View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. News
November 18, 2021updated 11 Jul 2022 2:17pm

Graphite Bio initiates enrolment in sickle cell disease therapy trial

For the trial, nearly 15 adolescent and adult patients with SCD will be enrolled at up to five trial centres in the US.

Graphite Bio has enrolled the first subject in the Phase I/II CEDAR clinical trial of its experimental therapy, GPH101, to treat sickle cell disease (SCD).

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

The company anticipates treating the first subject in the first half of next year.

A gene-edited autologous hematopoietic stem cell therapy, GPH101 is claimed to directly rectify the genetic mutation that causes SCD.

The open-label, multicentre trial will evaluate the safety, pharmacodynamics, success of engraftment, rate of gene correction, total haemoglobin in individuals with severe SCD treated with GPH101.

The trial will also assess various clinical and exploratory goals.

Nearly 15 adult and adolescent subjects will be enrolled at up to five trial centres in the US.

Graphite Bio noted that GPH101 is the first experimental treatment to leverage a well-differentiated technique to correct the mutation in the beta-globin gene.

This approach will aid in reducing the production of sickle haemoglobin and reinstate the expression of normal adult haemoglobin, potentially treating SCD.

The preliminary proof-of-concept results from the trial are expected by the end of next year.

Graphite Bio CEO Josh Lehrer said: “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 haemoglobin and restore the expression of normal adult haemoglobin.

“This approach has the potential to restore normal physiology and is viewed as the gold standard for curing sickle cell disease.”

A genetic blood disorder, SCD is caused by a mutation in the beta-globin gene. The mutation causes red blood cells to become misshapen leading to anaemia, blood flow obstructions, and a reduced lifespan, among others.

Related Companies

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena