Month: December 2020

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...

Data support novel approach to develop and manufacture a best-in-class, durable medicine for people living with hemoglobinopathiesIND filing for EDIT-301 planned by end of 2020CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced preclinical data and successful development of a large-scale manufacturing process for EDIT-301, a potentially best-in-class, one-time, durable, autologous cell therapy medicine to treat sickle cell disease and beta-thalassemia. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. The Company reported these data today at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH) being held virtually.The data showed that high levels of editing in CD34+...

CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that preclinical data with FTX-6058 for the treatment of sickle cell disease will be presented in three posters at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 5-8, 2020. FTX-6058 is a highly potent small molecule EED inhibitor that induces expression of fetal hemoglobin (HbF). Elevating HbF can compensate for the mutated adult hemoglobin that has been identified as the root cause of several hemoglobinopathies and can ameliorate or eliminate the symptoms of sickle cell disease.“We are encouraged by the robust preclinical data package and...

Interim analysis from the investigational ELARA study shows, 65% of patients with r/r follicular lymphoma evaluated for efficacy achieved a complete response and the overall response rate was 83%1Longer-term median follow-up of 40 months from the JULIET study showed a 33% two-year progression-free survival rate in patients with r/r DLBCL2Both ELARA and JULIET trials reaffirm safety profile of Kymriah with no new short- or long-term safety signals identifiedBasel, December 5, 2020 — Novartis announced analyses from two separate trials with Kymriah® (tisagenlecleucel) in patients with certain advanced lymphomas. In the interim analysis of the investigational Phase II ELARA study, Kymriah led to a complete response (CR) in 65% of patients with relapsed or refractory (r/r) follicular lymphoma (FL) and an overall response rate (ORR) of 83%...

BEAM-101 Data Highlight Precision of Base Editor with No Off-Target Editing Observed; Will Support Planned IND Submission in the Second Half of 2021In Vivo Data from Makassar Base Editing Program Demonstrate Long-term Engraftment and Editing Retention at 16 WeeksCAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) — Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that updated data from the company’s complementary base editing approaches to treat hemoglobinopathies are being presented during poster sessions at the 62nd American Society of Hematology Annual Meeting and Exposition (ASH 2020). The meeting is being held virtually December 5-8, 2020.“Base editing is ideally suited to precisely edit the genetic mutations that cause disease without disrupting...