Gliomagenesis is orchestrated by the Oct3/4 regulatory network. – Physician’s Weekly

Glioblastoma multiforme (GBM) is a lethal brain tumor characterized by developmental hierarchical phenotypic heterogeneity, therapy resistance and recurrent growth. Neural stem cells (NSCs) from human central nervous system (CNS), and glioblastoma stem cells from patient-derived GBM (pdGSC) samples and cultured in both 2D well-plate and 3D monoclonal neurosphere culture system (pdMNCS). The pdMNCS model shows promise to establish a relevant 3D-tumor environment that maintains GBM cells in the stem cell phase within suspended neurospheres. Utilizing the pdMNCS, we examined GBM cell-lines for a wide spectrum of developmental cancer stem cell markers, including the early blastocyst inner-cell mass (ICM)-specific Nanog, Oct3/4,B, and CD133. We observed that MNCS epigenotype is recapitulated using gliomasphere-derived cells. CD133, the marker of GSC is robustly expressed in 3D-gliomaspheres and localized within the plasma membrane compartment. Conversely, gliomasphere cultures grown in conventional 2D culture quickly lost CD133 expression, indicating its variable expression is dependent on cell-culture conditions. Critically, this experiment demonstrates incomplete differentiation of cytoskeleton microtubules and intermediate filaments (IFs) of patient derived cells, similar to commercially available GBM cell lines. Subsequently, in order to determine whether Oct3/4 it was necessary for CD133 expression and cancer stemness, we transfected 2D and 3D culture with siRNA against Oct3/4 and found a significant reduction in gliomasphere formation. These results suggest that expression of Oct3/4,Aand CD133 suppress differentiation of GSCs.

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Gliomagenesis is orchestrated by the Oct3/4 regulatory network. - Physician's Weekly

Fate Therapeutics Reports Second Quarter 2021 Financial Results and Highlights Operational Progress – StreetInsider.com

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First Patient Treated for Relapsed / Refractory ALL in Landmark Phase 1 Clinical Trial of FT819, the First-ever iPSC-derived CAR T-cell Therapy; Off-the-Shelf Product Candidate Derived from Clonal Master iPSC Line with Novel CD19-specific 1XX CAR Integrated into TRAC Locus

FT516 Interim Phase 1 Data for Relapsed / Refractory Lymphoma Featured at ASCO; 8 of 11 Patients in Dose Cohorts 2 and 3 Achieved Objective Response, including 6 Patients with Complete Response

Interim Phase 1 Data from FT516 and FT538 Programs for Relapsed / Refractory AML Highlighted at May Investor Event; 5 of 12 Patients Achieved Objective Response with Complete Clearance of Bone Marrow Leukemic Blasts

New Phase 1 Clinical Data from FT516 and FT596 Programs in Relapsed / Refractory Lymphoma to be Featured at Investor Event on August 19

SAN DIEGO, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, today reported business highlights and financial results for the second quarter ended June 30, 2021.

We are very pleased with the early clinical safety and activity we have observed with our off-the-shelf, iPSC-derived NK cell programs in relapsed / refractory lymphoma and acute myeloid leukemia, where interim Phase 1 data indicate FT516 and FT538 are well tolerated and can deliver complete responses for patients. We look forward to sharing additional clinical data from our FT516 and FT596 programs in B-cell lymphoma at our upcoming investor event, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. Additionally, treatment of the first patient with FT819, the first-ever iPSC-derived T-cell therapy to undergo clinical investigation, is a landmark achievement and further demonstrates the Companys leadership in off-the-shelf, iPSC-derived cell therapy and the versatility of its proprietary iPSC Product Platform.

B-cell Malignancy Disease Franchise

AML Disease Franchise

Multiple Myeloma Franchise

Solid Tumor Franchise

Other Corporate Highlights

Second Quarter 2021 Financial Results

Today's Conference Call and WebcastThe Company will conduct a conference call today, Wednesday, August 4, 2021 at 5:00 p.m. ET to review financial and operating results for the quarter ended June 30, 2021. In order to participate in the conference call, please dial please dial 800-773-2954 (toll free) or 847-413-3731 (toll) and refer to conference ID 50196101. The live webcast can be accessed under "Events & Presentations" in the Investors section of the Company's website at http://www.fatetherapeutics.com. The archived webcast will be available on the Company's website beginning approximately two hours after the event.

About Fate Therapeutics iPSC Product Platform The Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT516 FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in a multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in a multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About FT596 FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and hnCD16 targeting receptors enhances cytotoxic activity, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a humanized mouse model of lymphoma, FT596 in combination with the anti-CD20 monoclonal antibody rituximab showed enhanced killing of tumor cells in vivo as compared to rituximab alone. FT596 is being investigated in a multi-center Phase 1 clinical trial for the treatment of relapsed / refractory B-cell lymphoma as a monotherapy and in combination with rituximab, and for the treatment of relapsed / refractory chronic lymphocytic leukemia (CLL) as a monotherapy and in combination with obinutuzumab (NCT04245722).

About FT538 FT538 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three functional components: a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; an IL-15 receptor fusion (IL-15RF) that augments NK cell activity; and the deletion of the CD38 gene (CD38KO), which promotes persistence and function in high oxidative stress environments. FT538 is designed to enhance innate immunity in cancer patients, where endogenous NK cells are typically diminished in both number and function due to prior treatment regimens and tumor suppressive mechanisms. In preclinical studies, FT538 has shown superior NK cell effector function, as compared to peripheral blood NK cells, with the potential to confer significant anti-tumor activity to patients through multiple mechanisms of action. FT538 is being investigated in a multi-dose Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and in combination with daratumumab, a CD38-targeted monoclonal antibody therapy, for the treatment of multiple myeloma (NCT04614636).

About FT819 FT819 is an investigational, universal, off-the-shelf, T-cell receptor (TCR)-less CD19 chimeric antigen receptor (CAR) T-cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line, which is engineered with the following features designed to improve the safety and efficacy of CAR19 T-cell therapy: a novel 1XX CAR signaling domain, which has been shown to extend T-cell effector function without eliciting exhaustion; integration of the CAR19 transgene directly into the T-cell receptor alpha constant (TRAC) locus, which has been shown to promote uniform CAR19 expression and enhanced T-cell potency; and complete bi-allelic disruption of TCR expression for the prevention of graft-versus-host disease (GvHD). FT819 demonstrated antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines comparable to that of primary CAR T cells, and persisted and maintained tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia (Valamehr et al. 2020). FT819 is being investigated in a multi-center Phase 1 clinical trial for the treatment of relapsed / refractory B-cell malignancies, including B-cell lymphoma, chronic lymphocytic leukemia, and acute lymphoblastic leukemia (NCT04629729).

About Fate Therapeutics, Inc. Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs). Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Companys results of operations, financial condition and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to its product candidates, clinical studies and preclinical research and development programs, the Companys progress, plans and timelines for the manufacture and clinical investigation of its product candidates, the timing for the Companys receipt of data from its clinical trials and preclinical studies, the initiation of additional clinical trials and additional dose cohorts in ongoing clinical trials of the Companys product candidates and the submission of IND applications for additional programs, the Companys development and regulatory strategy, the therapeutic and market potential of the Companys product candidates, and the parties rights and obligations under the Companys collaboration agreements. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Companys product candidates may not demonstrate the requisite safety or efficacy to achieve regulatory approval or to warrant further development, the risk that results observed in prior studies of the Companys product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Companys product candidates or in the initiation of, or enrollment of patients in, any clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, the amount and type of data to be generated, or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Companys ongoing and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), risks related to the impact of the COVID-19 pandemic on various aspects of the Companys business and operations, including its ability to initiate, conduct and complete its clinical trials, and the risk that the Companys expenditures may exceed current expectations for a variety of reasons. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Availability of Other Information about Fate Therapeutics, Inc. Investors and others should note that the Company routinely communicates with investors and the public using its website (www.fatetherapeutics.com) and its investor relations website (ir.fatetherapeutics.com) including, without limitation, through the posting of investor presentations, SEC filings, press releases, public conference calls and webcasts on these websites. The information posted on these websites could be deemed to be material information. As a result, investors, the media, and others interested in Fate Therapeutics are encouraged to review this information on a regular basis. The contents of the Companys website, or any other website that may be accessed from the Companys website, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (unaudited)

Condensed Consolidated Balance Sheets (in thousands) (unaudited)

Contact: Christina Tartaglia Stern Investor Relations, Inc. 212.362.1200 christina@sternir.com

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Fate Therapeutics Reports Second Quarter 2021 Financial Results and Highlights Operational Progress - StreetInsider.com

Improving the Treatment Gap in Diffuse Large B-Cell Lymphoma – Targeted Oncology

Gilles Salles, MD, discusses the need for new therapies to treat diffuse large B-cell lymphoma.

Gilles Salles, MD, the lymphoma service chief at Memorial Sloan Kettering Cancer Center, discusses the need for new therapies to treat diffuse large B-cell lymphoma (DLBCL).

According to Salles, once patients fail primary therapy, limited options remain. For approximately 50% of the patient population, those with limited comorbidities or those under a certain age, the standard of care is salvage chemotherapy followed by stem cell transplant.

However, for the approximately half of patients who are not eligible for this route, the treatment is usually immuno-chemotherapy, according to Salles. For most regimens, the response rate is limited. For patients who do respond, the duration of response (DOR) is typically only between 4 to 6 months. The median survival after the second-line therapy is about a year.

News agents have made progress in this space. For example, chimeric antigen receptor T cells show promise, but eligibility and access remain a major hurdle for many patients. New agents are also in the pipeline, though according to Salles, many have a short DOR.

Excerpt from:
Improving the Treatment Gap in Diffuse Large B-Cell Lymphoma - Targeted Oncology

MUHS, Nashik to conduct fellowship course in regenerative medicine – BSI bureau

Maharashtra University Of Health Sciences (MUHS), Nashik has been granted first affiliations to conduct a fellowship course in regenerative medicine and stem cell-based therapies on a research basis at Dr Mahajans Hospital & Stem Rx Bioscience Solution, Navi Mumbai. The move will address the education and training gaps in this new area of regenerative medicine.

MD/MS/M.Ch,/DM/DNB qualified surgeons/physicians/super specialists from allopathy, undergraduate or postgraduate degrees equivalent to and recognised by the Medical Council of India are eligible for admission.

Candidates should have six months of basic molecular biological background in basic research. The basic research in molecular biology of human body science is the first time in India to give research background to qualified physicians and surgeons to practice medicine.

The two-year fellowship programme will have FDA regulations, ethics, emerging technologies in stem cell, pre-clinical studies, mesenchymal stem cell isolation and transplantation, etc

Dr Pradeep Mahajan, Regenerative Medicine Researcher, StemRx Bioscience Solutions, Navi Mumbai said, The fellowship course has been designed to ensure that qualified individuals get the required theoretical training as well as practical exposure to learn the intricacies of research in the field of RM. Candidates will be able to pursue their research in this field and contribute to the ever-increasing need for novel therapeutic technologies. It would improve the practice of regenerative and cellular medicine and hopefully obviate the need for regulatory action in some cases.

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MUHS, Nashik to conduct fellowship course in regenerative medicine - BSI bureau

FDA Hands Out Surprising Rejection of Meduxus and Medac’s Treosulfan – BioSpace

JHVEPhoto/Shutterstock

Medexus Pharmaceuticals and medacGmbHreceiveda Complete Response Letter (CRL) from the Food and Drug Administration (FDA) for its New Drug Application (NDA) for treosulfan. The drug was being submitted for use in combination with fludarabine as a preparation for allogeneic hematopoietic stem cell transplantation (allo-HSCT). The companies had a target action date of August 11.

A preparative regimen is given to the patient before stem cell transplantation with the goal of decreasing the tumor burden. And in the case of allogeneic transplantation, it allows for engraftment of the donor cells.

The CRL indicated that it couldn't be approved in its present form. It offered specific recommendations, including additional clinical and statistical data related to the primary and secondary endpoints of the pivotal Phase III trial. The companies say they are reviewing the CRL to decide on a course of action.

Ken d'Entremont, chief executive officer of Medexus, said, "Given the recent Health Canada approval, European Medicines Agency approval in 2019, as well as supporting data from more than 100 publications, we were all surprised by the FDA's response."

"That being said, Medexus and medac look forward to continuing to work with the FDA to address their requests in a timely manner, and we remain optimistic for a future, albeit delayed, approval of treosulfan in the United States, complete with Orphan Drug Designation."

He went on to say that the current standard of care "is not suitable for numerous at-risk groups, due to the high toxicity effects, and treosulfan has demonstrated excellent survival data among those groups."

Medexus, headquartered in Toronto, Ontario, Canada, and medac, based in Wedel, Germany,inkeda licensing deal on July 12 to commercialize treosulfan in Canada. It is being marketed in Canada under the name Trecondyv. Medexus will handle sales and marketing, while medac will handle manufacturing and supply.

The therapy has been distributed in Canada under the Special Access Program and recently received approval for commercialization from Health Canada for adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS) who are at increased risk for standard conditioning therapies. It was also approved for children older than one-year-old with AML or MDS.

At the time, Magnus Kuster, vice president of International Sales & Regions for medac, noted, "The treosulfan-based conditioning regimen stands out for its combination of being highly effective similar to the potency of myeloablative procedures while simultaneously exhibiting significantly reduced toxicity. We at medac are very proud of our first-in-class conditioning agent as it fully meets our company's goals of improving patients' lives and supporting healthcare professionals in the best possible way."

In response to the CRL, Meduxus's general manager of U.S. Operations, Michael Adelman, said, "We are disappointed with the immediate result, but are encouraged by an incredible amount of support from key opinion leaders and the medical community for use of treosulfan in the United States. With the extensive launch preparations we have taken to date, we are well positioned to meet the expected strong demand for treosulfan."

They will work with the FDA to address the issues in the CRL while ready to trigger their U.S. marketing plan when it gets approved.

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FDA Hands Out Surprising Rejection of Meduxus and Medac's Treosulfan - BioSpace

Dr. Flowers on Efforts to Improve Frontline Treatment in DLBCL – OncLive

Christopher Flowers, MD, discusses efforts to improve the frontline standard of care in diffuse large B-cell lymphoma.

Christopher Flowers, MD,department chair, Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses efforts to improve the frontline standard of care in diffuse large B-cell lymphoma (DLBCL).

The frontline standard of care therapy has not changed significantly over the past 20 years in DLBCL, Flowers says. In the early 2000s, it was observed that the addition of rituximab to standard CHOP (R-CHOP) therapy improved survival for elderly patients with DLBCL compared with CHOP alone, Flowers explains.

Although several research efforts have attempted to improve upon frontline R-CHOP, no survival advantages have been demonstrated, Flowers says. For example, randomized phase 3 trials evaluating intensified treatment approaches, stem cell transplantation, and the addition of novel agents, including bortezomib (Velcade), lenalidomide (Revlimid), and ibrutinib (Imbruvica), have been reported without demonstrating improvement vs R-CHOP in most patients with DLBCL, Flowers concludes.

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Dr. Flowers on Efforts to Improve Frontline Treatment in DLBCL - OncLive

Future Directions in the Treatment of Polycythemia Vera – OncLive

A thought leader in hematologic malignancies provides key insights into the future of treatment for polycythemia vera by considering the potential role of treatment sequencing or combination therapy.

EP. 1 : Myeloproliferative Neoplasms: Diagnosing Polycythemia Vera

EP. 2 : Risk Stratification and Treatment Considerations in PV

EP. 3 : PV Treatment Options and Dosing

EP. 4 : The JAK2 Pathway in Polycythemia Vera

EP. 5 : Ruxolitinib in PV: The RESPONSE Trial

EP. 6 : Ruxolitinib in PV: The RESPONSE-2 Trial

EP. 7 : Future Directions in the Treatment of Polycythemia Vera

Jamile Shammo, MD, FASCP, FACP: We now have optionsfor patients who have polycythemiavera [PV]. I know that other options may be available in the future for patients who have this entity. I think that its a good thing that we should be able to offer our patients multiple options.Clearly, patients whohavehad issues with enlarged spleen arent able to tolerate hydroxyurea, and people who had heavy burden with phlebotomy may not be able to tolerate phlebotomy,soruxolitinib presents a very good option for the treatment of their disease.For patients who are interested in having children in a few months, thismay represent a good option for them to achieve that goal.For patients who dont have that possibility or who had failedall ofthe abovetreatments, we will have clinical trials so that we can offer them novel agents that would have the potential for controlling their disease moving forward.

It would be important to see what the clinical trials would bring. I would think that combination therapies may be most helpful in more advanced disease entities like myelofibrosis where you can justify the risk of combination therapy because its a much more aggressive disease than it would be the case with PV.Perhaps you could consider that in advanced PV, or maybe you could consider this in patients with PV and multiple mutations.Ithas tobe considered in a certain group of patients. I dont know that I would necessarily consider combination therapy in all-comers with PV. Ithas tobe thought of in a rational fashion.

We need to understand this group of patients better on a molecular level.We know a lot about the landscape of their molecular profiles, but we know less about how to treat that. We know less about the landscape of people who relapseafter a particular treatmentand how to address thatparticular treatment, for example. We probably need to know more about how to utilize transplant in that setting.Theres a lot to be learned about how to use this.

Of course, there is also awhole slew of JAK[Janus kinase]inhibitors that may have various properties as well. It isinteresting to think about utilizing them pre-and post-transplant. Thereare a whole slew of biological agents that could be combined with JAK inhibitors to see how we can make the most out of that combination. We can learnhowthey canbe explored either alone or in combination with stem cell transplantation.Theres a lot that needs to be done, but all thathas tobe done in the context of a clinical trial.It probably starts with understanding more about the disease biology,taking it to some type of a clinical trial, andkeeping our eyes opentoget an observation to translate that into some type of conclusion that may be beneficial to patients.

TRANSCRIPT EDITED FOR CLARITY

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Future Directions in the Treatment of Polycythemia Vera - OncLive

FDA Grants Priority Review to Genentech’s Tecentriq as Adjuvant Treatment for Certain People With Early Non-small Cell Lung Cancer – Business Wire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has accepted the companys supplemental Biologics License Application (sBLA) and granted Priority Review for Tecentriq (atezolizumab) as adjuvant treatment following surgery and platinum-based chemotherapy for people with non-small cell lung cancer (NSCLC) whose tumors express PD-L11%, as determined by an FDA-approved test. The FDA is reviewing the application under the Real-Time Oncology Review pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. The FDA is expected to make a decision on approval by December 1, 2021.

New treatment options are urgently needed in early-stage non-small cell lung cancer to help the nearly 50% of people who currently experience a recurrence following surgery, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Tecentriq is the first cancer immunotherapy to show a clinically meaningful benefit in the adjuvant lung cancer setting, and were working closely with the FDA to bring this significant advancement to patients as quickly as possible.

This application is based on disease-free survival (DFS) results from an interim analysis of the Phase III IMpower010 study, the first and only Phase III study of a cancer immunotherapy to demonstrate positive results in the adjuvant lung cancer setting. The study showed that treatment with Tecentriq following surgery and platinum-based chemotherapy reduced the risk of disease recurrence or death (DFS) by 34% (hazard ratio [HR]=0.66, 95% CI: 0.50-0.88) in people with Stage II-IIIA NSCLC whose tumors express PD-L11%, compared with best supportive care (BSC). In this population, median DFS was not yet reached for Tecentriq compared with 35.3 months for BSC. Follow-up on the IMpower010 trial will continue with planned analyses of DFS in the overall intent-to-treat (ITT) population, including Stage IB patients, which at the time of analysis did not cross the threshold, and overall survival (OS) data, which were immature at the time of interim analysis. Safety data for Tecentriq were consistent with its known safety profile and no new safety signals were identified. Results from the IMpower010 trial were presented at the 2021 ASCO Annual Meeting.

About the IMpower010 study

IMpower010 is a Phase III, global, multicenter, open-label, randomized study evaluating the efficacy and safety of Tecentriq compared with BSC, in participants with Stage IB-IIIA NSCLC (UICC 7th edition), following surgical resection and up to 4 cycles of adjuvant cisplatin-based chemotherapy. The study randomized 1,005 people with a ratio of 1:1 to receive either Tecentriq (up to 16 cycles) or BSC. The primary endpoint is investigator-determined DFS in the PD-L1-positive Stage II-IIIA, all randomized Stage II-IIIA and ITT Stage IB-IIIA populations. Key secondary endpoints include OS in the overall study population, ITT Stage IB-IIIA NSCLC.

About lung cancer

According to the American Cancer Society, it is estimated that more than 235,000 Americans will be diagnosed with lung cancer in 2021, and NSCLC accounts for 80-85% of all lung cancers. Today, about half of all people with early lung cancer still experience a cancer recurrence following surgery, but treating lung cancer early, before it has spread, may help prevent the disease from returning and provide people with the best opportunity for a cure.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of lung cancer called non-small cell lung cancer (NSCLC).

A type of lung cancer called small cell lung cancer (SCLC).

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues in any area of the body and can affect the way they work. These problems can sometimes become severe or life threatening and can lead to death. Patients can have more than one of these problems at the same time. These problems may happen anytime during their treatment or even after their treatment has ended.

Patients should call or see their healthcare provider right away if they develop any new or worse signs or symptoms, including:

Lung problems

Intestinal problems

Liver problems

Hormone gland problems

Kidney problems

Skin problems

Problems can also happen in other organs.

These are not all of the signs and symptoms of immune system problems that can happen with Tecentriq. Patients should call or see their healthcare provider right away for any new or worse signs or symptoms, including:

Infusion reactions that can sometimes be severe or life-threatening. Signs and symptoms of infusion reactions may include:

Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if patients undergo transplantation either before or after being treated with Tecentriq. A healthcare provider will monitor for these complications.

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider will check patients for these problems during their treatment with Tecentriq. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may also need to delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information about the benefits and side effects of Tecentriq.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

Please see http://www.Tecentriq.com for full Prescribing Information and additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentechs approved PD-L1 checkpoint inhibitor, the companys broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have five approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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FDA Grants Priority Review to Genentech's Tecentriq as Adjuvant Treatment for Certain People With Early Non-small Cell Lung Cancer - Business Wire

Cellino Appoints Industry Pioneer Robert J. Palay to the Board – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Cellino, a personalized regenerative medicine company developing an AI-guided laser editing platform for autologous cell-based therapies, today announced the appointment of industry pioneer Robert J. Palay, J.D., M.B.A., to the Cellino Board of Directors.

Mr. Palay is one of the pioneers of the induced pluripotent stem cell (iPSC) industry. He was Founder, Chairman of the Board, and Chief Executive Officer of Cellular Dynamic International (NASDAQ: ICEL), one of the first companies to harness IPSCs and their derivatives for use in drug discovery and therapeutics. CDI was acquired by FujiFilm for $307M in 2015. Prior to Cellular Dynamic International, he was a co-founder and Chairman of the Board of NimbleGen Systems, a early adopter of light directed synthesis for manufacturing genomic micro-arrays. NGS was acquired by Roche in 2007 for $272.5M. He was previously a Fellow at Harvards Advanced Leadership Initiative where he focused on emerging biothreats.

"We are pleased to welcome Robert to the Cellino Board," said Nabiha Saklayen, Ph.D, CEO & Co-Founder, Cellino. "Roberts deep experience in pioneering the light-directed synthesis and iPSC industries will be invaluable to Cellino as we grow our business at that unique interface and pursue our mission to serve patients."

"I am thrilled to join the Cellino Board," said Robert J. Palay. "Cellinos platform is the future of the stem cell industry. They have the unique ability to bring laser precision and artificial intelligence to the manufacture of IPSCs and their derivative cells. Their innovation offers a clear path to dramatic improvements in the quality and quantity of manufactured human cells, while simultaneously dramatically reducing cost. No one else has technology offering such a robust and powerful platform."

Mr. Palay was also a co-founder and Chairman of the Board of Genetic Assemblies, a synthetic biology company, which was acquired by Codon Devices in 2006. Mr. Palay is currently Chairman of Tactics II Equity LLC, a life science investment and advisory firm. He is currently a member of the board of directors of the Center for Strategic Risks and a founder of the Alliance to End Biological Threats. Mr. Palay received a J.D. and M.B.A. from Northwestern University, and an A.B. from Harvard College.

About Cellino

Cellino is an autologous regenerative medicine company developing an AI-guided single-cell laser editing platform for cell-based therapies. The company has developed a scalable platform that automates and standardizes autologous stem cell production, accelerating the development of life-saving medicines for patients. Cellino is based in Cambridge, MA. For additional information, please visit http://www.cellinobio.com.

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Cellino Appoints Industry Pioneer Robert J. Palay to the Board - Business Wire

Tachyon and AbCellera Collaborate to Develop Novel Antibody Therapeutic Targeting TGF- Superfamily Member for the Treatment of Cancer – Yahoo Finance

LEFTY1, a secreted dual inhibitor of the NODAL/SMAD2 and BMP7/SMAD5 pathway, is implicated in driving cancer stem cell biology and unregulated tumor cell growth

HOUSTON & VANCOUVER, British Columbia, August 03, 2021--(BUSINESS WIRE)--Tachyon Therapeutics, Inc. (Tachyon), a research and development focused biotechnology company, and AbCellera (Nasdaq: ABCL), a technology company with a centralized operating system for next-generation antibody discovery, today announced a collaboration to facilitate the discovery and development of a therapeutic antibody targeting LEFTY1, a member of the transforming growth factor (TGF-) superfamily and validated extracellular drug target expressed in advanced cancers. Under the terms of the agreement, AbCellera is eligible to receive milestone payments and royalties on products that are derived from its antibody discovery platform. In addition, AbCellera has the option to invest in preclinical and clinical development in exchange for an increased share of product sales.

"At Tachyon, we are focused on innovation, precision, science, and speed to develop first-in-class therapeutics against significant new drug targets in cancer biology," said Frank Perabo, M.D., Ph.D., Chief Executive Officer of Tachyon. "AbCellera has consistently demonstrated these four qualities as they accelerate the discovery of first-in-class therapeutic antibodies for novel biology. We look forward to working with their team to target LEFTY1, a major signaling regulator of the TGF- superfamily, and unlock a new pathway to treat advanced cancers."

Members of the TGF- superfamily control numerous cellular functions, including proliferation, apoptosis, differentiation, epithelial-mesenchymal transition, adhesion, and migration. Because of their ubiquitous and regulatory roles in both normal and cancer cell biology, TGF- superfamily members, such as LEFTY1 are highly sought after, yet challenging drug targets. Research led by Tachyons scientific founder, Michael F. Clarke, Ph.D., who was the first to identify and characterize cancer stem cells in solid tumors, revealed that LEFTY1 suppresses NODAL/SMAD2 and BMP7/SMAD5 pathways to promote long-term proliferation of normal and malignant mammary epithelial cells.1 As a secreted extracellular protein, LEFTY1 represents an important target to control SMAD-dependent signals that promote the long-term growth and self-renewal of cancer stem cells.

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"Our collaboration with Tachyon represents an opportunity to expedite the translation of breakthrough research of a novel and significant cancer drug target into a first-in-class antibody therapeutic for patients with advanced cancers and limited treatment options," said Carl Hansen, Ph.D., CEO of AbCellera. "This collaboration exemplifies innovative deal structures that align our interest with our partners and provides AbCellera with optionality to deepen our participation in the success of the antibodies we discover. We are excited to partner with Tachyon to pursue a previously unexplored mechanism and develop therapies that we anticipate will impact cancer treatment."

1. Zabala, et al., LEFTY1 Is a Dual-SMAD Inhibitor that Promotes Mammary Progenitor Growth and Tumorigenesis. Cell Stem Cell. 2020.

About Tachyon Therapeutics Inc.

Tachyon Therapeutics, Inc. is a R&D focused biotechnology company developing novel, first-in-class therapeutics for the treatment of advanced cancers. Tachyon operates with a dedicated internal core development team and a virtual external network of expertise to achieve one goal advance development of promising drug targets with speed and innovation, without compromising the quality or integrity of science. For more information, please visit http://www.tachyontx.com.

About AbCellera Biologics Inc.

AbCellera is a technology company that searches, decodes, and analyzes natural immune systems to find antibodies that its partners can develop into drugs to prevent and treat disease. AbCellera partners with drug developers of all sizes, from large pharmaceutical to small biotechnology companies, empowering them to move quickly, reduce cost, and tackle the toughest problems in drug development. For more information, please visit http://www.abcellera.com.

AbCellera Forward-looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The forward-looking statements are based on managements beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including statements regarding our ability to develop, commercialize and achieve market acceptance of our current and planned products and services, our research and development efforts, and other matters regarding our business strategies, use of capital, results of operations and financial position, and plans and objectives for future operations.

In some cases, you can identify forward-looking statements by the words "may," "will," "could," "would," "should," "expect," "intend," "plan," "anticipate," "believe," "estimate," "predict," "project," "potential," "continue," "ongoing" or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors are described under "Risk Factors," "Management's Discussion and Analysis of Financial Condition and Results of Operations" and elsewhere in the documents we file with the Securities and Exchange Commission from time to time. We caution you that forward-looking statements are based on a combination of facts and factors currently known by us and our projections of the future, about which we cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent our views as of the date hereof. We undertake no obligation to update any forward-looking statements for any reason, except as required by law.

Source: AbCellera Biologics Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210803005352/en/

Contacts

Tachyon Inquiries Frank Perabo, M.D., Ph.D., Chief Executive Officer; ir@tachyontx.com, +1 (832) 952-0829

AbCellera Inquiries Media: Jessica Yingling, Ph.D., media@abcellera.com, +1 (236) 521-6774 Business Development: Neil Berkley, bd@abcellera.com, +1 (604) 559-9005 Investor Relations: Melanie Solomon, ir@abcellera.com, +1 (778) 729-9116

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Tachyon and AbCellera Collaborate to Develop Novel Antibody Therapeutic Targeting TGF- Superfamily Member for the Treatment of Cancer - Yahoo Finance