Gene Therapies

A three-month FDA delay for a new LEQEMBI injection highlights the high-stakes race for a convenient Alzheimer's therapy and its complex safety hurdles.

Facing financial pressures, the biotech divests a key cancer program, repays debt, and now explores all options to maximize value for its remaining pipeline.

With a strong balance sheet and promising data, Enliven Therapeutics is pushing its lead leukemia drug, ELVN-001, into a pivotal late-stage trial.

With key clinical data on the horizon for its cancer and ALS drugs, LeonaBio adds deep financial, operational, and strategic expertise to its leadership.

The architect of HUMIRA's launch and Horizon's $28B sale is now shaping the next wave of drug companies from within private equity. What does it mean?

As EyePoint's losses widen to fund its lead drug, DURAVYU, the company is on the cusp of pivotal trial results that could redefine retinal disease treatment.

Preclinical data for CITY-RBP4 shows promise in treating Stargardt disease, a leading cause of juvenile blindness with no approved therapies.

A new report reveals a dramatic shift in Alzheimer's drug development, with 75% of trials now targeting pathways beyond amyloid for a new age of treatment.

A German biotech's novel in vivo CAR T platform uses DNA for durable tumor control, aiming to make advanced cancer therapy an off-the-shelf medicine.

The clinical-stage biotech adds seasoned veterans from Eli Lilly and Iovance to accelerate its promising DNA Damage Response pipeline toward commercialization.

Valitor's VLTR-559 shows superior durability in preclinical data, potentially slashing wet AMD treatment from monthly injections to just two per year.

With $2.5M in seed funding, Hamilton's Stoked Bio is using a novel AI platform to tackle the world's toughest diseases, from superbugs to brain cancer.

Alkeus Pharmaceuticals will present crucial data on its oral drug for Stargardt disease, a leading cause of juvenile blindness with no current cure.

As life-saving gene therapies face billion-dollar payment hurdles, a key appointment signals a strategic shift from scientific breakthrough to market access.

TRexBio has dosed the first patient in a new trial for its novel drug, TRB-061, which aims to rebalance the immune system to treat atopic dermatitis.

A new stem cell therapy promises a one-time cure for hemophilia A, and the FDA is clearing the path. Could lifelong, costly infusions become a memory?

A new cell-free therapy, Ultrasome™, shows a 93% response rate in a pilot study, offering a scalable, regenerative solution for millions with knee pain.

Umoja Biopharma's UB-VV500 aims to revolutionize myeloma care by creating a dual-antigen CAR T therapy directly inside the patient's body.

A new study shows treating chronic Lyme disease reversed key Alzheimer's biomarkers, challenging medical guidelines and offering a new path for brain research.

RedHill Biopharma flips a capital deficit to positive equity, driven by savvy partnerships for its drugs Talicia and Bekinda and a promising R&D pipeline.