Heat Biologics issued another patent on gp96 combination platform therapy

Pre-clinical studies combining Heat’s secreted gp96 plus OX40L T cell co-stimulator in a single therapy, administered locally, have demonstrated superior activity in preclinical studies compared to gp96 in combination with conventional OX40 antibody administered systemically by IV infusion. Additional potential advantages of this novel combination approach include enhanced memory T cell response, limited systemic toxicity and cost advantages compared to multiple systemic therapies.

The company believes this combination therapy holds enormous promise in the treatment of cancer and infectious diseases, potentially including *redacted*.

Mustang Bio gets Orphan Drug Designation for MB-107 in XSCID

MB-107 is currently being assessed in a Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital (St. Jude), UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. In May 2020, Mustang submitted an investigational new drug application (IND) to the FDA to initiate a multi-center Phase 2 clinical trial of MB-107 in newly diagnosed infants with XSCID who are between two months to two years of age. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-center trial led by St. Jude, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplantation (HSCT). The primary efficacy endpoint will be event-free survival. The initiation of this trial is expected early in the fourth quarter of 2020. Mustang is targeting topline data from this trial in the second half of 2022.

Another Phase 1/2 clinical trial for XSCID in patients over the age of two years, who have received prior HSCT, is underway at the National Institutes of Health, and Mustang expects to file an IND to the FDA to initiate a multi-center Phase 2 clinical trial in this population in the fourth quarter of 2020. This product candidate for XSCID in patients over the age of two years, who have received prior HSCT, is designated MB-207. The FDA granted a Rare Pediatric Disease Designation to MB-207 in August 2020.

X-linked severe combined immunodeficiency is a rare genetic disorder that occurs in approximately 1 per 225,000 births. It is characterized by the absence or lack of function of key immune cells, resulting in a severely compromised immune system and death by one year of age if untreated. Patients with XSCID have no T-cells or natural killer cells. Although their B-cells are normal in number, they are not functional. As a result, XSCID patients are usually affected by severe bacterial, viral or fungal infections early in life and often present with interstitial lung disease, chronic diarrhea and failure to thrive.

The specific genetic disorder that causes XSCID is a mutation in the gene coding for the common gamma chain (γc), a protein that is shared by the receptors for at least six interleukins. These interleukins and their receptors are critical for the development and differentiation of immune cells. The gene coding for γc is known as IL-2 receptor gamma, or IL2RG. Because IL2RG is located on the X-chromosome, XSCID is inherited in an X-linked recessive pattern, resulting in almost all patients being male.

Magenta named co-recipient of NIH grant for gene editing approach in HIV cure

This cross-institutional research program brings together leaders in the fields of gene editing, HIV and stem cell transplant. The team, which includes researchers from the University of Southern California, the University of Washington, the Fred Hutchinson Cancer Research Center, Harvard University, Massachusetts General Hospital; the Ragon Institute and Magenta Therapeutics, will explore novel hematopoietic stem and progenitor cell (HSPC) engineering and transplantation approaches aimed at achieving complete remission of HIV-1 infection. 

This collaboration with the multi-institution research team is to help advance gene editing approaches with Magenta’s novel targeted antibody drug conjugate (ADC) conditioning platform to one day be able to cure patients living with HIV. These studies leverage the company’s proprietary stem cell biology pipeline and ADC platform to provide important insights into which conditioning strategy is best suited to aim for HIV.