CD22 x CD20 x CD19

Expect to disclose initial SC262 clinical data in 2024SEATTLE, Jan. 05, 2024 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application to initiate a study of SC262 in patients with relapsed or refractory B-cell malignancies, initially in patients who have received prior CD19-directed CAR T therapy. Engineered CAR T cell therapies for B-cell malignancies use binders to target proteins expressed on the surface of B cells. One such protein, CD19, has been the target of all approved autologous CAR T therapies for B-cell lymphoma and B-cell acute lymphoblastic leukemia to date. Unfortunately, incomplete responses or relapses occur in approximately 60% of CD19 CAR T-treated patients. CD22, which is also a B-cell surface protein, has emerged as an alternative to address failure to achieve durable complete responses with CD19-directed CAR T therapy. SC262 expresses the same CAR, including the same CD22 binder, used in CD22-directed CAR T therapies tested in multiple academic clinical trials. To date, these trials have shown durable complete responses in a substantial number of patients in the relapse setting following treatment with a CD19-directed CAR T therapy. “Patients who have failed a CD19-directed CAR T therapy represent a significant unmet need, and this population is growing as more patients receive these therapies,” said Doug Williams, PhD, Sana’s President of Research and Development. “SC262 represents an important potential option for these patients and is the next step in building Sana’s hypoimmune CAR T therapy platform. Over the past twelve months, Sana has received three IND regulatory clearances, as well as supported the authorization of an investigator-sponsored CTA, to begin new studies utilizing our hypoimmune platform in seven different indications in oncology, B-cell mediated autoimmune diseases, and type 1 diabetes. We look forward to presenting data from all of these studies this year, including initial proof of concept data for SC262 later this year.” About SC262 in B-cell MalignanciesSC262 is a hypoimmune, CD22-directed allogeneic CAR T cell therapy derived from healthy donor CD4+ and CD8+ T cells. SC262 is developed with Sana’s hypoimmune platform, which is designed to overcome the immunologic rejection of allogeneic cells and may result in longer CAR T cell persistence and a higher rate of durable complete responses for patients with B-cell malignancies. CD22 is expressed on lymphoma and leukemia cells in most patients with these diseases, including those that have failed CD19- and/or a CD20-directed therapies. SC262 is initially being explored in patients that have failed a previous CD19-directed therapy. The hypoimmune technology includes disruption of major histocompatibility (MHC) class I and MHC class II expression to allow cells to evade the adaptive immune system, which includes antibody and T cell responses, as well as overexpression of CD47 to evade the innate immune cell system, in particular macrophages and natural killer (NK) cells. The company has presented data across multiple preclinical models highlighting the potential of this platform to cloak cells from immune recognition and the potential of SC262 as a therapeutic for patients with B-cell malignancies. About Hypoimmune PlatformSana’s hypoimmune platform is designed to create cells ex vivo that can “hide” from the patient’s immune system to enable the transplant of allogeneic cells without the need for immunosuppression. We are applying the hypoimmune technology to both donor-derived allogeneic T cells, with the goal of making potent and persistent CAR T cells at scale, and pluripotent stem cells, which can then be differentiated into multiple cell types at scale. Preclinical data published in peer-reviewed journals demonstrate across a variety of cell types that these transplanted allogeneic cells are able to evade both the innate and adaptive arms of the immune system while retaining their activity. Our most advanced programs utilizing this platform include an allogeneic CAR T program targeting CD19+ cancers, an allogeneic CAR T program for B-cell mediated autoimmune diseases, an allogeneic CAR T program targeting CD22+ cancers, and stem-cell derived pancreatic islet cells for patients with type 1 diabetes. About Sana BiotechnologySana Biotechnology, Inc. is focused on creating and delivering engineered cells as medicines for patients. We share a vision of repairing and controlling genes, replacing missing or damaged cells, and making our therapies broadly available to patients. We are a passionate group of people working together to create an enduring company that changes how the world treats disease. Sana has operations in Seattle, Cambridge, South San Francisco, and Rochester. For more information about Sana Biotechnology, please visit https://sana.com/. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements about Sana Biotechnology, Inc. (the “Company,” “we,” “us,” or “our”) within the meaning of the federal securities laws, including those related to the Company’s vision, progress, and business plans; expectations for its development programs, product candidates and technology platforms, including its pre-clinical, clinical and regulatory development plans and timing expectations; the Company’s expectations regarding the timing and significance of data from its clinical trials; the potential of CD22-directed CAR T therapies to address failure to achieve durable complete responses; expectations regarding the growth of the potential patient population for SC262; the potential significance of the IND clearance for SC262; the ability to use the Company’s hypoimmune platform to evade immune recognition and overcome the immunologic rejection of allogeneic cells without immunosuppression and the potential benefits associated therewith; the potential of SC262 as a therapeutic for patients with B-cell malignancies; and the ability to apply the HIP technology to donor-derived allogeneic T cells to make potent and persistent CAR T cells at scale and pluripotent stem cells, which can then be differentiated into multiple cell types at scale. All statements other than statements of historical facts contained in this press release, including, among others, statements regarding the Company’s strategy, expectations, cash runway and future financial condition, future operations, and prospects, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “target,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. The Company has based these forward-looking statements largely on its current expectations, estimates, forecasts and projections about future events and financial trends that it believes may affect its financial condition, results of operations, business strategy and financial needs. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. These statements are subject to risks and uncertainties that could cause the actual results to vary materially, including, among others, the risks inherent in drug development such as those associated with the initiation, cost, timing, progress and results of the Company’s current and future research and development programs, preclinical and clinical trials. For a detailed discussion of the risk factors that could affect the Company’s actual results, please refer to the risk factors identified in the Company’s SEC reports, including but not limited to its Quarterly Report on Form 10-Q dated November 8, 2023. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements for any reason. Investor Relations & Media:Nicole Keithinvestor.relations@sana.commedia@sana.com

Problems with cell therapy include limited durability of effect and life-threatening safety risks. Cargo Therapeutics aims to address many limitations of currently available cell therapies, but its lead program is focused on overcoming one: the resistance cancers can develop. A potentially pivotal study is underway and Cargo has raised $281.3 million in IPO cash to support the clinical research. Late Thursday, Cargo priced its offering of 18.75 million shares at $15 apiece, which was the low end of the $15 to $17 price range it had planned. Those shares will trade on the Nasdaq under the stock symbol “CRGX.” The first generation of cell therapies are made by engineering a patient’s T cells to go after a cancer protein called CD19. Cancers can develop resistance by losing this protein, rendering a CD19-targeting therapy ineffective. In the IPO filing, Cargo points to clinical tests of the Gilead Sciences CAR T-cell therapy Yescarta in patients with large B-cell lymphoma (LBCL). Results showed that about 60% of treated patients experienced relapse or disease progression after 24 months. Yescarta was approved in 2017 as a third-line LBCL treatment. Last year, the FDA approved the therapy for second-line use. “As CD19 CAR T-cell therapies continue to expand into earlier lines of therapy and additional geographies, there is a large growing unmet need for the majority of patients who do not experience a durable response,” Cargo said in the IPO filing. Lead Cargo program CRG-022 is an autologous cell therapy engineered to target a different cancer protein, CD22. In Phase 1 testing in patients with advanced cases of LBCL, Cargo reported 53% (20 of 38 patients) achieved a complete response to the therapy as of the most recent data cutoff date in May. In August, Cargo began a Phase 2 study testing its cell therapy in LBCL patients whose disease has relapsed or has not responded to a CD19-targeting cell therapy. Cargo expects interim results from this study in 2025, according to the IPO filing. Cargo is based in San Mateo, California, but its origins are on the other side of the country. Company co-founder Crystal Mackall developed a CD22-targeting chimeric antigen receptor (CAR) during her tenure at the National Cancer Institute. In 2016, she took on positions at Stanford University. Cargo formed in 2019, based on Mackall’s CAR, which was licensed from the NCI. The company raised seed financing in 2021. Its $200 million Series A financing came together early this year, co-led by Third Rock Ventures, RTW Investments, and Perceptive Advisors. Cargo’s aspirations to address multiple limitations of cell therapy is reflected by its name. After the company closed its Series A financing, CEO Gina Chapman told MedCity News that Cargo’s research includes developing cells engineered to carry therapeutic cargo that endow them with new properties. A second platform technology was licensed from Stanfard. Called STASH, this technology enables the engineering of various types of immune cells. “This platform allows us to incorporate multiple transgene therapeutic ‘cargo’ designed to enhance CAR T-cell persistence and trafficking to tumor lesions, as well as to help safeguard against tumor resistance and T cell exhaustion,” Cargo said in the IPO filing. But Cargo’s near-term focus is its CD22-targeting cell therapy. At the end of June, Cargo reported a cash position of $42.4 million. Together with the IPO proceeds, the company said in the filing that it plans to spend $220 million for the Phase 2 test of this lead cancer cell therapy program. Another $20 million is budgeted for the internal R&D of other therapeutic candidates. The most advanced preclinical Cargo program, CRG-023, engineers a T cell to hit three cancer proteins: CD19, CD20, and CD22. Cargo also said in the filing it may apply some of the IPO proceeds toward business development, such as acquisitions or licensing deals. With the completion of the IPO, the company expects it will have enough capital to last through 2025.

Cargo Therapeutics priced an initial stock offering on Thursday, bringing in $281 million to fund the biotechnology startups plans for developing better cancer drugs.The San Mateo, California-based company priced 18.75 million shares at $15 apiece, on the low end of its proposed price range. Its IPO was the fourth largest this year for a biotech, according to BioPharma Dive data.Cargos focus is on CAR-T therapies, a type of cellular treatment for several kinds of blood cancers. While some patients benefit greatly from CAR-T therapy companies like Gilead have recently received expanded clearances for their CAR-T drugs based on strong data not all do. Others relapse months after treatment.Cargos lead program, dubbed CRG-022, is in Phase 2 clinical testing for patients with large B-cell lymphoma that is relapsed or refractory to existing CAR-T therapies. While CAR-T therapies approved by the Food and Drug Administration for lymphoma target a protein called CD19, CRG-022 is aimed at a different protein, CD22. Cargo claims its treatment can treat cancers that don't respond to or have evaded CD19-directed treatment.CRG-022 is also being tested in patients who have not had other CAR-T therapies as well as children with B-cell acute lymphoblastic leukemia. A second program, CRG-023, is in preclinical studies for B-cell malignancies.Cargo announced it had raised a $200 million Series A round in March, backed by notable biotech investors such as Samsara BioCapital and Third Rock Ventures.Given Cargos progress to date and its experienced leadership team, Cargo is well-positioned to be first-to-market with an autologous CD22 CAR T-cell therapy, Reid Huber, a partner at Third Rock Ventures, said at the time.That projection hinges on the biotechs ability to fund its clinical trial plans. In a securities filing ahead of its public offering, Cargo wrote that its cash on hand and proceeds from two tranches of stock issued in March and October will not be sufficient to fund our operations for at least one year from the issuance date of our financial statements.However, the proceeds from the IPO should stretch its financial runway into 2025, the company said in the document.Cargo is now one of seven cancer-focused drugmakers and the 19th biotech overall to price a new stock offering in 2023, according to BioPharma Dive data.At $281M, Cargos offering is also the second-largest by a cancer drugmaker in 2023, behind RayzeBios $311 million IPO in September. It matches the profile of biotechs that have successfully pulled off IPOs this year, when investors looking for surer bets have gravitated toward companies with clinical data. Even then, newly public companies have struggled once on the market this year, with most now trading below their IPO price.Earlier this week, gene therapy biotech Lexeo Therapeutics priced an offering that came below its expected share price, raising $100 million for experimental cardiovascular and neurodegenerative disease drugs. '