'Lack of good CDMOs’ factor for Krystal’s $90m gene therapy plant

Krystal Biotech has broken ground on a commercial gene therapy manufacturing facility in Findlay Township, Pennsylvania.

The 100,000 square-foot ‘ASTRA’ facility, set to create 75 jobs in the Pennsylvanian town, is expected to open in late 2021 to support Krystal Biotech’s gene therapy pipeline.

Krystal is developing several ‘off-the-shelf’ gene therapies for rare skin diseases using its Skin TARgeted Delivery (STAR-D) platform consisting of an engineered HSV-1 vector and skin optimized gene transfer technology.

Image: iStock/klenger

Lead candidate B-VEC (previously known as KB103) is targeting Dystrophic epidermolysis bullosa (DEB), a potentially fatal skin blistering condition caused by a lack of collagen protein, currently in Phase I/II trials.

The investment of between $70 million and $90 million in the ASTRA facility is an important part of the firm’s strategy to grow its inhouse capabilities.

According to a Krystal spokesperson, the main reasons for this are to protect Krystal’s intellectual property, and to reduce the firm’s reliance on contract development and manufacturing organizations (CDMOs).

Presently, the sector is beset by “high costs and time delays caused by the imbalance between too many gene therapy companies and too few good CDMOs,” we were told.

Lack of capacity

The comments reflect concerns in the gene therapy space, with the lack of viral vector production capacity slowing down development in the field and putting the cost of securing space at a CDMO at a premium.

Krystal joins a growing number of firms opting to build their own facilities to overcome the capacity shortage.

Pfizer has cited an internal network as a competitive advantage in the field, while Astellas stated a large-scale gene therapy plant was a key factor in its $3 billion acquisition of Audentes late last year.

Meanwhile, the lack of capacity has driven M&A activity in the CDMO space, and also led to opportunities for outside players to enter the potentially lucrative field.