Our H1 2024 review of the gene therapy sector explores R&D partnerships, M&A activity, venture investments, and industry trends. In January, Voyager and Novartis entered a partnership with Voyager, receiving $80 million upfront and $20 million in equity to develop gene therapies for Huntington’s disease and spinal muscular atrophy, with potential milestones of up to $1.2 billion. That same month, GenEdit granted Genentech exclusive rights to develop nucleic acid-based therapies for autoimmune diseases, securing $15 million upfront and up to $629 million in milestones. Florabio and NewBiologix inked a deal to develop AAV vectors in February, though financials were undisclosed. May saw MilliporeSigma acquiring Mirus Bio for $600 million upfront, acquiring critical gene therapy assets. Also in January, NAYA Biosciences acquired Florida Biotechnologies for $20 million or 4 million shares, focusing on a phase II therapy for Leber’s Hereditary Optic Neuropathy. Beacon Therapeutics raised $170 million in series B in July to support programs for X-linked retinitis Pigmentosa and Dry Age-related Macular Degeneration. In May, Latus Bio secured $54 million in series A to develop therapies for central nervous system disorders, particularly CLN2 disease. Finally, Asgard Therapeutics raised $33 million in series A in March to advance its cancer immunotherapy program, AT-108, to IND readiness by 2026.
Gene Therapy & Vectors R&DÂ Partnerships
During H1 2024, the gene therapy sector witnessed 4 R&D partnerships valued at $1.9 billion, with upfront payments and equity amounting to $100 million. This is a significant decline compared to 2023 when 25 deals totaled $9.1 billion and $300 million in upfront cash and equity. H1 2024 accounted for 29 deals, generating $11.0 billion in value and $400 million in upfront payments and equity.
Top Gene Therapy R&D Partnerships H1 2024
Voyager and Novartis – January 2024
Voyager granted Novartis non-exclusive, worldwide rights to develop gene therapies using Voyager’s TRACER capsids for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Voyager will receive $80 million in upfront cash and $20 million in equity by issuing 2,145,002 common shares. For SMA, Voyager is eligible for up to $200 million in development and regulatory milestones and up to $400 million in sales milestones. For HD, the eligibility includes up to $225 million in development and regulatory milestones and up to $375 million in sales milestones.
GenEdit and Genentech Forge Strategic Partnership – January 2024
GenEdit granted Genentech exclusive, worldwide rights to develop and commercialize nucleic acid-based therapies for autoimmune diseases using GenEdit’s NanoGalaxy platform and hydrophilic nanoparticles (HNPs). GenEdit will receive $15 million upfront and is eligible for up to $629 million in development and commercial milestones, plus tiered royalties.
Florabio and NewBiologix Ink AAV Cell Line Deal – February 2024
Florabio granted NewBiologix exclusive, worldwide rights to develop and commercialize recombinant adeno-associated virus (AAV) vectors for gene therapies using Florabio’s HEK293 cell line. Financials were not disclosed.
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Gene Therapy & Vectors M&A
During H1 2024, M&A in gene therapy and vectors saw 3 deals totaling $7.4 billion, including $6.7 billion in non-contingent cash. By comparison, 2023 had one less at 2 with no cash contributions. This brings the total to 5 worth $7.4 billion, with $6.8 billion in non-contingent cash.
Top Gene Therapy M&As H1 2024
MilliporeSigma acquiring Mirus Bio – May 2024
MilliporeSigma is acquiring Mirus Bio for $600 million upfront. Mirus Bio’s critical assets include VirusGEN transfection reagents, RevIT AAV titer enhancer, VirusGEN AAV transfection kits, VirusGEN LV transfection kits, VirusGEN GMP products, TransIT-X2 dynamic delivery system, TransIT-mRNA transfection kit, Ingenio electroporation solution, and the VirusGEN platform.
NAYA Biosciences acquiring Florida Biotechnologies – January 2024
NAYA Biosciences is acquiring Florida Biotechnologies, whose lead asset is a phase II gene therapy for Leber’s Hereditary Optic Neuropathy (LHON). Florida Biotechnologies will receive $20 million upfront, or 4,000,000 shares of NAYA’s common stock at $5 per share and is eligible for up to $5 million or 1,000,000 shares upon achieving certain milestones.
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Gene Therapy & Vectors, All Ventures
In H1 2024, gene therapy and vectors raised $100 million through 4 venture rounds, a sharp decline from 2023, which saw 21 rounds raising $1.6 billion. Overall, 25 rounds raised $1.7 billion, highlighting a significant drop in venture funding in H1 2024.
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Top Gene Therapy Ventures H1 2024
Beacon Therapeutics – Series B – $170M – July 2024
Beacon Therapeutics, a London-based ophthalmic gene therapy company, raised $170 million in series B led by Forbion, with participation from Syncona Limited, Oxford Science Enterprises, University of Oxford, TCGX, and Advent Life Sciences. The funds will support AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and generate phase 1/2 clinical trial data for the Dry Age-related Macular Degeneration (dAMD) program.
Latus Bio – Series A – $54M – May 2024
Based in Philadelphia, PA, Latus Bio secured $54 million in series A, led by 8VC and DCVC Bio. Partners include Samsung Life Science Fund, The Children’s Hospital of Philadelphia Foundation, Benjamin Franklin Technology Partners, Modi Ventures, and Gaingels. The funds will accelerate the development of gene therapy candidates for central nervous system disorders, focusing on CLN2 disease. First-in-human dosing is planned for late 2025.
Asgard Therapeutics – Series A – $32.6M – March 2024
Asgard Therapeutics, specializing in vivo direct cell reprogramming for cancer immunotherapy, raised €30 million ($33M) in series A co-led by RV Invest and Johnson & Johnson Innovation – JJDC, Inc., with participation from Novo Holdings, Boehringer Ingelheim Venture Fund, and Industrifonden. The funds will advance the lead program AT-108 to IND readiness by 2026.
