Day: December 5, 2020

Cellectis’ Proprietary Program UCART22 was Safely Administered in BALLI-01 Phase 1 Study with No Dose-Limiting Toxicity or Evidence of Graft-Vs-Host Disease2 out of 3 Patients at DL1 Achieved CR/CRi and 1 out of 2 Patients at DL2 Achieved a Significant Reduction in Bone Marrow BlastsBALLI-01 Currently Enrolling at DL2 with Addition of Alemtuzumab to the FC Lymphodepletion Regimen; Next Data Update Expected in 2021NEW YORK, Dec. 05, 2020 (GLOBE NEWSWIRE) — Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), announced preliminary results from Cellectis’ dose escalation Phase 1 BALLI-01 study of UCART22 product candidate in relapsed/refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) were presented...

Le produit candidat UCART22 de Cellectis a été administré en toute sécurité dans l’étude de Phase I BALLI-01 sans manifestation de toxicité limitant la dose, ni de maladie du greffon contre l’hôte2 patients sur 3 au palier de dose 1 ont atteint une rémission complète avec une récupération hématologique incomplète, et 1 patient sur 2 au palier de dose 2 a atteint une réduction significative des blastes dans la moelle osseuseInclusion de patients en cours dans l’essai clinique BALLI-01 au palier de dose 2 incorporant l’alemtuzumab au régime de lymphodéplétion composé de fludarabine et cyclophosphamide ; prochaine publication des données clinique prévue en 2021NEW YORK, 05 déc. 2020 (GLOBE NEWSWIRE) — Cellectis (Euronext Growth : ALCLS – Nasdaq : CLLS), une société biopharmaceutique spécialisée dans le développement...

Updated data from the ALLCAR study suggests AUTO1’s potential for transformational activity in adult patients with r/r ALLConference call and webcast to be held Monday, December 7, 2020 at 4:00 pm ET / 9:00 pm GMTLONDON, Dec. 05, 2020 (GLOBE NEWSWIRE) — Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced new data highlighting progress on its AUTO1 program, the company’s CAR T cell therapy being investigated in the ongoing ALLCAR Phase 1 study in relapsed / refractory adult B-Acute Lymphocytic Leukemia (ALL), during the American Society of Hematology (ASH) All-Virtual Annual Meeting, held between December 5-8, 2020.As of the November 12, 2020 data cut-off date, 20 patients with r/r ALL had received AUTO1. AUTO1 was well tolerated,...

– 9 of 14 Patients Showed Reduction in Tumor Size, Including Two Recently Reported Complete Responses –– Company to Host Conference Call and Webcast Today at 2:00 p.m. ET –MOUNTAIN VIEW, Calif., Dec. 05, 2020 (GLOBE NEWSWIRE) — IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, today announced the presentation of preliminary clinical results from the Company’s Phase 1 trial evaluating IGM-2323, a bispecific IgM antibody targeting CD20 x CD3, at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The data was featured today in a poster presentation titled “Preliminary Results of a Phase 1 Dose Escalation Study of the First-in-Class IgM Based Bispecific Antibody IGM-2323 (anti-CD20 x anti-CD3) in...

Results presented at Foundation for Angelman Syndrome Therapeutics (FAST) Global SummitAdditional data, including EEG findings, support prior initial indications of activity and there were no new adverse eventsSARASOTA, Fla. and NOVATO, Calif., Dec. 05, 2020 (GLOBE NEWSWIRE) — GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced the presentation of data from the Phase 1/2 study of GTX-102 at the Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit. Details regarding the scientific basis for GTX-102 targeting in Angelman syndrome were presented along with additional supportive clinical data on EEG and other endpoints, along with further description of...

Long-term follow-up data from the phase III MURANO trial showed sustained progression-free survival with fixed-duration Venclexta/Venclyxto plus MabThera/RituxanMURANO and phase III CLL14 trials confirm chronic lymphocytic leukaemia patients treated with Venclexta/Venclyxto-based regimens achieve higher rates of undetectable minimal residual disease*, which may be associated with a lower risk of future disease progression or death             Basel, 5 December 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from the pivotal phase III MURANO and CLL14 studies support the efficacy of fixed-duration, chemotherapy-free Venclexta®/Venclyxto® (venetoclax)-based combinations in certain people with chronic lymphocytic leukaemia (CLL) and provide more evidence on the potential value of minimal residual disease (MRD)....

First Reported Allogeneic CAR T Data in Multiple Myeloma Highlights Initial Safety and Efficacy in Heavily Pretreated, Refractory PatientsIn the Ongoing Study, 31 Patients Were Evaluable for Safety and 26 Patients Were Evaluable for Efficacy as of Data CutoffApproximately 90% of Patients Treated Within Five Days of Study EnrollmentNo Bridging Therapy Required Prior to TreatmentALLO-715 with ALLO-647 Lymphodepletion Were Well Tolerated with No Graft-vs-Host Disease or Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS)60% Overall Response Rate (ORR) with Higher Cell Dose ALLO-715 (320M) and FCA Lymphodepletion; 40% Achieved a Very Good Partial Response (VGPR) or BetterMinimal Residual Disease (MRD) Assessed in Five Patients with VGPR or Better Response. All Five were MRD NegativeALLO-715 Cell Expansion and Persistence Observed...

– Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion –– Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion –– Nineteen patients have been dosed with CTX001 across both programs –– The New England Journal of Medicine publishes CTX001 manuscript containing the first report of investigational use of CRISPR/Cas9-based gene editing to treat inherited diseases in humans –ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 05, 2020 (GLOBE NEWSWIRE) — CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing...

– Evidence of biologic activity observed in each dose-escalation cohort treated to date –– Clinical activity includes one CR in a patient with a NPM1 mutation and one CR in a patient with a SETD2/RUNX1 mutation –– Continuous daily dosing well tolerated and with manageable safety profile to date –– Enrollment continues in dose escalation of Phase 1/2A clinical trial in patients with relapsed/refractory AML –– Anticipate recommending Phase 2 dose and advancing into expansion cohorts in first quarter of 2021 –– Management to host virtual investor event today at 2:00 p.m. ET / 11:00 a.m. PT –SAN DIEGO, Dec. 05, 2020 (GLOBE NEWSWIRE) — Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, today announced preliminary clinical data from...

Lead immuno-oncology development candidate NTLA-5001 shows high anti-tumor activity as promising cancer treatment in proof-of-concept mouse models of acute leukemiasProprietary process enhances tumor control in preclinical models and enables efficient, scalable genome editing and T cell manufacturing for NTLA-5001First-in-human trial will evaluate safety and activity of NTLA-5001 in AML patientsCAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting new preclinical data in support of NTLA-5001, the company’s wholly owned Wilms’ Tumor 1 (WT1)-directed T cell receptor (TCR)-T cell therapy candidate for the treatment of acute myeloid leukemia (AML), at the 62nd American Society of Hematology (ASH) Annual Meeting, taking place virtually from December 5-8, 2020. NTLA-5001 capitalizes...