With a new CEO at the helm, Gamida is closing in on an FDA approval, but a lack of cash could hobble its US launch efforts for allogeneic cell therapy product omidubicel.

The US biotech’s LOXL2 inhibitor candidate has reduced collagen fibrosis of the bone marrow in a small number of Phase II patients, helping validate a historically tricky approach attempted by few.

After slashing the prices of their blockbuster drugs to gain wider patient access through China's main reimbursement program, multinationals are now beginning to face increasing challenges from local competitors.

The company is seeking FDA accelerated approval of the closely watched gene therapy based on a surrogate marker and has proposed its fully enrolled Phase III trial to serve as a confirmatory study.

New data from anemic myelofibrosis patients showed the drug reduced transfusion dependence, which that could unlock a more sizeable portion of the market for CTI BioPharma.

As the first CRISPR-based therapies draw closer to approval, the Flagship-backed biotech believes its gene writing tech is the ‘definitive’ genetic medicine platform.

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 Dutch firm’s US filing for its first-in-class PI3K inhibitor has been accepted under priority review, bringing the asset closer to becoming the first disease-modifying option for a rare, genetic disorder.

Emerging Company Profile: US firm Peptilogics is advancing through the clinic with its lead orthopedic anti-infective drug candidate based on an AI platform that utilizes algorithms to improve peptide design.

Ponda Motsepe-Ditshego, who leads Amgen’s clinical trial diversity efforts, admits they are in early stages but noted progress internally and externally at better representing diverse patient groups.

GSK has poached its new chief financial officer from fashion brand Burberry, but Julie Brown’s experience at AstraZeneca and Roche will give her insights into how to lead GSK into its next phase.

Oversee returnee-founded Chinese biotech start-ups Nuwacell and Arnatar secure $14m in series A and seed rounds respectively, while four other major deals backed by private equity and venture capital firms raise a combined $116m.