CELL AND GENE THERAPY
Graphite Bio | November 26, 2021
Graphite Bio, Inc. a clinical-stage, next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, announced that the first patient has been enrolled in the company’s Phase 1/2 clinical trial of GPH101, an investigational gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes sickle cell disease. The company expects to treat the first patient with GPH101 in the first half of 2022, with initial proof-of-concept data anticipated by the end of 2022.
“GPH101 is the first investigational therapy to enter clinical development that uses our next-generation gene editing platform technology to directly correct the mutation in the beta-globin gene that causes sickle cell disease. Using our gene correction approach, we have demonstrated in preclinical studies an ability to decrease the production of harmful sickle hemoglobin and restore the expression of normal adult hemoglobin. This approach has the potential to restore normal physiology and is viewed as the gold standard for curing sickle cell disease. We are thrilled that our first patient is now enrolled in our CEDAR clinical trial, and we look forward to evaluating GPH101’s potential as we continue to advance its development with urgency in hopes of delivering a curative therapy to the sickle cell community.”
Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio
The CEDAR trial is an open-label, multi-center Phase 1/2 clinical trial of GPH101 designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics of GPH101 in patients with severe SCD. The trial will enroll approximately 15 adult and adolescent participants at up to five clinical trial sites in the United States.
Graphite Bio will present information about the CEDAR trial at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, being held virtually and in Atlanta December 11-14. The company’s poster presentation will take place on Saturday, Dec. 11, at 5:30-7:30 p.m. ET.
About Sickle Cell Disease (SCD)
SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. SCD is caused by a single mutation in the beta-globin gene that leads red blood cells to become misshapen, resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span of approximately 20-30 years. Despite advancements in treatment and care, progressive organ damage continues to cause early mortality and severe morbidity, highlighting the need for curative therapies.
GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore normal adult hemoglobin (HbA) expression, thereby potentially curing SCD.
Graphite Bio is evaluating GPH101 in the CEDAR trial, an open-label, multi-center Phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing company harnessing high efficiency targeted gene integration to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases. Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. Graphite Bio’s platform allows it to precisely correct mutations, replace entire disease-causing genes with normal genes or insert new genes into predetermined, safe locations. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.
Blue Water Ventures International | January 20, 2022
Psycheceutical, Inc. a biotechnology company dedicated to the development and commercialization of psychedelic medicines, announced the company has been fully acquired by Blue Water Ventures International, Inc.
“We are thrilled that this deal has closed and that Psycheceutical is now a wholly-owned subsidiary of a publicly-listed company and has secured important near-term capital to pursue our growth plans. We will immediately begin the next steps of our growth strategy as we work to bring our revolutionary psychedelic drug delivery technologies to the market, starting with initial clinical trials.”
Psycheceutical CEO Chad Harman
BWVI acquired Psycheceutical, Inc. by way of a merger, resulting in Psycheceutical becoming a wholly-owned subsidiary of BWVI. Now that the acquisition has occurred, BWVI intends to cease its previous business activities and the company will focus solely on psychedelic drug development. Once regulatory approvals have been received, the company will consummate related corporate actions, including a name and symbol change.
About Psycheceutical, Inc.
Psycheceutical, Inc. is seeking to develop cutting-edge delivery technologies for safe and effective psychedelic pharmaceutical medicines. Powered by a team with more than 100 years' combined experience in development, regulatory approval processes and commercialization across the pharmaceutical industry, Psycheceutical is on a mission to bring safety and efficacy to psychedelic compounds., today announced the company has been fully acquired by Blue Water Ventures International, Inc.
Acticor Biotech | October 05, 2020
Acticor Biotech, a clinical stage biotechnology company involved in the acute phase of thrombotic diseases, including acute ischemic stroke, today announced the completion of its ACTIMIS Dose Escalation Phase of Glenzocimab Study as add-on to standard of care in Patients with acute ischemic stroke. This Dose Escalation Phase was successfully completed with 60 patients enrolled from 6 European countries (France, Belgium, Germany, Spain, Switzerland and Italy). 5 cohorts of patients presenting with an acute ischemic stroke episode of moderate to severe intensity were enrolled in this study and randomly received glenzocimab at one of 4 ascending doses or a placebo, in a blind fashion as a 6-hour single-dose infusion. Patients were evaluated continuously for 24 hours, then 7 and 90 days after the episode. Approximately 50% were also treated with the thrombolytic agent rtPA (ACTILYSE®), and the other 50% received rtPA and underwent a mechanical thrombectomy. The DSMB met on 5 occasions in between each cohort and at the end of the last administration, and they analyzed the safety data with a particular focus on the advent of intra-cerebral hemorrhages and other bleeding related events. The last analysis performed after the target dose of 1000mg had been administered to 12 patients confirmed both the absence of increased bleeding when glenzocimab is added to rtPA and to rtPA and thrombectomy, and the absence of any dose-related trend in the number and nature of adverse events recorded.