Gene Editing

CRISPR gene editing

 

Update: 30 May 2019

  • The FDA has granted Fast Track Designation for CTX001 for the treatment of transfusion dependent beta thalassaemia.

Sources:

https://www.globenewswire.com/news-release/2019/04/16/1804601/0/en/CRISPR-Therapeutics-and-Vertex-Announce-FDA-Fast-Track-Designation-for-CTX001-for-the-Treatment-of-Beta-Thalassemia.html

 

Update: 29 March 2019

  • The Phase 1/2 Study in Beta Thalassaemia is an open-label trial aiming to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with transfusion-dependent thalassaemia (TDT), with non-β00genotype subtypes. Two patients will be initially treated sequentially and, pending data from these, the trial will open for broader enrolment.

The first patient has been treated already.

  • The Phase 1/2 Study in Sickle Cell Disease (SCD) is an open-label trial aiming to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. Similar to the trial in beta thalassemia two patients will be initially treated sequentially and, pending data from these, the trial will open for broader enrolment.

The first patient has been enrolled in the U.S. and is expected to be infused with CTX001 in mid-2019.

Sources:

http://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-progress-clinical

 

Update: 01 February 2019

  • The FDA has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD).
  • The Fast Track Program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.  A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA).

Sources:

http://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-fda-fast-track

 

 Update: 02 October 2018

CRISPR Therapeutics and Vertex Pharmaceuticals have become the first commercial sponsors of a human clinical trial for a CRISPR gene-editing system.

Clinical Trial Application for CTX001 was submitted in Europe to support initiation of Phase 1/2 clinical study in β-thalassemia in 2018

 The trial will enrol 30 β-thalassaemia patients at a single hospital in Regensburg, Germany.

This treatment will introduce a second genetic mutation into the patient’s genome that causes a naturally-occurring condition called Hereditary Persistence of Fetal Hemoglobin, or HPFH. HPFH is benign, asymptomatic, and requires no treatment. The only difference between those with HPFH and those without, is the type of haemoglobin they produce. HPFH patients produce fetal haemoglobin, while patients with thalassaemia produce altered and ineffective haemoglobin molecules.

 

Sources:

https://seekingalpha.com/article/4206056-crispr-therapeutics-tries-hand-thalassemia-vertex

http://ir.crisprtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-co-develop-and-co-commercialize

 

Update: 20 December 2018

  • In October the FDA has lifted the hold (in place since May) for the initiation of human clinical trials for SCD patients.
  • The FDA approved trials for β-thalassaemia patients in August.
  • Approvals have already been obtained for initiation of trials multiple countries outside the U.S. for both β-thalassemia and SCD.
  • The initiation of Phase 1/2 clinical study in SCD by the end of 2018 remains on track and currently patients with transfusion dependent β-thalassemia are enrolling in a Phase 1/2 trial in β-thalassemia in Europe.

 Source: http://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-fda-has-lifted-clinical

 

Bioverativ gene editing

 

Update: 30 May 2019

No update available.

 

Update: 29 March 2019

The results received from the first patient treated with ST-400 in the Phase 1/2 THALES study have shown:

  • Stable haemoglobin levels at 7 weeks post ST-400 infusion (~9 g/dL)
  • Continuous increase in levels of fetal haemoglobin (from 1% of total hemoglobin at the time of infusion to 31% as of the most recent measurement)
  • Presence of indels (genome edits) around white blood cells, indicating that the successful ex vivo editing of the BCL11A gene

It is noted that the patient has the most severe form of transfusion-dependent beta thalassemia (β00), and for the two years prior to treatment in the study, received packed red blood cell (PRBC) transfusions every other week.

The patient received several PRBC transfusions for approximately two weeks after the ST-400 infusion. During the subsequent five weeks no further PRBC transfusions have been required.

During the ST-400 infusion, the patient experienced a serious adverse event, a transient allergic reaction considered related to the cryoprotectant present in the product. Thereafter, the post-transplant clinical course was routine.

More date is expected to be presented in late 2019.

Sources:

https://investor.sangamo.com/news-releases/news-release-details/sangamo-provides-clinical-development-update-including-early

 

Update: 02 October 2018

Sangamo Therapeutics and Bioverativ Therapeutics have begun a Phase 1/2 study of gene editing in patients with transfusion-dependent β-thalassemia in the United States.

This trial will investigate whether increasing the production of fetal haemoglobin can reduce or eliminate the need for blood transfusions.

Participating patients will have their CD34+ blood stem cells removed and genetically edited so as to boost their production of fetal haemoglobin. These edited stem cells will then be infused back into patients. The trial will enrol 6 patients between the ages of 18 and 40 with transfusion-dependent β-thalassemia. The first trial site has just opened in Oakland and additional trial sites are expected to open soon.

Source:http://www.thalassemia.org/sangamo-therapeutics-bioverativ-therapeutics-begin-new-clinical-trial-transfusion-dependent-beta-thalassemia/

 

Update: 20 December 2018

No update available.