The rapid migration of gene editing technologies from the bench to the clinic has opened up new therapeutic possibilities for patients with previously intractable genetic diseases and difficult-to-treat cancers. But mobilizing gene editing components into a target cell or organ remains a critical step for the field. Integra Therapeutics SL, an early stage Spanish firm, is now engaged in that process with a novel gene writing platform.
Galimedix Therapeutics Inc. plans to move its lead compound, GAL-101, into a phase IIa proof-of-concept trial next year, in order to test an intriguing hypothesis associated with certain retinal degeneration conditions. GAL-101 selectively binds misfolded amyloid beta species, and the company believes that this mechanism may benefit patients with the dry form of age-related macular degeneration or with glaucoma.
For many multiple sclerosis patients, the approval over the past 30 years of a lengthy list of immunomodulatory therapies has helped to reduce the frequency of relapses and to slow disease progression. However, there has been little parallel progress in the development of remyelination therapies, to tackle the other key pathophysiological dimension of the disease. Patients still have no therapies that can help to repair at least some of the damage that results from flare-ups, and the resulting neuronal loss contributes to further disease progression and disability. Rewind Therapeutics NV, of Leuven, Belgium, is one of a small clutch of firms attempting to tackle this problem.
Rapport Therapeutics Inc. raised $150 million in a series B round, less than six months after unveiling its $100 million series A investment. It’s further evidence that its distinctive precision neuromedicine approach has gained traction with some deep-pocketed investors and adds further momentum both to its clinical development and its discovery research efforts.
Shares in Galecto Inc. (NASDAQ:GLTO) plummeted by 71%, closing Aug. 15 at 67 cents, on news that its lead drug candidate, GB-0139, flamed out in a phase IIb trial in idiopathic pulmonary fibrosis (IPF). The inhaled galectin-3 inhibitor actually performed considerably worse than placebo in the 52-week placebo-controlled study, the endpoint of which was the annual rate of decline from baseline in forced vital capacity (FVC).
Nuclidium AG, an early stage radiopharmaceuticals and radiodiagnostics developer, aims to bypass the production constraints that have hampered other firms in the field by employing copper radioisotopes for both therapeutic and imaging purposes.
Tenpoint Therapeutics Ltd. raised $70 million in a series A funding round to pursue ambitious plans to reverse vision loss using both ex vivo cell engineering and in vivo cell reprogramming approaches.
Tolerogenixx GmbH raised €7 million (US$7.6 million) in an extension to its series A round, which will enable it to continue phase IIb development of a cell therapy that induces donor-specific immune tolerance in kidney transplant recipients. At the same time, the company disclosed five-year follow-up data from a phase Ib trial of the therapy, which demonstrated that recipients continue to have stable graft function and to avoid acute rejection and severe opportunistic infections while on a reduced regimen of immunosuppressive drugs.