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4D Molecular Therapeutics Fabry Gene Therapy Granted A Fast Track Designation By The FDA

4D-310, new gene therapy for Fabry disease that is being developed by 4D Molecular Therapeutics, has been recently granted a fast track designation by the US FDA. The fast track designation gives the developer access to more frequent communication with the FDA during the development process.

Fabry disease is caused by mutations in the GLA gene, which provides instructions for making the enzyme alpha-galactosidase A. The mutations result in a missing enzyme or non-functional, which leads to the toxic accumulation of molecules that this enzyme usually is responsible for breaking down. This is of particular relevance to Fabry, in which heart disease is a major cause of mortality.

David Kirn CEO and Founder of 4D Molecular Therapeutics says that 4D-310 is designed to drive high-level AGA enzyme expression directly within the diseased target tissues themselves, including the heart, kidney and blood vessels, in addition to driving high and stable blood concentrations, which they believe has the potential to benefit patients with the disease.

(Source: Fabry Disease News, 2020)


Andelyn Biosciences Selects Its Gene Therapy Manufacturing Affiliate. 

Andelyn Biosciences and Ohio State University have reached a deal on a west campus site for the hospital’s gene therapy manufacturing affiliate. Andelyn Biosciences plans a $74 million biotechnology manufacturing facility for genetic material used in research and clinical trials.

Andelyn Biosciences plans to build an 85,000-square-foot facility, with many multiples of the current capacity, targeted for a 2023 opening. It includes the creation of 170 jobs with the potential to bring the number to up to 200. The biotech facility fits in with Ohio State’s Innovation District planned for what’s now prairie and intramural sports fields.

Additionally, Andelyn is teaming up with OSU’s Wexner Medical Center on a $100 million proton therapy cancer center for the planned outpatient complex in the district.

(Source: Biz Journal, 2020)


Acepodia Hopes to Cut Cell Therapy Manufacturing Costs

According to Acepodia, Natural Killer (NK) cells can eradicate tumors which could help in cutting the cost of cell therapies. The company recently began Phase I trials of its lead drug candidate, a cancer treatment based on a new cell therapy platform. Antibody Cell Conjugation (ACC) is used to attach tumor-fighting or other antibodies to the surface receptors of the NK cells.

Unlike many CAR-T therapies, Acepodia doesn’t genetically engineer patient cells to express cancer-fighting antibodies. Instead, the company uses a proprietary line of NK cells selected for their potency. These cells have their killer cell Ig-like (KIR) receptors selected to work with a variety of patients, and express receptor profiles that they believe make for more potent effector cells. 

According to Sonny Hsiao, PhD, founder and CEO of Acepodia, the ACC technology can attach 100,00 antibodies to NK cells which will induce it to eradicate tumours. He added that this approach is much safer than the genetic engineering approach.

(Source: Gen Eng News, 2020)