Blood disorders Archives

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

News and Trends 27 Nov 2018

Berlin Biotech Raises €24M to Progress Septic Shock Candidate to Clinic

Adrenomed closed a €24M Series D round to fund further development of its antibody drug that treats septic shock by preventing excessive fluid loss and preserving the blood vessel walls. Wellington Partners and HBM Healthcare Investments, who specialize in investing in life science companies, joined existing shareholders to lead the fundraising round. Sepsis and resulting […]

November 27, 2018 - 3 minutesmins - By Helen Albert

Sickle Cell Disease Modus Therapeutics Cover

News and Trends 16 Jul 2018

Series A Boost for Swedish Biotech’s Sickle Cell Disease Treatment

Modus Therapeutics closed SEK 140M (€13.5M) in Series A financing to complete a Phase II study testing its sickle cell disease drug and to prepare for further clinical trials. The financing round was led by HealthCap, a European venture capital firm focused on life science investments, which will contribute SEK 60M (€5.8M). The remaining funds […]

July 16, 2018 - 3 minutesmins - By Alexander Burik

News and Trends 20 Apr 2018

Gene Therapy For Beta-Thalassemia Could Eliminate Need for Transfusions

Bluebird bio has obtained results from two clinical studies showing its new gene therapy could eliminate the need for red blood cell transfusions in patients with beta-thalassemia. The company plans to file for an EU approval of its treatment later this year. US firm bluebird bio has results from two clinical studies showing its LentiGlobin gene […]

April 20, 2018 - 2 minutesmins - By Alexander Burik

News and Trends 19 Dec 2017

This Gene Therapy for Hemophilia Can Reduce Bleeding by 70%

UniQure has published clinical data revealing that its gene therapy for hemophilia B is effective for years and lets patients stop weekly replacement therapy. From its headquarters in Amsterdam, uniQure is developing what could be one of the first gene therapies for hemophilia. A publication in the journal Blood yesterday details the results of a […]

December 19, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

Best in Biotech 18 Dec 2017

Smile! Here are the 12 Biggest Biotech Clinical Successes of 2017

Now that we’ve got the bad news out of the way, we’re here to spread some Christmas cheer with the biggest clinical successes in biotech over the past 12 months. It’s unlikely that one whole year will ever pass without a single hiccup, especially in a field as complex as biotech. For that reason, we […]

December 18, 2017 - 9 minutesmins - By Alex Dale

CRISPR Therapeutics clinical trials CRISPR Cas9

News and Trends 13 Dec 2017

CRISPR Therapeutics Plans First CRISPR/Cas9 Clinical Trial in Europe for 2018

CRISPR Therapeutics and Vertex Pharmaceuticals are taking action to start a first clinical trial with CRISPR/Cas9 in Europe in 2018. The blood disorder β-thalassemia could soon be the first human disease to be treated using CRISPR/Cas9 technology in Europe. CRISPR Therapeutics and its partner Vertex Pharmaceuticals have announced the first candidate from their collaboration that will go […]

December 13, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 4 Oct 2017

Ablynx Launches $175M IPO on Wall Street After Phase III Success

Fresh from announcing positive Phase III results, Ablynx has wasted no time in filing a $175M (€148M) IPO to continue developing its nanobodies technology. On Monday, the Belgian biotech shared the news that its nanobody, caplacizumab, received encouraging results for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP), a life-threatening blood disorder. This news boosted the company’s […]

October 4, 2017 - 2 minutesmins - By Alex Dale

News and Trends 2 Oct 2017

Belgian Biotech Breaks New Ground in Fight Against Life-Threatening Blood Disorder

Ablynx has triumphed where others have fallen by receiving positive Phase III results for the treatment of a dangerous blood disorder with caplacizumab. Ablynx develops nanobodies, molecules with the specificity of antibodies but much smaller in size. Its product, caplacizumab, has received encouraging results for the treatment of the life-threatening autoimmune blood disorder, acquired thrombotic thrombocytopenic purpura (aTTP), which […]

October 2, 2017 - 3 minutesmins - By Alex Dale

Bicycle Therapeutics Bioverativ Hemophilia blood disorders

News and Trends 6 Sep 2017

A €344M Partnership will Develop Bicycle Drugs for Hemophilia

Bicycle Therapeutics has partnered with Bioverativ to develop a new class of drugs for patients with rare blood disorders like hemophilia and sickle cell disease. Bicycle Therapeutics, a money-magnet biotech founded by Greg Winter, has closed a deal with US firm Bioverativ for the discovery, development and commercialization of bicycle drugs for rare blood disorders. […]

September 6, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

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Blood disorders

CRISPR Cell Therapy Shows Early Promise for Treating Blood Disorders

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

Gene Therapies for Blood Disorders Marching Through the Clinic

Berlin Biotech Raises €24M to Progress Septic Shock Candidate to Clinic

Series A Boost for Swedish Biotech’s Sickle Cell Disease Treatment

Gene Therapy For Beta-Thalassemia Could Eliminate Need for Transfusions

This Gene Therapy for Hemophilia Can Reduce Bleeding by 70%

Smile! Here are the 12 Biggest Biotech Clinical Successes of 2017

CRISPR Therapeutics Plans First CRISPR/Cas9 Clinical Trial in Europe for 2018

Ablynx Launches $175M IPO on Wall Street After Phase III Success

Belgian Biotech Breaks New Ground in Fight Against Life-Threatening Blood Disorder

A €344M Partnership will Develop Bicycle Drugs for Hemophilia

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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