Sickle Cell: Good Outcomes for Haploidentical Transplants Sickle Cell: Good Outcomes for Haploidentical Transplants
It is much easier to find eligible haploidentical donors — half-matched or partially matched — than eligible hematopoietic donors.MDedge News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - December 13, 2023 Category: Cancer & Oncology Tags: Hematology-Oncology Source Type: news

Vertex to pay Editas up to $100M, plus hefty annual fee, for CRISPR license
Vertex Pharmaceuticals Inc. will pay a hefty price to a local competitor before it starts selling its newly approved sickle-cell treatment. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 13, 2023 Category: Pharmaceuticals Authors: Rowan Walrath Source Type: news

'Half-Matched' BMTs Effective in Severe Sickle Cell Disease
(MedPage Today) -- SAN DIEGO -- A reduced-intensity "half-matched" bone marrow transplant (BMT) protocol demonstrated durable donor engraftment, with encouraging survival rates, in adults with severe sickle cell disease (SCD) for whom stem cell... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 13, 2023 Category: Hematology Source Type: news

A Closer Look at the Approval of CRISPR/Cas9 Gene Therapy for Sickle Cell Disease
(MedPage Today) -- On Friday, the FDA approved two gene therapies to treat patients with sickle cell disease (SCD). One of the therapies, called exagamglogene autotemcel (Casgevy), is the first FDA-approved gene therapy utilizing CRISPR/Cas9, a... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - December 11, 2023 Category: American Health Source Type: news

Sickle Cell Gene Therapy'Truly Transformative'Sickle Cell Gene Therapy'Truly Transformative '
Patients who received lovo-cel had durable remissions and reductions in vaso-occlusive events, pain crises, and hospitalizations in the longest running gene therapy trial for sickle cell disease.Medscape Medical News (Source: Medscape Transplantation Headlines)
Source: Medscape Transplantation Headlines - December 10, 2023 Category: Transplant Surgery Tags: Hematology-Oncology Source Type: news

bluebird bio Announces FDA Approval of LYFGENIA ™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events
SOMERVILLE, Mass.--(BUSINESS WIRE)--Dec. 8, 2023-- bluebird bio, Inc. (Nasdaq: BLUE) (“bluebird bio” or“bluebird”) today announced the U.S. Food and Drug Administration (FDA) has approved... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 10, 2023 Category: Drugs & Pharmacology Source Type: news

Lovo-Cel 'Life-Changing, Transformative' in Sickle Cell Disease
(MedPage Today) -- SAN DIEGO -- A one-time treatment with lovotibeglogene autotemcel (Lyfgenia, lovo-cel) achieved near complete resolution of severe pain crises in patients with sickle cell disease (SCD) in an analysis presented here. Of 34... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 10, 2023 Category: Hematology Source Type: news

Why the FDA ’s approval of revolutionary sickle cell gene therapy is a ‘big deal’
Sickle cell disease is a chronic, debilitating condition that affects nearly 100,000 Americans, most of them with African ancestry. Now, the FDA has approved a groundbreaking treatment for it that uses the gene-editing tool CRISPR. John Yang speaks with Yale School of Medicine assistant professor…#african #fda #johnyang #yaleschoolofmedicine #cececalhoun #ginakolata (Source: Reuters: Health)
Source: Reuters: Health - December 9, 2023 Category: Consumer Health News Source Type: news

FDA approves first CRISPR therapy
The Food and Drug Administration on Friday approved two gene therapies to treat sickle cell disease, one of the which is the first CRISPR/Cas9-based treatment to win regulatory approval in the US. The announcement is a landmark in the treatment of sickle cell disease, a devastating condition in…#africanamericans #nicoleverdun #fda #hba #hbs #crisprcas9 #hbf #deform #cas9 #rna (Source: Reuters: Health)
Source: Reuters: Health - December 9, 2023 Category: Consumer Health News Source Type: news

Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy
The Food and Drug Administration’s approval on Friday of two gene therapies for sickle cell disease sets up what could be an intense competition between Bluebird Bio, maker of Lyfgenia, and the partnership of Vertex Pharmaceuticals and CRISPR Therapeutics, whose drug is Casgevy. For the first…#bluebirdbio #lyfgenia #crisprtherapeutics #vertex #crispr (Source: Reuters: Health)
Source: Reuters: Health - December 9, 2023 Category: Consumer Health News Source Type: news

AI and Breast Cancer Screening; Cancer After Treatment for Sickle Cell
(MedPage Today) -- TTHealthWatch is a weekly podcast from Texas Tech. In it, Elizabeth Tracey, director of electronic media for Johns Hopkins Medicine in Baltimore, and Rick Lange, MD, president of the Texas Tech University Health Sciences Center... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 9, 2023 Category: Hematology Source Type: news

Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY ™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease
(Source: Reuters: Health)
Source: Reuters: Health - December 9, 2023 Category: Consumer Health News Source Type: news

New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most
Nasra Gwoto, 10, and her brother, Ramadhani, 12, traveled with their mother from Tanzania to India to get a bone-marrow transplant for sickle cell disease. The procedure is risky, and their mother wishes they could have received a new gene therapy instead. There is no clear path for African…#nasragwoto #tanzania #african #lyfgenia #bluebirdbio #vertex #saudiarabia #africa #whatsapp #shanimgaraganza (Source: Reuters: Health)
Source: Reuters: Health - December 9, 2023 Category: Consumer Health News Source Type: news

Vertex/CRISPR price sickle cell disease gene therapy at $2.2 mln
Vertex Pharmaceuticals (VRTX.O) and its partner CRISPR Therapeutics (CRSP.BN) said on Friday their sickle cell disease gene therapy Casgevy would be available at a list price of $2.2 million in the United States. Bluebird bio (BLUE.O) said it has set a list price of $3.1 million for its treatment,…#crisprtherapeutics #blueo #lyfgenia (Source: Reuters: Health)
Source: Reuters: Health - December 9, 2023 Category: Consumer Health News Source Type: news

F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing
People with the genetic disease have new opportunities to eliminate their symptoms, but the treatments come with obstacles that limit their reach. (Source: NYT Health)
Source: NYT Health - December 9, 2023 Category: Consumer Health News Authors: Gina Kolata Tags: Sickle Cell Anemia Food and Drug Administration Drugs (Pharmaceuticals) Crispr (DNA) CRISPR Therapeutics Vertex Pharmaceuticals Inc Genetic Engineering Source Type: news