Blood disorders Archives

More News! 16 Nov 2022

Mineralys has positive topline results from trial evaluating treatment of hypertension

U.S.-based Mineralys Therapeutics, Inc., announced today (November 16) topline results from its phase 2 Target-HTN trial in individuals with uncontrolled and resistant hypertension. MLS-101, a highly selective investigational aldosterone synthase inhibitor, at doses of 50 mg and 100 mg once daily, met its primary endpoint with statistically significant and clinically meaningful reduction in systolic blood […]

November 16, 2022 - 4 minutesmins - By Liza Laws

News and Trends 8 Nov 2022

Rallybio announces positive phase 1 results for drug treating PNH and gMG

Affibody’s licensee Rallybio Corporation has announced positive topline results from a phase 1 single ascending dose (SAD) study in healthy participants of RLYB116. RLYB116 is a novel, potentially long-acting, subcutaneously administered inhibitor of complement factor 5, or C5, in development for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and generalized myasthenia gravis (gMG). […]

November 8, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 28 Sep 2022

Primary Aldosteronism Foundation creates alliance to tackle hypertension

The Primary Aldosteronism Foundation has announced the creation of a multi-stakeholder alliance to drive diagnosis and treatment of primary aldosteronism, a common cause of hypertension. Among the partners is Mineralys Therapeutics, Inc, a biopharmaceutical company developing a novel therapy for the treatment of hypertension. The initiative will facilitate the design and implementation of interventions to […]

September 28, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 22 Sep 2022

National Hemophilia Foundation launches research fund

The National Hemophilia Foundation (NHF) in the U.S. has announced the launch of Pathway to Cures (P2C), a new venture philanthropy investment fund focused on creating transformational impact across all inheritable blood disorders. Investing alongside and partnering with traditional private capital funds and other industry organizations, P2C hopes to spur the development of inheritable blood […]

September 22, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 11 Aug 2022

Gemini Therapeutics and Disc Medicine announce merger agreement

Gemini Therapeutics and Disc Medicine have entered into a definitive merger agreement to combine in an all-stock transaction. The announcement came yesterday (August 10) and the combined company will focus on advancing Disc’s pipeline of hematology programs and is expected to operate under the name Disc Medicine Inc. upon shareholder approval. The work will include […]

August 11, 2022 - 2 minutesmins - By Liza Laws

beta-thalassemia blood APS antiphospholipid syndrome

News and Trends 11 Aug 2022

Study of drug to treat porphyria disorders initiated by Disc Medicine

A phase 2 clinical study of bitopertin in patients with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) has been initiated by Disc Medicine. The announcement of the study, called BEACON, was made August 10. EPP or XLP belong to a group of the porphyria disorders, which are all characterized by abnormally high levels of chemicals, […]

August 11, 2022 - 3 minutesmins - By Liza Laws

News and Trends 4 May 2021

Bluebird Bio Withdrawal Raises Gene Therapy Doubts in Europe

Last month, the US firm bluebird bio abandoned the sale of its gene therapy Zynteglo in Germany after a pricing dispute with health authorities — a blow that could have repercussions for the rollout of other gene therapies in Europe. Zynteglo was approved in the EU in 2019 as the first-ever gene therapy for the […]

May 4, 2021 - 5 minutesmins - By Anita Chakraverty

News and Trends 14 Jan 2020

Bluebird Bio Launches First Gene Therapy for Beta Thalassemia in Germany

The US company bluebird bio has launched the first gene therapy for the blood disorder transfusion-dependent beta thalassemia in the EU, with a hospital in Germany becoming its first qualified treatment center. Bluebird bio’s gene therapy, branded as Zynteglo, was given conditional market approval by the EU in June 2019. It was the first gene […]

January 14, 2020 - 3 minutesmins - By Jonathan Smith

News and Trends 20 Dec 2019

Freeline’s Rare Disease Gene Therapies Get €72M Series C Boost

The UK gene therapy biotech Freeline Therapeutics is to receive an investment worth €72M (£61.1M) from the UK VC firm Syncona in a Series C round to fund the clinical development of gene therapies for the blood disorder hemophilia B, Fabry disease, and other rare conditions. Of the €72M commitment, Syncona has already wired €36M […]

December 20, 2019 - 2 minutesmins - By Jonathan Smith

crispr therapeutics vertex beta thalassemia

News and Trends 19 Nov 2019

CRISPR Cell Therapy Shows Early Promise for Treating Blood Disorders

CRISPR Therapeutics and its US partner Vertex Pharmaceuticals have released the first clinical results for a cell therapy made using the gene editing tool CRISPR/Cas9 in patients with genetic blood disorders. The interim results concern two patients enrolled in different phase I/II clinical trials testing the same therapy in two blood disorders: transfusion-dependent beta-thalassemia and […]

November 19, 2019 - 3 minutesmins - By Jonathan Smith

News and Trends 30 Apr 2019

Orchard’s Gene Therapy Shows Potential in Treating Patients with Beta Thalassemia

Patients with the life-threatening blood disorder beta-thalassemia required fewer blood transfusions after receiving a gene therapy developed by the UK company Orchard Therapeutics, with four out of nine no longer depending on transfusions. Beta thalassemia is a genetic disorder that affects hemoglobin, a protein that blood cells use to carry oxygen. In the most severe […]

April 30, 2019 - 3 minutesmins - By Jonathan Smith

uniQure Freeline aruvant blood disorders

News and Trends 4 Dec 2018

Gene Therapies for Blood Disorders Marching Through the Clinic

Gene therapy holds great potential as a cure for blood disorders such as hemophilia and sickle cell disease, which have limited treatment options. This week, several gene therapy companies have released promising clinical trial results in hemophilia and sickle cell anemia. Presenting at the 2018 annual meeting of the American Society of Hematology, Dutch company uniQure […]

December 4, 2018 - 3 minutesmins - By Jonathan Smith

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