The trajectory of Rocket Pharmaceuticals, a clinical-stage gene therapy company, has been marked by a series of impressive milestones. At least partial credit for those accomplishments goes to CEO Gaurav Shah, who co-founded the company in 2015 and has combined scientific, business and managerial savvy to progress the company through two regulatory filings this year. Shah recently spoke to In Vivo about the company and his own leadership style.

Partnered with AbbVie on a wet AMD gene therapy, Regenxbio is also advancing wholly owned candidates for Duchenne muscular dystrophy and MPS I and MPS II.

The California-based biotech is reducing its workforce to help finance a Phase II study of Ixo-vec, its eye gene therapy which has been blighted by safety concerns.

Companies such as Novartis, Roche or Takeda are heavily invested in the advanced therapeutics field, usually spreading their bets across multiple modalities. The remaining challenges offer opportunities for the next wave of innovation to transform the field once again and make a dent in the larger market of common diseases and solid tumors.