Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President: Toichi Takino; “Ono”) announced that the U.S. Food and Drug Administration (FDA) ...

Two months after Allogene abandoned plans for its CAR-T in leukemia, analysts have suggested that fresh phase 1 data in ...

Recent research from Michigan Medicine takes aim at the a variant in gene SCN1B, which causes a severe form of developmental epileptic encephalopathy.

Crispr Therapeutics AG has revenue declines but holds strong financials. Promising Sickle Cell therapy & 2025 data readouts ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

Despite research showing the benefits of circulating tumor DNA as an indicator of disease remission in B-cell lymphoma, ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

ViGeneron also received approval for dose escalation in the ongoing phase 1b clinical trial.

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, ...