Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing American sickle cell disease patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies. The therapies are approved for those 12 and older, and both are made from a patient’s own blood stem cells, which are then modified and returned via a single-dose infusion. Prior to the infusion, patients must undergo myeloablative conditioning, or high-dose chemotherapy, in order to clear out the mutated cells. Casgevy represents the first approved treatment in the country that uses the novel genome editing technology, CRISPR/Cas9, from Crispr Therapeutics AG.

Positive phase II results in early breast cancer send Intensity shares on a wild ride

Phase II data from the study in early-stage breast cancer called Invincible blasted upward the shares of Intensity Therapeutics Inc. (NASDAQ:INTS), which were trading midday at $9.92, up $5.72, or 136%. The data with INT230-6 emerged during the San Antonio Breast Cancer Symposium, where attendees heard that the treatment – a combination of cisplatin and vinblastine, injected directly into the tumor – turned up a systemic increase in the median diversity of T-cell repertoire in patients' blood compared to baseline that was also much larger than a control saline injection. Shelton, Conn.-based Intensity is planning a phase III program in metastatic sarcoma with the drug as a monotherapy, and has in the works a phase II/III effort to test INT230-6 in combination with standard of care neoadjuvant therapy.

Biopharma funding hits $5.65B in November, with 18% increase YTD

In November, the biopharma industry raised $5.65 billion in total financings. While down from $11.6 billion tracked by BioWorld in October, overall financings are tracking ahead of last year. Biopharma funding has maintained an average of $6.1 billion per month in 2023, surpassing the $5.07 billion monthly average throughout 2022. Cumulatively, biopharmas raised $66.69 billion through November 2023, showing 17.91% growth compared to the $56.56 billion recorded in the comparable period in 2022. Value remains down, however, from the levels seen in 2020 and 2021.

Kamau Therapeutics emerges from stealth with Graphite Bio’s nula-cel

Following a strategic transaction with Graphite Bio Inc., Kamau Therapeutics is emerging from stealth with sickle cell treatment nulabeglogene autogedtemcel (nula-cel). Kamau received an option to acquire all of Graphite’s genome editing assets, including a platform technology that integrates precision DNA repair using homology directed repair and CRISPR/Cas9, as well as nula-cel, which offers a potential cure for sickle cell disease derived from the patient's cells.

Tessellate Bio draws $8.4M in seed for its synthetic lethality approach

With an initial €8 million (US$8.4 million) in seed funding in the bank, Tessellate Bio has emerged from stealth to tackle cancers that rely on the less well explored synthetic lethality mechanism of alternative lengthening of telomeres (ALT). Based in the Netherlands, the company was created in the middle of the COVID-19 pandemic, in 2020, after Tessellate’s co-founders and researchers Hilda Pickett and Claus Azzalin discovered an enzyme that is critical to controlling the ALT process – the method by which tumor cells maintain telomeres long enough that they can continue dividing and causing disease. “And that’s also our lead program, called FANCM, or the FANCM complex,” Andree Blaukat, Tessellate’s CEO told BioWorld in an interview.

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Abvc, Altrubio, Anixa, Artbio, Aura, Biosenic, Brainstorm Cell, Bristol Myers Squibb, Faron, Hamlet, Hansa, Harmony, Ipsen, Korro, Kymera, Lobe Sciences, Mc2, Merck, Nextpoint, Nikang, Oculis, Proqr, Puretech, Regeneron, Rigel, Sanofi, Sosei, Spago, Spybiotech, Spyre, Théa, Treadwell, Vesper, Vittoria, Wave, Xilio