What are the new developments in gene editing, stem cell therapy, and cancer vaccines?

At the JPM conference, new molecular treatments such as mRNA, gene editing, and cell therapy are one of the focuses of the industry

Vertex drives development of multiple treatment modalities

Several cystic fibrosis therapies developed by Vertex Pharmaceuticals have changed the lives of this patient population

Image source: Vertex official website

Developed in conjunction with Semma Therapeutics, stem cell-differentiated islet cell therapy VX-880 demonstrated significant results in the first treated type 1 diabetes patient, with a 92% reduction in insulin use after 150 days of treatment, and a variety of Glycemic control indicators improved

Image source: Vertex official website

In 2022, the company will complete the dosing of VX-880 in more patients in a Phase 1/2 clinical trial and will submit an IND application to test the effect of encapsulated islet cell therapy in patients with type 1 diabetes

Image source: Vertex official website

Editas Medicine announces the latest research and development progress and layout

Editas Medicine, which develops gene-editing therapies, last year announced preliminary data from a Phase 1/2 clinical trial of its in vivo gene-editing therapy EDIT-101, a treatment for Leber congenital amaurosis 10 (LCA10)

The company's in vitro gene editing therapy EDIT-301, designed to increase fetal hemoglobin expression through gene editing, has been approved by the FDA for clinical trials to treat patients with sickle cell anemia and beta thalassemia

In addition, the company is developing EDIT-202, a natural killer cell therapy for induced pluripotent stem cell (iPSC) generation, a highly differentiated cell therapy that enhances therapy through dual knock-in and knock-out of multiple treatment characteristics

Gritstone bio boosts immunotherapy efficacy with neoantigen vaccine

Immune checkpoint inhibitors have revolutionized cancer treatment, but these immunotherapies do not work well for tumors with low tumor mutation burden

▲Mechanism of action of neoantigen cancer vaccine (Image source: Gritstone bio official website)

The company's neoantigen cancer vaccine, which combines AAV vectors and self-amplifying mRNA technology, has achieved promising results in Phase 1/2 clinical trials

References:

[1] JPM22, Day 4: Vertex aims to be cystic fibrosis leader into the 2030s; Gritstone hopes for new colorectal cancer biomarker.

[2] Editas Medicine Reports On Recent Progress And Outlook At JP Morgan Healthcare Conference.

[3] Intellia Corporate Overview at 40th Annual JP Morgan Healthcare Conference.

[4] Gritstone Announces First Patient Enrolled for Phase 2/3 Trial Evaluating Individualized Neoantigen Vaccine GRANITE for First Line (1L) Maintenance Treatment of Metastatic, Microsatellite-Stable Colorectal Cancer (MSS-CRC).

[5] Vertex JP MORGAN HEALTHCARE CONFERENCE.