. “The Vincerx team has done an outstanding job delivering ahead of expectations. Not only were we able to dose our first VIP943 patient within a matter of weeks of FDA clearance, but we have quickly moved through our first cohort and are beginning enrollment in our second cohort. This momentum is a testament to the promising safety profile seen in the first cohort and the enthusiasm from investigators participating in the trial. We expect to report preliminary data from this study in mid-2024.” “We also continued to make excellent progress with our other clinical programs. VIP236, our first-in-class SMDC for advanced solid tumors, is dosing patients under an optimized dosing schedule of once every three weeks, and we are pleased with the early safety profile we are seeing. We expect to report preliminary data in early 2024. In addition, enitociclib, our CDK9 inhibitor, continues to show promise. The transformed follicular lymphoma patient in our monotherapy study remains on treatment and has achieved long-term stable disease for more than 16 months with a 51% reduction in target lesions. Furthermore, our collaboration with the National Institute of Health (NIH) on a Phase 1 dose-escalation study of enitociclib with venetoclax and prednisone continues to make progress, with four patients dosed and an overall favorable safety profile. Even more exciting, the first patient on the second dose level remains on study with a partial response with evidence of an 80% reduction in the pulmonary lesion and resolution of skin lesions from their peripheral T-cell lymphoma,” added Dr. Hamdy. “We continue to take a very disciplined approach to our portfolio, focusing our resources on our two lead programs, VIP943 and VIP236. Concurrently, we are exploring business development opportunities that can advance all of our clinical programs as well as leverage the power of our VersAptx Platform. The therapeutic and safety potential of ADCs and other conjugates is being widely recognized, as evidenced by the overall excitement and activity in the ADC space, including collaborations, acquisitions, and positive clinical results. We believe that our next-generation ADC technology represents a significant step forward in the treatment of cancer by potentially overcoming the safety and efficacy challenges associated with many ADCs. As a result, we feel we are well-positioned to capitalize on industry enthusiasm for bioconjugates,” concluded Dr. Hamdy. Third Quarter 2023 Corporate Highlights: VIP943: CD123-KSPi ADC for leukemias and myelodysplastic syndrome VIP943, the first ADC from our VersAptx Platform, consists of an anti-CD123 antibody, a unique linker cleaved intracellularly by legumain, and a novel kinesin spindle protein inhibitor (KSPi) payload enhanced with our CellTrapper® technology. Our proprietary effector chemistry (linker + payload) was designed to reduce non-specific release of the payload and ensure payload accumulation in cancer cells versus healthy cells. The increased therapeutic index has the potential to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities.VIP943 is in a Phase 1 dose-escalation trial evaluating patients with relapsed/refractory acute myeloid leukemia, myelodysplastic syndrome, and B-cell acute lymphoblastic leukemia who have exhausted standard therapeutic options (NCT06034275).The VIP943 IND was approved by the FDA within 30 days, and we dosed our first patient in less than three weeks of FDA clearance. In addition, enrollment has moved quickly (Cohort 1, n=3), and we are now enrolling our second cohort. We are pleased with the early safety profile.We will present new data on robust preclinical activity at the 65th American Society for Hematology Meeting (ASH 2023) in December.We expect to report preliminary clinical trial data in mid-2024. VIP236: SMDC with αvβ3 integrin binder linked to optimized CPT payload for solid tumors VIP236, the first-in-class SMDC from our VersAptx Platform, consists of an αvβ3 integrin binder, a neutrophil elastase linker cleaved in the tumor microenvironment, and a camptothecin (CPT) payload optimized for high permeability and low efflux. VIP236 was designed to deliver its payload to advanced/metastatic tumors that express αvβ3.Preclinical data show enhanced efficacy, independent of HER2 status, in patient-derived and cell line-derived gastric cancer models compared with ENHERTU®, an approved ADC. VIP236 is being evaluated in a Phase 1 dose-escalation trial treating patients with advanced or metastatic solid tumors (NTC05371054). As VIP236 is a first-in-class drug, the Phase 1 trial is evaluating various dosing schedules. To date, 10 patients with advanced or metastatic disease that has relapsed or is refractory to standard of care have received VIP236; the early safety profile of once every three weeks dosing is promising. We expect to report preliminary clinical trial data in early 2024. VIP924: CXCR5-KSPi ADC for B-cell malignancies VIP924, the second ADC from our VersAptx Platform, consists of an anti-CXCR5 antibody, a unique linker cleaved intracellularly by legumain, and a novel KSPi payload enhanced with our CellTrapper technology. Our proprietary effector chemistry (linker + payload) was designed to reduce non-specific release of the payload and ensure payload accumulation in cancer cells versus healthy cells. The increased therapeutic index has the potential to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities. VIP924 preclinical data demonstrate significant tumor growth inhibition in a panel of lymphoma cell lines and cell line- and PDX-derived lymphoma models. VIP924 also induces sustained tumor regression in mantle cell lymphoma (MCL) and diffuse large B-cell lymphoma (DLBCL) models, including ibrutinib-refractory MCL models. At ASH 2023, preclinical data will be presented showing that our first-in-class anti-CXCR5 ADC compares favorably to the approved ADCs, Polivy® and Zynlonta®. Despite compelling preclinical data, we continue to judiciously pace our investment in VIP924 to focus resources on VIP236 and VIP943. Enitociclib: CDK9 inhibitor for hematologic malignancies and solid tumors Enitociclib, a highly selective CDK9 inhibitor, prevents activation of RNA polymerase II, resulting in reduction of known oncogenes MYC and MCL1. Enitociclib is in a dose-escalation Phase 1 trial (NTC05371054) in collaboration with the NIH evaluating the combination of enitociclib, venetoclax and prednisone in DLBCL and peripheral T-cell lymphoma (PTCL). The first dose level completed enrollment with no drug-related safety signal (n=3; 1=DLBCL, 2=PTCL). The first patient on the second dose level (n=1; 1=PTCL) remains on study with a partial response due to an 80% reduction in the pulmonary lesion on computerized tomography (CT) scan and resolved skin lesions. Investigators are pleased with the safety profile of this novel combination and continue with enrollment. Additional combination studies will be determined based on financing/partnering support. Proof-of-concept monotherapy efficacy has been generated in early-stage clinical studies: Hematologic malignancies: two patients with double-hit DLBCL achieved complete metabolic responses after 10 cycles of treatment and remain in remission, off treatment, for over four years; one patient with transformed follicular lymphoma remains on treatment, achieving long-term stable disease for over 16 months with a 51% reduction in target lesions.Solid tumors: 13 responses of stable disease, including durable disease control, in patients with ovarian, pancreatic, and salivary gland cancer. Strong preclinical evidence for additional indications (eg, multiple myeloma) and pediatric indications. Collaborators from the University of Calgary will present a poster at ASH 2023 showing preclinical activity in pediatric leukemia. Additional Corporate Highlights Our next-generation bioconjugation platform is now known as the VersAptx Platform. This name reflects the versatile and adaptable features and strengths of this innovative technology. We received a one-year extension of Small and Medium-Sized Enterprise (SME) status by the European Medicines Agency’s (EMA) Micro, Small and Medium-Sized Enterprise, enabling us to become eligible for EMA fee reductions and waiver and other financial incentives. Third Quarter 2023 Financial Results Vincerx had approximately $20.8 million in cash, cash equivalents and marketable securities as of September 30, 2023, as compared to approximately $52.5 million as of December 31, 2022. Based on its current business plans and assumptions, Vincerx believes its available capital will be sufficient to meet its planned operating requirements into late 2024.Research and development expenses for the three- and nine-months ended September 30, 2023 were approximately $6.8 million and $25.3 million, as compared to approximately $11.1 million and $40.8 million for the same periods in 2022. The decrease for the three months ended September 30, 2023 compared with the same period in 2022 is primarily the result of decreases in manufacturing services associated with our ADC program of approximately $4.1 million and clinical services of approximately $0.9 million, partially offset by the $1.0 million development milestone in connection with Vincerx’s IND filing for VIP943. The decrease for the nine months ended September 30, 2023 compared with the same period in 2022 is primarily the result of decreases in manufacturing services associated with our ADC program of approximately $5.5 million, stock-based compensation expense of approximately $5.1 million, clinical services of approximately $2.7 million, and payroll related costs of approximately $1.7 million as a result of our headcount reduction in June 2022.General and administrative expenses for the three- and nine-months ended September 30, 2023 were approximately $3.5 million and $11.8 million, as compared to approximately $4.5 million and $14.9 million for the same periods in 2022. These decreases are primarily due to decreases in stock-based compensation expense of approximately $0.5 million and $2.3 million for the three- and nine-months ended September 30, 2023, respectively, as well as a decrease in professional services of $0.5 million and $0.8 million for the three and nine months ended September 30, 2023, respectively.For the quarter ended September 30, 2023, Vincerx reported a net loss of approximately $9.7 million, or $0.46 per share, compared to a net loss of approximately $16.9 million, or $0.80 per share, for the quarter ended September 30, 2022. About Vincerx Pharma, Inc. Vincerx Pharma, Inc. is a clinical-stage biopharmaceutical company committed to developing differentiated and novel therapies to address the unmet medical needs of patients with cancer. Vincerx has assembled a seasoned management team with a proven track record of successful oncology drug development, approvals, and value creation. Vincerx’s diverse pipeline consists of the next-generation antibody-drug conjugate, VIP943, in Phase 1; small molecule-drug conjugate, VIP236, in Phase 1; preclinical antibody-drug conjugate, VIP924; CDK9 inhibitor, enitociclib, currently in an NIH-sponsored Phase 1; and VersAptx, its versatile and adaptable, next-generation bioconjugation platform. Vincerx is based in Palo Alto, California, and has a research facility in Monheim, Germany. For more information, please visit www.vincerx.com. Forward-Looking Statement This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended, that are intended to be covered by the “safe harbor” created by those sections. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, expectations and events, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “suggest,” “seek,” “intend,” “plan,” “goal,” “potential,” “on-target,” “on track,” “project,” “estimate,” “anticipate,” or other comparable terms. All statements other than statements of historical facts included in this press release are forward-looking statements. Forward-looking statements include, but are not limited to: Vincerx’s business model, expected cash runway, pipeline, strategy, timeline, product candidates and attributes, and preclinical and clinical development, timing, and results. Forward-looking statements are neither historical facts nor assurances of future performance or events. Instead, they are based only on current beliefs, expectations, and assumptions regarding future business developments, future plans and strategies, projections, anticipated events and trends, the economy, and other future conditions. Forward-looking statements are subject to inherent uncertainties, risks, and changes in circumstances that are difficult to predict and many of which are outside of Vincerx’s control. Actual results, conditions, and events may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause actual results, conditions, and events to differ materially from those indicated in the forward-looking statements include, but are not limited to: general economic, financial, legal, political, and business conditions; risks associated with preclinical or clinical development and trials, including those conducted prior to Vincerx’s in-licensing; failure to realize the benefits of Vincerx’s license agreement with Bayer; risks related to the rollout of Vincerx’s business and the timing of expected business and product development milestones; changes in the assumptions underlying Vincerx’s expectations regarding its future business or business model; Vincerx’s ability to successfully develop and commercialize product candidates; Vincerx’s capital requirements and availability and uses of capital; and the risks and uncertainties set forth in Form 10-Q for the quarter ended June 30, 2023 and other reports filed with the Securities and Exchange Commission by Vincerx. Forward-looking statements speak only as of the date hereof, and Vincerx disclaims any obligation to update any forward-looking statements. Vincerx, the Vincerx logo, CellTrapper, and VersAptx are our trademarks. This press release also contains trademarks and trade names that are the property of their respective owners. Contacts Joyce LonerganLifeSci Advisors, LLC781-528-5276jlonergan@lifesciadvisors.com Vincerx Pharma, Inc.Condensed Consolidated Balance Sheets(in thousands) September 30, 2023 December 31, 2022 (unaudited) ASSETS Current assets: Cash and cash equivalents$15,078 $11,663 Short-term marketable securities 5,724 40,796 Prepaid expenses 322 134 Other current assets 1,694 3,371 Total current assets 22,818 55,964 Right-of-use assets 2,447 3,064 Property, plant and equipment, net 137 177 Other assets 874 81 Total assets$ 26,276 $ 59,286 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable$3,062 $4,065 Accrued expenses 4,526 3,923 Lease liability 1,126 1,024 Common stock warrant liabilities 132 144 Total current liabilities 8,846 9,156 Lease liability, net of current portion 1,639 2,412 Other noncurrent liabilities 50 50 Total liabilities 10,535 11,618 Total stockholders' equity 15,741 47,668 Total liabilities and stockholders' equity$ 26,276 $ 59,286 Vincerx Pharma, Inc.Condensed Consolidated Statements of Operations(unaudited)(in thousands, except per share amounts) For the three months ended For the nine months ended September 30, September 30, 2023 2022 2023 2022 Operating expenses: General and administrative$3,517 $4,525 $11,816 $14,903 Research and development 6,800 11,066 25,260 40,779 Restructuring - 1,310 - 2,469 Total operating expenses 10,317 16,901 37,076 58,151 Loss from operations (10,317) (16,901) (37,076) (58,151) Other income (expense) Change in fair value of warrant liabilities 112 (79) 12 6,334 Interest income 260 204 1,053 204 Other income (expense) 230 (103) 804 (111) Total other income (expense) 602 22 1,869 6,427 Net loss $ (9,715) $ (16,879) $ (35,207) $ (51,724) Net loss per common share, basic and diluted$(0.46) $(0.80) $(1.66) $(2.46) Weighted average common shares outstanding, basic and diluted 21,345 21,083 21,269 20,992

-Update provided for both Part A (single dose with optional re-dosing) and Part B (consolidation dosing) of the Phase 1 CARBON™ clinical trial- -Part A data presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition confirm that CTX110® can lead to long-term durable complete remissions (CRs) for heavily pre-treated Large B-cell Lymphoma (LBCL) patients; three patients remain in CR beyond two years- -Emerging data from Part B demonstrate the benefits of consolidation dosing with a positively differentiated safety profile consistent with Part A- -Based on discussions with regulatory agencies, the Company has initiated a Phase 2 single-arm potentially registrational trial with drug product manufactured using a commercial-ready process and specifications- ZUG, Switzerland and BOSTON, Dec. 12, 2022 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today provided an update for both Part A and Part B of the Company’s ongoing Phase 1 CARBON trial evaluating the safety and efficacy of CTX110®, its wholly-owned allogeneic CAR T cell therapy targeting CD19+ B-cell malignancies. Part A data, presented at ASH, showed the potential for CTX110 to achieve long-term durable complete remissions (CRs) with a positively differentiated safety profile in heavily pre-treated patients, and emerging data from Part B showed an encouraging efficacy profile with several patients in ongoing CR beyond six months. “We are excited by these results in our CARBON trial, which demonstrate the potential of a single course of allogeneic CAR T treatment to produce long-lasting complete remissions in heavily pre-treated patients with LBCL,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “Furthermore, we are encouraged by the emerging data using consolidation dosing, which has the potential to further improve the efficacy profile while maintaining a positively differentiated safety profile. Based on these results, we have initiated a potentially registrational Phase 2 trial of CTX110 incorporating consolidation dosing with the hope of bringing this important therapy to patients in the near term.” CARBON Trial OverviewThe Phase 1 CARBON trial is an open-label, multicenter clinical trial evaluating the safety and efficacy of CTX110 in adult patients with relapsed or refractory CD19+ B-cell malignancies who have received at least two prior lines of therapy. To date, enrollment has been focused on patients with the most aggressive disease presentations, including Diffuse Large B-cell Lymphoma (DLBCL) not otherwise specified (NOS), high-grade double- or triple-hit lymphomas, transformed follicular lymphoma, and grade 3B follicular lymphoma. In Part A of the trial, patients were infused with a single dose of CTX110 following three days of a standard lymphodepletion regimen consisting of fludarabine (30mg/m2/day) and cyclophosphamide (500mg/m2/day). Patients received CTX110 at doses ranging from Dose Level (DL) 1 (30 million CAR+ T cells) to DL4 (600 million CAR+ T cells), with an option to re-dose CTX110 based on clinical benefit. In Part B of the trial, patients received CTX110 at DL4 following standard lymphodepletion, as well as a consolidation dose of CTX110 at the same dose level between four and eight weeks after the initial dose for patients that demonstrate clinical benefit. The primary endpoints include safety as measured by the incidence of dose limiting toxicities (DLTs) and overall response rate (ORR). Key secondary endpoints include complete response (CR) rate, duration of response (DOR) and overall survival (OS). PART A Data presented at ASH (Poster #4629) show the potential for single infusions of CTX110 to achieve long-term durable complete remissions with a positively differentiated safety profile. In a heavily pre-treated patient population with relapsed or refractory (R/R) LBCL (47% with ≥3 prior lines of therapy), CTX110 at DL≥3 (n=27) resulted in an ORR of 67% and CR rate of 41%. Three patients remain in ongoing CR two years after treatment, and two additional patients remain in CR past one year.No DLTs, no Graft versus Host Disease (GvHD) of any grade, and no Grade ≥3 cytokine release syndrome (CRS) events were observed.These data formed the basis for Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for CTX110, granted in November 2021. PART B Emerging data from Part B shows an encouraging efficacy profile with several patients in ongoing CR beyond six months. Clear evidence of the benefits of consolidation dosing was observed, with deepening of CRs and conversions of stable disease and partial response to ongoing CRs after the second dose.Safety profile remained consistent with Part A, confirming the tolerability of the consolidation regimen.Peak expansion and overall pharmacokinetics of CTX110 were comparable between the initial and consolidation doses.The Company plans to present additional Part B data at a future medical meeting. Following discussions with regulatory agencies, the Company has initiated a single-arm, potentially registrational trial of CTX110, which incorporates consolidation dosing at DL4 and standard lymphodepletion. Dosing in this trial is expected to begin in early 2023 using drug product manufactured with a commercial-ready process and specifications. About CTX110 and CARBON TrialCTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial, a Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies. CTX110 has been granted RMAT designation by the FDA. About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com. CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni in this press release, as well as statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) the safety, efficacy and clinical progress of its CTX110 program, including the actual or potential benefits of regulatory designations; (ii) the status of clinical trials (including, without limitation, the expected timing of data releases) and expectations regarding the data and information being presented from our CARBON clinical trial; (iii) the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies, including as compared to other therapies. Without limiting the foregoing, the words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics’ product candidates; uncertainties regarding the intellectual property protection for CRISPR Therapeutics’ technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics’ most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. CRISPR THERAPEUTICS® standard character mark and design logo and CTX110® are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners. Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com Media Contact:Rachel Eides+1-617-315-4167 rachel.eides@crisprtx.com