On May 9, 2022 aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of innovative medicines based on its proprietary tRNA synthetase biology platform, reported first quarter 2022 results and provided a corporate update (Press release, aTyr Pharma, MAY 9, 2022, View Source [SID1234614113]).

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"We are pleased with the start of 2022 and the continued progress throughout the first quarter for our efzofitimod clinical program in pulmonary sarcoidosis, our initial interstitial lung disease (ILD) indication," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "With the receipt of U.S. Food and Drug Administration (FDA) orphan drug designation and a positive End-of-Phase 2 meeting with the FDA, we are on track to initiate a planned registrational study of efzofitimod in pulmonary sarcoidosis in the third quarter of this year."

"The second quarter is shaping up to be an important period, as we prepare to present the clinical data for the Phase 1b/2a study of efzofitimod in pulmonary sarcoidosis at the upcoming American Thoracic Society (ATS) International Conference and anticipate the potential publication of a related manuscript. We are focused on preparation for the planned registrational study so that the balance of the year may center upon initiating the study in the U.S. and Europe and supporting our partner Kyorin Pharmaceutical with the anticipated launch of the study in Japan."

First Quarter 2022 and Subsequent Period Highlights

Announced posters accepted for presentation for efzofitimod in pulmonary sarcoidosis at the upcoming ATS 2022 International Conference. The posters will present clinical data from the recently completed Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated safety, tolerability and consistent dose response for efzofitimod on key efficacy endpoints and improvements compared to placebo, including measures of steroid reduction, lung function, sarcoidosis symptom measures and inflammatory biomarkers.
Announced a company reception at the upcoming ATS 2022 International Conference in San Francisco, CA, on Monday, May 16, 2022. The event, which will convene sarcoidosis medical experts, principal investigators, advocacy organizations, analysts and investors, will review results from the Phase 1b/2a study of efzofitimod in pulmonary sarcoidosis and discuss an outlook for the planned registrational study that the company expects to initiate in the third quarter of 2022.
Received FDA orphan drug designation for efzofitimod for the treatment of systemic sclerosis (SSc, or scleroderma), a chronic, progressive autoimmune disease in which many patients may develop associated ILD, known as SSc-ILD. Orphan drug designation is granted to support the development of medicines for patients with unmet needs for rare disorders affecting fewer than 200,000 people in the U.S. and provides certain benefits, including the potential for seven years of market exclusivity following regulatory approval. The company expects to explore the potential expansion of its efzofitimod clinical program into other forms of ILD with high unmet medical need.
Presented preclinical research in a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting characterizing the effects of ATYR2810, the company’s lead anti-Neuropilin-2 (NRP2)/VEGF antibody and IND candidate, in highly aggressive tumor subtypes, including triple-negative breast cancer. The findings suggest that ATYR2810 reduces metastasis and enhances chemosensitivity by downregulating key genes linked to these processes. The company expects to initiate a phase 1 study of ATYR2810 in cancer patients in the second half of 2022.
First Quarter 2022 Financial Highlights and Cash Position

Cash & Investment Position: Cash, cash equivalents and investments as of March 31, 2022, were $98.7 million.
R&D Expenses: Research and development expenses were $8.9 million for the first quarter of 2022, which consisted primarily of product development and manufacturing costs for the efzofitimod and ATYR2810 programs.
G&A Expenses: General and administrative expenses were $3.5 million for the first quarter of 2022.
Shares Outstanding: Common shares outstanding were 28,056,249 as of March 31, 2022.
Conference Call and Webcast Details

aTyr will host a conference call and webcast today at 5:00 p.m. EDT / 2:00 p.m. PDT to discuss its financial results and provide a corporate update. Interested parties may access the call by dialing toll-free 844-358-9116 from the U.S., or 209-905-5951 internationally and using conference ID 9273437. Links to a live audio webcast and replay may be accessed on the aTyr website events page at: View Source An audio replay will be available for at least 90 days following the event.

About Efzofitimod

aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease. Efzofitimod, a fusion protein comprised of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates innate and adaptive immune response in inflammatory disease states. aTyr’s lead indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease. Clinical proof-of-concept for efzofitimod was recently established in a Phase 1b/2a multiple-ascending dose, placebo-controlled study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated safety and a consistent dose response and trends of benefit of efzofitimod compared to placebo on key efficacy endpoints, including steroid reduction, lung function, clinical symptoms and inflammatory biomarkers. aTyr intends to initiate a planned registrational study of efzofitimod in pulmonary sarcoidosis in the third quarter of 2022.

On May 9, 2022 Aadi Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company focused on developing and commercializing precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, reported partnerships with prominent next generation sequencing (NGS) providers and leaders in genomic testing and profiling, including Foundation Medicine, Tempus and others (Press release, Aadi Bioscience, MAY 9, 2022, View Source [SID1234614205]). We expect these collaborations to expedite patient identification for the ongoing PRECISION 1 trial of nab-sirolimus in patients harboring tumors with inactivating alterations in TSC1 or TSC2 genes.

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Neil Desai, PhD, Founder, President and Chief Executive Officer of Aadi, stated, "We are very pleased to have these leading NGS partnerships in place. Recently published data project an incidence of approximately 12,000 advanced cancer patients with TSC1 or TSC2 definite impact alterations in the US. These collaborations should help physicians to identify patients who may be candidates for nab-sirolimus in our PRECISION 1 registrational trial. We expect to report preliminary data from this trial in the first half of next year."

Loretta Itri, M.D., Chief Medical Officer of Aadi, stated, "We are excited to announce partnerships with the leading molecular diagnostic companies, including Foundation Medicine, Tempus and others. Many of our clinical trial sites had already been utilizing the NGS reports provided by these companies. Now our partnerships will allow us to also leverage their established patient enrollment programs, which we believe will significantly augment our efforts to identify and recruit patients with TSC1 and TSC2 alterations for our PRECISION 1 registrational trial. We are pleased with our progress to-date and remain confident we will meet our enrollment goals."

These partners will collect and analyze clinical and molecular data to determine which patients may be eligible to participate in Aadi’s PRECISION 1 clinical trial. The goals are to increase patient match rate, reduce screen failure rate and increase speed of enrollment into the trial.

About the PRECISION 1 Trial

The PRECISION 1 trial is a multi-center, open-label, tumor-agnostic pivotal study, of nab-sirolimus designed as a basket trial that will evaluate approximately 120 adult and adolescent patients with solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes. The trial will have two independent arms of 60 patients each to separately evaluate patients with either TSC1 or TSC2 inactivating alterations. Aadi has received Fast Track designation to evaluate nab-sirolimus in this indication from the FDA. The first patient on the PRECISION 1 trial was dosed in March 2022.

On May 9, 2022 Gnubiotics Sciences SA (Gnubiotics) reported a strategic partnership agreement with ADM, a global leader in human and animal nutrition (Press release, Gnubiotics Sciences, MAY 9, 2022, View Source [SID1234613872]). The strategic partnership is centered on the commercialization of innovative microbiome solutions for companion animal health and wellbeing.

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This strategic partnership with ADM is a major accomplishment for Gnubiotics as it represents a validation of our science and technology.

"We´re excited about this partnership with Gnubiotics, and the capabilities they bring, enabling pet parents to better understand their pets’ microbiome. With this knowledge, ADM can leverage its comprehensive pantry of pet and animal well-being solutions to improve individual pet health through precise nutrition and supplementation," said Gustavo Zenaide, Vice President of ADM Pet & Animal Well-being.

The gut microbiome is essential to the health and well-being of companion animals. Study data from Gnubiotics show Animal Milk Oligosaccharides (A.M.O.s) manufactured by Gnubiotics helped protect the microbiome of dogs from the negative effects of antibiotics. In 2021, Gnubiotics also announced patents covering microbiome-based markers to predict, diagnose and treat feline obesity, a major cause of diabetes and chronic kidney disease (CKD) in felines.

"We are pleased to work with ADM, a global leader in human and animal health and nutrition," said Yemi Adesokan, CEO and Co-Founder, Gnubiotics. "We have developed products which have functional and clinically demonstrated applications for animal health and well-being. This strategic partnership with ADM is a major accomplishment for Gnubiotics as it represents validation of our science and technology. We are excited to collaborate with ADM to continue to develop and bring to market, data-driven microbiome-targeted solutions."

On May 9, 2022 Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the "Company"), a clinical-stage macrophage reprogramming immunotherapy company, reported the poster presentation of a substantial survival benefit of AllocetraTM combined with immune checkpoint inhibition in a preclinical mesothelioma study at the International Society of Cell and Gene Therapy Annual 2022 Meeting (Press release, Enlivex Therapeutics, MAY 9, 2022, View Source [SID1234613905]). The poster was titled, "Allocetra-OTS, an Early Apoptotic Cellular Therapy Synergize with Chimeric Antigen Receptor (CAR) T Cell Therapy or Immune Check Point Inhibitor Against Peritoneal Solid Tumor."

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Mesothelioma, Treatment Landscape, and Macrophage-Solid Tumor Dynamics

Mesothelioma is one of the deadliest solid cancers with few treatment options, all of which have limited efficacy. People who are most at risk to develop mesothelioma generally have had long-term exposure to asbestos (e.g., construction workers, pipe fitters, and shipyard workers). It takes many years for mesothelioma to develop; it can appear 30 years after asbestos exposure. Immune checkpoint inhibitors targeting CTLA4 and PD1 are FDA-approved as first-line treatments for patients with unresectable malignant pleural mesothelioma.

In mesothelioma and in some other solid cancers, tumor cells, as part of their defense mechanisms, facilitate the recruitment of macrophages which become "pro-tumor" tumor associated macrophages (TAMs) rather than "anti-tumor" macrophages. The TAMs typically form a physical layer on top of the solid tumor, and induce immunosuppression in the solid tumor microenvironment. This in-turn promotes tumor growth and metastasis and makes it very difficult for the immune system or any anti-cancer drug to efficiently attack the cancerous cells. Allocetra is a cell therapy in development that is targeted at these TAMs, and its proposed mechanism of action is to change the balance of macrophage populations to skew towards anti-tumor macrophages and away from pro-tumor macrophages.

The Mesothelioma Model

To test the potential effect of cell therapy-induced macrophage reprogramming on difficult-to-treat solid tumors, Enlivex ran preclinical studies in which five groups of mice were implanted with mesothelioma cancer cells. The difference between the groups was the treatment given, which started on day 12 after the cancer’s initial growth period.

Results from multiple studies strongly support the potential of AllocetraTM to assist in the process of macrophage reprogramming in the solid tumor microenvironment and significantly change survival outcomes. As expected, only 1/16 (6%) of the untreated mice remained alive at day 42 (that one last remaining mouse died on day 65), compared with up to 25% survival for the mice in the group that was treated with the anti-CTLA4 immune checkpoint inhibitor. Surprisingly, AllocetraTM as a stand-alone treatment provided comparable survival rates as anti-CTLA4 monotherapy, with a 28.5% survival rate and a delayed cancer growth rate observed. Notably, the synergistic effect of treating with both drugs resulted in up to 100% survival with complete disappearance of the cancer. The effect was correlated with the AllocetraTM treatment dose. A lower dose of AllocetraTM provided slightly lower survival and cancer complete remission rates (67% complete remission for the low-dose treatment vs. up to 100% for the higher dose). The following chart provides a visual guide to the progressions/regression of the mesothelioma cancer in the different groups, as recorded for study mesothelioma AB12 137:

Across multiple studies of the AB12 mesothelioma model, anti-CTLA4 therapy significantly improved survival duration from mean 34±9 to 44.9 ±20 days (p<0.05). Allocetra as a stand-alone therapy improved survival duration to 52.3 ±20 days (p<0.02). The synergistic effect of the combination therapy of anti-CTLA4 + AllocetraTM improved survival duration to 86.7±20 days (p<0.0001) with complete cancer remission in 60-100% of mice, depending on the dose administered.

Prof. Dror Mevorach, Chief Scientific Officer of Enlivex, stated: "This reproducible and statistically significant data strongly support Allocetra’s proposed therapeutic mechanism of action of reprogramming macrophages in solid tumors. We believe that, in contrast to CAR-T, CAR-NK and other anti-cancer cell therapies directed at tumor antigens, AllocetraTM restores macrophage homeostasis in the tumor environment via reprogramming of pro-tumor macrophages. This may ultimately allow immune checkpoint inhibitors to be exponentially more effective."

Oren Hershkovitz, Ph.D., CEO of Enlivex, added: "Based on these results, Enlivex is planning to initiate a series of clinical trials during 2022 that are designed to evaluate Allocetra in combination with FDA-approved immune checkpoint inhibitor therapies in patients with advanced-stage solid tumors. To date, we have infused AllocetraTM in dozens of hospitalized patients in fragile condition resulting from sepsis and COVID-19-related organ dysfunction. We believe that the proposed differentiated mechanism of action of AllocetraTM, together with the safety and tolerability it has demonstrated in these prior studies, as well as the encouraging efficacy results of our preclinical solid tumor models, highlight an intriguing opportunity for Enlivex to provide another layer of life-saving therapy for patients who have few available options."

ABOUT ALLOCETRA

Allocetra is being developed as a universal, off-the-shelf cell therapy designed to reprogram macrophages into their homeostatic state. Diseases such as solid cancers, sepsis, and many others reprogram macrophages out of their homeostatic state. These non-homeostatic macrophages contribute significantly to the severity of the respective diseases. By restoring macrophage homeostasis, Allocetra has the potential to provide a novel immunotherapeutic mechanism of action for life-threatening clinical indications that are defined as "unmet medical needs", as a stand-alone therapy or in combination with leading therapeutic agents.

On May 9, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the first quarter ended March 31, 2022 (Press release, CRISPR Therapeutics, MAY 9, 2022, View Source [SID1234613921]).

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"I am pleased with the ongoing momentum across our broad portfolio of innovative gene therapy candidates and anticipate important company milestones in 2022. Alongside our partner Vertex, we remain on track to submit global regulatory filings for CTX001 in late 2022 and have dosed more than 75 patients across both trials to date. We have also initiated two new Phase 3 trials of CTX001 in pediatric patients with TDT and SCD," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "We are also advancing our wholly-owned immuno-oncology pipeline, with new updates expected this year. In addition, enrollment and dosing continues in the Phase 1 clinical trial of VCTX210 for T1D with our partner, ViaCyte. We believe we are well positioned and well capitalized to advance our pipeline and platform to develop transformative medicines for patients suffering from serious diseases."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

Following the completion of enrollment in the ongoing Phase 3 clinical trials for CTX001 in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), announced last quarter, more than 75 patients across both trials have been dosed to date. CRISPR Therapeutics and Vertex anticipate presenting updated data from the clinical trials, with more patients and longer follow-up, at medical conferences in 2022.

CRISPR Therapeutics and Vertex have initiated two new Phase 3 studies of CTX001 in pediatric patients with TDT and SCD.

The companies anticipate submitting global regulatory filings for CTX001 in TDT and SCD in late 2022.

Immuno-Oncology Programs

CRISPR Therapeutics continues to enroll and dose patients in the pivotal trial of CTX110, its wholly-owned allogeneic chimeric antigen receptor T cell (CAR-T) investigational therapy targeting CD19+ B-cell malignancies. The Company expects to report additional data in 2022.

CRISPR Therapeutics’ Phase 1 clinical trials for CTX-120, its wholly-owned allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma, and CTX130, its wholly-owned allogeneic CAR-T investigational therapy targeting CD70 for the treatment of both solid tumors and certain hematologic malignancies, are ongoing. Each trial is assessing safety and efficacy of several dose levels. The Company expects to provide updates from each trial in the first half of 2022.

Regenerative Medicine and In Vivo Programs

Enrollment and dosing are ongoing in the Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes (T1D). VCTX210 is an investigational, allogeneic, gene-edited, stem cell-derived product developed in collaboration by applying CRISPR Therapeutics’ gene-editing technology to ViaCyte’s proprietary stem cell capabilities for the generation of pancreatic cells designed to evade recognition by the immune system. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin.

Based upon ongoing progress with its in vivo approaches for liver gene editing utilizing both viral and non-viral delivery vehicles, CRISPR Therapeutics continues to expect to move multiple programs utilizing in vivo approaches into the clinic in the next 18 to 24 months.

Other Corporate Matters

In April, CRISPR Therapeutics proposed to elect Maria Fardis, Ph.D., MBA, to its Board of Directors at the Company’s upcoming annual general meeting of shareholders to be held later this year. The Company believes her extensive leadership in scaling companies and bringing novel therapies to patients will be an invaluable asset to CRISPR Therapeutics.

In April, CRISPR Therapeutics and Nkarta, Inc. presented preclinical data focused on its natural killer cell platform and pipeline at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022. The data shows that CD70/CISH/CBLB triple KO CD70-CAR NK cells demonstrated enhanced anti-tumor activity against relevant solid tumor cell lines and provided greater resistance to tumor microenvironment inhibition. These data support the further exploration of CD70/CISH/CBLB triple gene knockout CD70 CAR NK cells for clinical application.

In April, CRISPR Therapeutics was awarded the 2022 Facility of the Year Category Award (FOYA) for Innovation by the International Society for Pharmaceutical Engineering (ISPE) for its state-of-the art manufacturing facility in Framingham, Massachusetts, USA. ISPE’s Facility of the Year Awards program is the premier global awards program recognizing innovation and creativity in the pharmaceutical and biotechnology manufacturing industries. Projects selected for recognition set the standard by demonstrating excellence in facility design, construction, and operations.
First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $2,221.3 million as of March 31, 2022, compared to $2,379.1 million as of December 31, 2021. The decrease in cash of $157.8 million was primarily driven by cash used in operating activities to support ongoing research and development of the Company’s clinical and pre-clinical programs.

Revenue: Total collaboration revenue was $0.2 million for the first quarter of 2022 and 2021.

R&D Expenses: R&D expenses were $118.2 million for the first quarter of 2022, compared to $70.6 million for the first quarter of 2021. The increase in expense was driven by development activities supporting the advancement of our wholly-owned immuno-oncology programs, as well as expenses related to our new U.S. research and development headquarters.

G&A Expenses: General and administrative expenses were $28.0 million for the first quarter of 2022, compared to $24.5 million for the first quarter of 2021. The increase in general and administrative expenses was primarily driven by headcount-related expense.

Collaboration Expense: Collaboration expense, net, was $30.6 million for the first quarter of 2022, compared to $19.9 million for the first quarter of 2021. The increase in collaboration expense, net, was primarily driven by increased pre-commercial and manufacturing scale-up costs associated with our hemoglobinopathies programs under our collaboration with Vertex.

Net Loss: Net loss was $179.2 million for the first quarter of 2022, compared to a net loss of $113.2 million for the first quarter of 2021.
About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex Collaboration
Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CLIMB-111
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131
This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About CTX110
CTX110, 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. CTX110 has been granted Regenerative Medicine Advanced Therapy designation from the FDA.

About CARBON
The ongoing 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.

About CTX120
CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively. CTX120 has been granted Orphan Drug designation for the treatment of T-cell lymphoma from the FDA.

About VCTX210
VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). VCTX210 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and ViaCyte, Inc.