Clinical Trial Updates (SCD)

Gene Editing

Update: 30 June 2021

BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle cell disease in the Phase 1/2 PRECIZN-1 study has been granted EMA Orphan Designation. The decision was based on early data from three patients that had 52 weeks, 13 weeks, and 29 days of follow-up, respectively.

Source: https://www.businesswire.com/news/home/20210317005807/en/Sangamo-Announces-EMA-Releases-Details-Supporting-Orphan-Designation-for-BIVV003-for-the-Treatment-of-Sickle-Cell-Disease

 

Update: 08 January 2021

No update available

 

Update: 05 November 2020

No update available

 

Update: 25 August 2020

No update available

 

Update: 31 May 2020

  • Enrolment of SCD patients continues.
  • Data from the study will be presented at a future date.

Source: https://www.fool.com/earnings/call-transcripts/2020/05/12/sangamo-therapeutics-inc-sgmo-q1-2020-earnings-cal.aspx

 

Update: 31 March 2020

No update available.

 

Update: 31 January 2020

Data presented at 61st Annual Meeting of the American Society of Hematology revealed that >90% of edited cells were biallelic, displaying on average 27-38% more HbF% despite variation in donor baseline levels.

Editing resulted in a 3-fold HbF increase.

Source: https://ashpublications.org/blood/article/134/Supplement_1/974/427026/Zinc-Finger-Nuclease-Mediated-Disruption-of-the?searchresult=1

Update: 30 June 2021

New data has been presented at EHA2021 on the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, showing a consistent and sustained response to treatment:

  • Follow-up data of 4 TDT patients infused with CTX0001 was presented. All patients demonstrated increases in total haemoglobin and HbF. No patient reported any VOC’s for the duration of follow-up (19.2 months).

Source: EHA2021

 

CRISPR gene editing (SCD) – UCSF Benioff Children’s Hospital Oakland

 Update: 30 June 2021

The U.S. Food and Drug Administration approved the start of the first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for SCD.

CRISPR_SCD001 uses the power of the CRISPR-Cas9 gene editing system to replace the mutated HBB gene in a patient’s hematopoietic stem cells with a healthy version.

Unlike other investigational gene editing approaches for sickle cell, CRISPR_SCD001 delivers the CRISPR-Cas9 machinery to cells without relying on a virus as a transport agent. Its method, called electroporation, uses electrical pulses to create temporary pores in cell membranes that allow for the gene-editing tool to enter.

Phase 1/2 trial, up to nine patients with severe SCD, ages 12–35 over 4 years.

Source: https://sicklecellanemianews.com/2021/04/01/first-trial-crispr-cas9-based-gene-editing-therapy-for-scd-approved/

 

CRISPR gene editing (SCD) – GPH101

 Update: 30 June 2021

The investigational gene editing therapy GPH101 has been approved by the FDA for a Phase 1/2 clinical trial. The new, open-label trial, called CEDAR, is designed to evaluate the safety and pharmacological properties of GPH101, as well as its preliminary efficacy in adults and adolescents with severe SCD.

GPH101 leverages the power of the CRISPR-Cas9 gene-editing tool, along with a natural DNA repair mechanism, to remove the mutation in the beta-globin (HBB) gene that causes SCD, and replace it with the correct DNA sequence.

Source: https://sicklecellanemianews.com/2021/01/19/clinical-trial-cleared-for-gph101-first-potentially-curative-sickle-cell-disease-scd-gene-editing-therapy/?utm_source=Sickle+Cell+Anemia+News&utm_campaign=2fa1fa2cf5-RSS_EMAIL_CAMPAIGN&utm_medium=email&utm_term=0_b01e3fbae8-2fa1fa2cf5-73559237

 

Update: 08 January 2021

New data has been presented at ASH2020 on the investigational CRISPR/Cas9- based gene-editing therapy, CTX001, showing a consistent and sustained response to treatment:

  • 6 patients with SCD have been dosed with CTX001.
  • 3 patients have reached at least 3 months follow up after CTX001 dosing. All three patients showed a similar pattern of response, with rapid and sustained increases in total hemoglobin and fetal hemoglobin, as well as elimination of VOCs for a duration between 3 – 15 months post infusion. Total hemoglobin in the range of 5 – 13.2 g/dL and fetal hemoglobin levels from 31.3% to 48.0%.
  • There were no SAEs considered related to CTX001, and the majority of non- serious adverse events were considered mild to moderate.

Source: https://news.vrtx.com/press-release/crispr-therapeutics-and-vertex-present-new- data-investigational-crisprcas9-gene

 

Update: 05 November 2020

  • The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001.
  • Data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD) will be presented at ASH2020 to be held in December 2020.
  • Additional data will be presented at ASH, including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.

Sources: https://news.vrtx.com/press-release/crispr-therapeutics-and-vertex-pharmaceuticals-announce-priority-medicines-prime

https://news.vrtx.com/press-release/crisprcas9-gene-editing-therapy-ctx001tm-severe-hemoglobinopathies-accepted-plenary

 

Update: 25 August 2020

Data presented at EHA25 Annual Congress of the CLIMB-121 Trial in Severe Sickle Cell Disease reflect longer-duration follow-up data for the first patient with SCD treated with CTX001.

Patient 1 with SCD experienced 7 vaso-occlusive crises (VOCs) and 5 packed red blood cell transfusions per year before enrolling in the clinical trial.

The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion.

New data show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months.

CLIMB-SCD-121: Dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.

Source: https://investors.vrtx.com/news-releases/news-release-details/crispr-therapeutics-and-vertex-announce-new-clinical-data

 

Update: 31 May 2020

  • Regenerative Medicine Advanced Therapy (RMAT) designation has been granted by the FDA.
  • RMAT seeks to fasten the development & review of new therapies, allowing for priority review and possibly faster approval by the FDA.
  • CTX001 uses CRISPR-Cas9 gene editing tool to produce higher levels of fetal haemoglobin in RBCs. Increased levels of fetal haemoglobin are expected to lower the frequency of vaso-occlusive crises (VOCs).
  • Preliminary data from the Phase 1/2 CLIMB-SCD-121 trial (NCT03745287), which is assessing the safety and effectiveness of a single dose of CTX001 in patients with severe SCD, has shown that the therapy safely increased the levels of fetal hemoglobin in the first patient dosed, effectively preventing the occurrence of VOCs.

 

Update: 31 March 2020

No update available.

Update: 30 June 2021

The U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the EDIT-301 clinical trial, and patients can be dosed. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease.

Source: https://www.biospace.com/article/releases/editas-medicine-announces-the-fda-has-cleared-initiation-of-the-edit-301-clinical-trialedit-301-is-in-development-as-a-best-in-class-durable-medicine-for-people-living-with-sickle-cell-disease/

 

Update: 08 January 2021

No update available

 

Update: 05 November 2020

  • The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for EDIT-301.
  • An investigational new drug application (IND) for EDIT-301 is planned to be filed by the end of 2020.

Source: https://www.globenewswire.com/news-release/2020/08/24/2082611/0/en/Editas-Medicine-Receives-Rare-Pediatric-Disease-Designation-for-EDIT-301-for-the-Treatment-of-Sickle-Cell-Disease.html

 

Update: 25 August 2020

No update available.

 

Update: 31 May 2020

Editas Medicine is developing EDIT-301 using Cas12a (Cpf1), a proprietary enzyme, as a potentially best-in-class medicine to treat sickle cell disease and β-thalassaemia. Preclinical in vivo toxicology studies are in progress and the Company expects to file to the FDA for Investigational New Drug (IND) for sickle cell disease by the end of 2020.

Source: https://www.globenewswire.com/news-release/2020/05/07/2029304/0/en/Editas-Medicine-Announces-First-Quarter-2020-Results-and-Update.html

Update: 30 June 2021

No update available

 

Update: 08 January 2021

No update available

 

Update: 05 November 2020

Patient enrolment has begun at MSKCC.

Source: https://www.mskcc.org/cancer-care/clinical-trials/20-411

 

Update: 25 August 2020

No update available.

 

Update: 31 May 2020

No update available.

 

Update: 31 March 2020

The FDA has authorised the start of a Phase 1/2 clinical trial testing a genome editing-based therapy for adults with severe complications of sickle cell disease (SCD).

The clinical trial uses CRISPR/Cas9 genome-editing technology to edit the genome to increase the development of fetal haemoglobin, thus increasing the efficiency of oxygen transportation in the body.

Animal studies have suggested an increase of up to 30% of fetal haemoglobin in red blood cells.

Source: https://sicklecellanemianews.com/2020/04/02/phase-1-2-trial-of-crispr-gene-editing-scd-cleared-by-fda/?utm_source=Sickle+Cell+Anemia+News&utm_campaign=a9ec38e189-RSS_EMAIL_CAMPAIGN&utm_medium=email&utm_term=0_b01e3fbae8-a9ec38e189-73559237

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