Biotech Investor

In the RUBY study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2 in autologous stem cells to treat sickle cell disease. Full study results: nejm.org/doi/full/10.10… Editorial: More Options for Gene Editing in Hemoglobinopathies nejm.org/doi/full/10.10… Further reading: 📄 Original Article: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease (BEACON study) nejm.org/doi/full/10.10… 📄 Original Article: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia (EdiThal study) nejm.org/doi/full/10.10…

In a study by Gori and colleagues, gene editing with a prime editor was used to treat two persons with chronic granulomatous disease caused by a small deletion in the gene NCF1. Full study results: nejm.org/doi/full/10.10… Editorial: Genetic Medicine — Primed and Ready nejm.org/doi/full/10.10… #Hematology

@Colecalciferol1 They just need to sell their patent and retire. They already cashed out enough money and on they do not know how to manage it $HUMA Humacyte

$HUMA - Immediate action plan Humacyte (1/12) Cc: @MichaelSen, @Fresenius, @FME_Global, @FreseniusKabi, @humacyte Humacyte is dying. Humacyte is not Lauras little biolab anymore. Humacyte is facing a cash drain of USD 20-30m per quarter, with R&D spending of c. USD 70m per year. Humacyte is stumbling. We, a group of investors with sizable investments, are requesting immediate action by Humacyte to ensure survival, and preserve the healthy core for trauma patients and potentially AV access, and to transform the company towards a Trauma-only production company; we expect the following, immediate action plan to be implemented by the management team (with details in thread): - Immediate closure of non-core programmes - Ramp down of production capacity (max. 5k) - Immediate termination of any employees not related to trauma / AV (until V012) - Disposal of non-core IP / any programmes not associated with trauma / AV - Potentially via separate entity - Management downsizing / re-allocation of responsibilities - Immediate price adjustment to ramp up market penetration (USD 9,999) - Review of North Carolina facilities - sub-lease any non-core space - legal review of lease agreement - Downsizing of Board of Directors - specifically Brady W. Dougan - Renegotiation of Avenue Capital credit facility to unlock conditional tranches - Increased spend on lobbying (USD 1-2m+) - Expected value accretion (50-300% upside)

Eli Lilly to sign $2bn deal for AI drug development with Hong Kong biotech ft.trib.al/lRujYfm