Rocket Pharma unveils Phase 1/2 trial data for its Fanconi Anemia drug
Category: #health  By Pankaj Singh  Date: 2019-09-12
  • share
  • Twitter
  • Facebook
  • LinkedIn

Rocket Pharma unveils Phase 1/2 trial data for its Fanconi Anemia drug
  • The manuscript includes data of the first four trial participants that were treated with RP-L102 under the Phase 1/2 FANCOLEN-I study.
     
  • First RP-L102 trail data demonstrates increase in bone marrow engraftment levels, which further leads to stabilization as well as restored bone marrow function.

Rocket Pharmaceuticals, Inc., an American multi-platform clinical stage biotechnology firm, reportedly announced long-term data from the company’s ongoing Phase 1/2 clinical trial of RP-L102, gene therapy which is based lentiviral vector (LVV) for Fanconi Anemia (FA).

The manuscript includes data of the first four trail participants that were treated with RP-L102 under the Phase 1/2 FANCOLEN-I study, which used first-generation “Process A” without using any conditioning regimen. Follow-up for all of the primary four patients was kept as 18-30 months from RP-L102 administration.

Senior Vice President as well as Chief Medical Officer of Rocket Pharmaceuticals, Jonathan Schwartz, M.D. stated that the data from the company’s first study of RP-L102 demonstrates increase in bone marrow engraftment levels, which further leads to stabilization as well as restored bone marrow function.

Jonathan added that all gathered data highlights the unique natural selective advantage that exists in Fanconi Anemia for stem cells that are gene corrected over stem cells that are diseased, this potentially removes the conventional need for conditioning.

Jonathan also stated that by the end of 2019, the company would have a first look at the primary data from the company’s Phase 1 trial RP-L102 of ‘Process B’. This trial uses new cells and uses a modified process of stem cell enrichment, final drug product, commercial-grade vector as well as transduction enhancers.

The information printed in the manuscript is from four pediatric patients aged between 3-6 years. These patients received RP-L102 using cryopreserved or fresh CD34+ cells which were collected as well as transduced.

 

Source credit: https://rocketpharmaceuticals.gcs-web.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-publication-data-phase-12-trial

 

  • share
  • Twitter
  • Facebook
  • LinkedIn


About Author

Pankaj Singh    

Pankaj Singh

With an experience as an insurance underwriter and a freelance writer under his belt, Pankaj Singh boasts of quite some expertise in the field of content creation. A qualified Post Graduate in Management, Pankaj has also completed a detailed course in Digital Marketin...

Read More

More News By Pankaj Singh

Unacademy takes over live game streaming platform Rheo TV
Unacademy takes over live game streaming platform Rheo TV
By Pankaj Singh

Unacademy, an Indian educational technology company, has recently announced the purchase of Rheo TV, a live game streaming platform focused on making professional game streaming a mainstream career choice in India. Launched in August 2019, Rheo...

Byju's acquires Epic to strengthen footprint in North America
Byju's acquires Epic to strengthen footprint in North America
By Pankaj Singh

Byju's, a major Ed-tech company based in India, has reportedly taken over Epic, a US-based digital reading platform for kids, in a $500 million deal, with plans to enter the lucrative international market. The acquisition comes over three months...

Razorpay acquires TERA Finlabs to reinforce B2B credit infrastructure
Razorpay acquires TERA Finlabs to reinforce B2B credit infrastructure
By Pankaj Singh

The acquisition aligns with Razorpay’s goal to develop affordable credit solutions for underbanked small businesses Razorpay, a fintech startup based in India, has recently announced the acquisition of TERA Finlabs, a Bengaluru-based startup w...