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BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases

12/27/2021

Excerpt from the Press Release: SAN RAFAEL, Calif. and SHANGHAI, Dec. 16, 2021 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, today announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated…

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Glycomine Announces First Dosing in Phase 1 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG

12/24/2021

Excerpt from the Press Release: SAN CARLOS, Calif.–(BUSINESS WIRE)–Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that the company has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for GLM101 for the treatment of PMM2-CDG and has initiated dosing healthy volunteers…

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Vincerx Pharma Announces First Patient Dosed in Phase 1 Dose-Escalation Study of VIP152 in Relapsed or Refractory Chronic Lymphocytic Leukemia or Richter Syndrome

12/24/2021

Excerpt from the Press Release: PALO ALTO, Calif., Dec. 17, 2021 (GLOBE NEWSWIRE) — Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today announced that the first patient has been dosed in the Company’s Phase 1 dose-escalation study of VIP152 in…

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Passage Bio Announces Positive Interim Safety and Biomarker Data and Advances Phase 1/2 Trial of PBGM01 in GM1 Gangliosidosis

12/23/2021

– Independent Data Monitoring Committee recommends proceeding with two additional planned patient cohorts, which are now being recruited in parallel – Positive safety profile, including no serious adverse events, no complications related to intra-cisterna magna injection and no evidence of dorsal root ganglion toxicity – Intra-cisterna magna delivery resulted in substantial increases in beta-galactosidase enzyme…

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EVUSHELD long-acting antibody combination retains neutralizing activity against Omicron variant in independent FDA study

12/23/2021

Only antibody authorized in the US for pre-exposure prophylaxis of COVID-19 Excerpt from the Press Release: WILMINGTON, Del.–(BUSINESS WIRE)–AstraZeneca’s EVUSHELD (tixagevimab co-packaged with cilgavimab), a long-acting antibody combination for the prevention of COVID-19, retained neutralizing activity against the Omicron SARS-CoV-2 variant (B.1.1.529), according to new preclinical data. In this study,EVUSHELD’s Inhibitory Concentration 50 (IC50), a measure…

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Notable Presents Clinical Validation Data from Two Phase 2 Trials in Adult and Pediatric Acute Myelogenous Leukemia at the 63rd American Society of Hematology (ASH) Annual Meeting

12/23/2021

-Studies demonstrate high correlation between predicted response by Notable’s predictive precision medicines platform and actual clinical response, short-term and at one year- -Interim analyses of studies with MD Anderson Cancer Center and Texas Children’s Hospital investigators corroborate platform prediction data from Standford University collaboration- Excerpt from the Press Release: FOSTER CITY, Calif.–(BUSINESS WIRE)–Notable Labs, Inc.…

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Forma Therapeutics’ Investigational Olutasidenib in Combination with Azacitidine Yields Durable Complete Remission in Patients with mIDH1 Acute Myeloid Leukemia

12/21/2021

First Phase 2 combination trial results presented in oral session at 2021 ASH Annual Meeting Olutasidenib with azacitidine well tolerated with a safety profile largely consistent with olutasidenib alone ​ Molecular characteristics of olutasidenib monotherapy response reported in ASH poster presentation Excerpt from the Press Release: WATERTOWN, Mass.–(BUSINESS WIRE)–Dec. 16, 2021– Forma Therapeutics Holdings, Inc.…

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Rubius Therapeutics to Highlight the Power of its RED PLATFORM®, Significant Advances Across Red Cell Therapeutic Oncology Pipeline and New Type 1 Diabetes Program at Platform and Pipeline Day

12/21/2021

Highly Versatile and Programmable RED PLATFORM Enables Multiple Modalities to Target Different Immune Pathways Across a Range of Diseases Demonstrated Tolerance Induction with Bystander Suppression in Type 1 Diabetes Preclinical Program with Findings Translatable to Multiple T Cell-Mediated Autoimmune Diseases Advancing Next-Gen Artificial-Antigen Presenting Cells (aAPCs) with Loadable MHC Class 1 Platform, Enabling Presentation of…

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uniQure Announces Clinical Update on First Patients in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

12/20/2021

~ Treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up ~ ~ Neurofilament Light Chain (NfL) rose as expected immediately following surgery andreturned to baseline in treated patients ~ ~ A total of 19 patient procedures have been performed in the U.S.…

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Hologic Presents Breast Cancer Index™ Data at SABCS 2021 Showing Net Benefit Predictive Value for Endocrine Therapy Continuation

12/17/2021

Breast Cancer Index reaffirmed as a significant predictive and prognostic diagnostic tool to personalize extended endocrine decisions Excerpt from the Press Release: MARLBOROUGH, Mass.–(BUSINESS WIRE)–Hologic, Inc. (Nasdaq: HOLX) and its subsidiary, Biotheranostics, Inc., today announced new data demonstrating that Breast Cancer Index™ (BCI) not only predicts preferential recurrence-prevention benefit from extended endocrine therapy (EET), but…

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