First Ever CRISPR Approval, $100M+ Raise in ALS, Neuromuscular & CF, and Alkermes to be CNS Exclusive.

Incredibly exciting news as Vertex and CRISPr Tx achieve the first ever approval of a CRISPR drug as the UK's MHRA give their positive decision on exa-cel today. ALS-biotech VectorYs victorious Series A also made the headlines with their spectacular €100+ Million raise, followed closely by NMD Pharma's Series B and ViaNautis' Series A. Other exciting regulatory wins as Atsena and Takeda receive RMAT and FDA approval for assets and in an interesting deviation from the current trend, Alkermes spins out its oncology assets to focus solely on neuroscience.

• Amsterdam-based VectorY raises a €129 Million ($138 Million) Series A, co-led by EQT Life Sciences and Forbion Growth Opportunities Fund. The biotech's lead asset, VTX-002, targets TDP-43 for the treatment of ALS. VectorY will use the financing to support the clinical development of its lead asset and the rest of its vectorised antibody programs for neurodegenerative diseases.
• NMD Pharma A/S raises €75 Million (~$80 Million) Series B, led by Jeito Capital. The biotech is developing small molecule inhibitors with the potential to restore muscle function in rare neuromuscular diseases. This Series B will be used to complete three Phase II studies with NMD670, the biotech's lead asset, for AChR and MuSK antibody positive myasthenia gravis, spinal muscular atrophy, and Charcot-Marie-Tooth disease.
• Vianautis closes $25 Million Series A destined to develop its polyNaut platform. The technology uses advanced polymer materials and in silico screening to guide genetic molecules to their primary targets. The biotech is focusing on cystic fibrosis treatments with an asset, VNS002, already in its pipeline.
• Gilde Healthcare closes new fund at €740 Million. Their Venture&Growth VI fund reached the hard cap after closing at €600 Million earlier this year. This fund will focus on fast-growth companies developing lower cost healthcare solutions.

Regulatory

• Vertex Pharmaceuticals and CRISPR Therapeutics achieve a world first as the UK MHRA organisation approves their asset exa-cel (Casgevy). This is the first-ever approved drug that is based on CRISPR gene editing and will be used to treat sickle cell disease and transfusion-dependent beta-thalassemia. The FDA will be making a decision on exa-cels approval by the 8th of December.
• Atsena Therapeutics gets FDA Regenerative Medicine Advanced Therapy (RMAT) designation for ATSN-101. The gene therapy targets GUCY2D-associated Leber Congenital Amaurosis (LCA1), an inherited retinal disease with no approved treatments.
• Takeda receives FDA approval for congenital thrombotic thrombocytopenic purpura (cTTP) asset. The recombinant enzyme replacement therapy is the first and only FDA-approved protein for cTTP that acts by replacing the ADAMTS13 enzyme.

Deals

• Alkermes spins out Mural Oncology in a complete separation of its oncology and neuroscience assets. The biotech emerges as a pure-play neuroscience company that will continue to focus exclusively on the development of neurological and psychiatric assets.
• Jazz Pharmaceuticals and GSK spinout Autifony Therapeutics enter into exclusive licensing deal. Potentially worth $770.5 Million, the partnership involves Autifony leading the preclinical development of two ion channel targets associated with neurological disorders. Jazz will lead clinical development, manufacturing, regulatory actions, and commercialisation.