News and Trends 4 Nov 2022 Centogene launches rare and neurodegenerative disease Biodata Network German company, Centogene N.V., today (November 4) announced its launch of Biodata Network, a portfolio of data-driven partnering solutions including licenses and insight report for biopharma and research institutions. The company, which works to find data-driven answers in rare and neurodegenerative diseases, wants to offer increased access to its insights. To do this, it has […] November 4, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
In Depth 3 Nov 2022 What’s on the horizon for gene editing-based therapies? Gene editing has come far in the past decade. However, the field is still finding its feet regarding challenges such as drug delivery, regulation and competition with first-generation gene therapies. Gene editing is a sizzling hot topic in the biotech industry, powered by advances in genetic tools such as CRISPR, TALENs and zinc-finger nucleases. Gene […] November 3, 2022 - 8 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 3 Nov 2022 Servier and OSE Immunotherapeutics complete enrollment in Sjögren’s syndrome trial Servier and French biotech company OSE Immunotherapeutics SA have completed patient enrollment in a phase 2a clinical trial evaluating the efficacy and safety of monoclonal antibody OSE-127/S95011 in primary Sjögren’s syndrome. The international, randomized, double-blind, placebo-controlled, phase 2a study is designed to evaluate the efficacy and tolerance of the monoclonal antibody OSE-127/S95011 in primary Sjögren’s […] November 3, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 24 Oct 2022 Syncona to acquire Applied Genetic Technologies Corporation Healthcare company Syncona Limited says its newly established portfolio company has agreed to acquire, by a tender offer, all outstanding shares of Applied Genetic Technologies Corporation (AGTC). AGTC is a clinical-stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an […] October 24, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
In Depth 20 Oct 2022 Opportunities and challenges for cell and gene therapy investments Companies in the cell and gene therapy space are struggling to raise investments amid adverse market conditions. However, this could be an opportunity for the strongest companies and investors to shine. After several years of record-breaking investments, funding is hard to come by in the gene and cell therapy space. Part of this trend relates […] October 20, 2022 - 9 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 13 Oct 2022 CMT Research Foundation funding boosts Samsara Therapeutics Charcot-Marie-Tooth treatment The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, has invested in Samsara Therapeutics Inc. Samsara Therapeutics is a biotech company focusing on discovering and developing therapeutics to restore autophagy – the process by which cells recycle damaged or dysfunctional components. Samsara is developing several novel, […] October 13, 2022 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 4 Oct 2022 ASPIRE set up to collaborate on amyloidosis A new group of biotech and pharma companies has been assembled by the Amyloidosis Research Consortium (ARC). ASPIRE (amyloidosis stakeholder partnerships for impact reach and equity), is a collaboration between biotech and pharmaceutical companies committed to advancing solutions, improving diagnosis and systems of care, and addressing obstacles to health equity on behalf of amyloidosis patients. […] October 4, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 4 Oct 2022 Aeglea BioTherapeutics updates homocystinuria program Aeglea BioTherapeutics, Inc. says dosing in the third cohort of the phase 1/2 clinical trial of pegtarviliase for the treatment of classical homocystinuria is under way. Patients in this cohort are receiving 1.35 mg/kg of pegtarviliase once weekly administered via subcutaneous injection; patients in cohorts 1 and 2 received doses of 0.15 mg/kg and 0.45 […] October 4, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 3 Oct 2022 FDA clears Kira Pharmaceuticals’ phase 2 trial for SLE-TMA treatment Kira Pharmaceuticals says the US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for KP104, a first-in-class bifunctional biologic that selectively and synergistically targets the alternative and terminal complement pathways. A phase 2 trial will evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104 in participants with […] October 3, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 3 Oct 2022 Alexion, AstraZeneca Rare Disease to acquire genomic medicine company LogicBio Therapeutics LogicBio Therapeutics, Inc. has entered into a definitive agreement under which Alexion, AstraZeneca Rare Disease (Alexion) will acquire LogicBio. The proposed acquisition brings LogicBio’s technology, experienced rare disease R&D team, and expertise in pre-clinical development to support Alexion’s growth in genomic medicines. Fred Chereau, president and chief executive officer at LogicBio, said: “We are excited […] October 3, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 3 Oct 2022 Poxel granted orphan drug designation for kidney disease treatment French biopharma company Poxel SA says the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PXL770 for the treatment of patients with autosomal-dominant polycystic kidney disease (ADPKD). PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator – and is also a phase 2 ready ADPKD asset, subject […] October 3, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 27 Sep 2022 Protocol for treatment of neurotrophic keratopathy submitted to FDA by ReGenTree Protocol for a second phase 3 clinical trial for a treatment for neurotrophic keratopathy (NK) – a degenerative disease of the corneal epithelium resulting from impaired corneal innervation, has been submitted. RegeneRx Biopharmaceuticals reported that its U.S. joint venture partner and licensee, HLB Therapeutics, together called ReGenTree, made the submission to the U.S. Food and […] September 27, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email