Abeona advances. US company Abeona Therapeutics has enjoyed a double helping of good news. First, the company’s EB-101 cell therapy for “butterfly disease” (recessive dystrophic epidermolysis bullosa) has bounced back from a 2019 FDA clinical hold to present phase III evidence of meeting co-primary endpoints of the VIITAL study (achieving 50 percent wound healing and reduced pain). Second, Abeona has raked in $35 million through sales of shares, which will be used to keep the company running through to late 2024. Currently, patients with the dystrophic form of epidermolysis bullosa have a life expectancy of 30 years.

Relapse/recovery. New findings in New York, by way of a joint project between Mount Sinai and the Memorial Sloan Kettering Cancer Center, have shed new light on options for treating patients who suffer a cancer relapse after receiving CAR T-cell therapy, and then have no strong options to turn to. The researchers applied a range of different therapies to relapsed patients who had received BCMA-directed CAR T, and found that treatments that engaged with T cells worked best against the cancers for the longest period of time. These treatments included bispecific antibodies and other forms of CAR T-cell therapy.

Relapse as a material question. A study from the Children’s Hospital of Philadelphia offers insights into how poverty affects American children treated with CD19 CAR T therapy. The researchers found that poverty had no significant effect on complete remission or hazard of death rates, but did find that patients from poor neighborhoods are at greater risk of relapse than their wealthier counterparts. The team has recommended further investigation of multicenter outcomes and access disparities. According to Save the Children and The Center for American Progress, around one in seven children in the US live in poverty – with the worst hit groups being black and latin minorities, and families living in rural areas.

Fine then! A recent decision by the US Supreme Court has dealt good news to Gilead, and bad news to Bristol Myers- Squibb. Just over one year ago, a federal appeals court overturned a pro-BMS verdict, scuppering the company’s attempt (through BMS’ subsidiary, Juno) to sue Gilead (through Gilead’s subsidiary, Kite) for $1.2 billion for (allegedly) copying its own patented CAR T therapy to help create and commercialize Gilead’s Yescarta. Since then, the case climbed the American legal ladder all the way to the Supreme Court, who simply declined to hear it. To use the Court’s own six words: “The appeal is dismissed as moot.”

$487 million deal sees Eli Lilly purchase inner ear gene therapy specialists Akouos, acquiring its portfolio of A AVdelivered therapies

Galapagos drops 200 staff, kidney disease program, and remnants of fibrosis program; aims to focus on CAR T for immunology and oncology

Charles River announces plans to expand in Memphis’ Bluff City; will add nine cell therapy processing suites, bringing total to 23

Mouse research in Rice University, Texas, shows immune profiling of AAV response can identify B cellspecific targets that enable vector re-administration with minimized immunosuppression

Small data sample from Intellia first-in-hunan study indicates that gene editing candidate NTLA-2002 may serve as functional cure for hereditary angioedema