Our Q3 2024 gene therapy review underscores a surge in R&D partnerships and venture funding, with crucial deals driving innovation across critical therapeutic areas. ImmuneOnco granted Instil Bio global rights for IMM-2510 and IMM-27M (solid tumors), with $10 million upfront, up to $40 million in near-term milestones, $270 million in development, $1.8 billion in commercial milestones, and royalties. YolTech partnered with Salubris to develop YOLT-101 (PCSK9 gene therapy for hypercholesterolemia) in China, with $29 million in development and $116 million in commercialization milestones, plus royalties. Passage Bio granted Gemma Biotherapeutics global rights for gene therapies targeting GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy, securing $10 million upfront and up to $114 million in milestones. Genevant partnered with Repair Therapeutics to develop mRNA-LNP therapies for atherosclerosis, potentially worth $107 million per product, plus royalties. Pantherna Therapeutics entered a research partnership with Bayer for advanced LNP technology for mRNA therapies targeting organs beyond the liver.
In ventures, Beacon Therapeutics raised $170 million in series B to advance gene therapies for X-Linked Retinitis Pigmentosa (XLRP) and Dry Age-related Macular Degeneration (dAMD). Genespire secured $52 million to launch a Phase I/II trial for GENE202 (methylmalonic acidemia) and develop its pipeline. Vironexis Biotherapeutics raised $26 million to support its cancer gene therapy pipeline, including FDA fast-track and rare pediatric disease tags. Amlogenyx, an Ultragenyx subsidiary, closed a $14 million seed round for Alzheimer’s and amyloid-related gene therapies. Bio-Sourcing secured $11 million to advance its BioMilk platform using goats to produce monoclonal antibodies. Details to follow.
Gene Therapy & Vectors R&D Partnerships
In Q3 2024, gene therapy and vectors experienced significant growth in R&D partnerships, with 7 deals valued at $2.5 billion. Notably, none of these deals involved upfront cash or equity, marking a shift from Q2 2024, which saw no activity. Across both quarters, there were 7 partnerships and licensing agreements, maintaining the $2.5 billion deal value, all without upfront financial exchanges.
Gene Tx & Vectors Median Upfront Cash & Equity
BioPharma Median Upfront Cash & Eq., Top Modalities – 2019-24 YTD
 Top 5 Gene Therapy & Vectors R&D Partnerships – Q3 2024
ImmuneOnco development and commercialization deal with Instil Bio – August 2024
ImmuneOnco granted Instil Bio exclusive rights (excluding Greater China) to develop and commercialize IMM-2510 (PD-L1xVEGF bispecific antibody) and IMM-27M (anti-CTLA-4 antibody) for solid tumors. ImmuneOnco will receive $10 million upfront, up to $40 million in near-term milestones, $270 million in development milestones, and $1.8 billion in commercial milestones, plus royalties.
YolTech development and commercialization deal with Salubris – August 2024
YolTech granted Salubris exclusive rights to develop YOLT-101, a PCSK9-targeting gene therapy for hypercholesterolemia in China. YolTech is eligible for up to $29 million in development milestones, $116 million in commercial milestones, and tiered royalties.
Passage Bio development and commercialization deal with Gemma Biotherapeutics – July 2024
Passage Bio granted Gemma Biotherapeutics global rights to develop gene therapies for GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy. Passage Bio will receive $10 million upfront, $10 million for business milestones, and up to $114 million in additional milestones, plus single-digit royalties.
Genevant development and commercialization deal with Repair – September 2024
Genevant granted Repair Therapeutics nonexclusive global rights to develop mRNA-LNP therapies for atherosclerosis, combining Repair’s Cholesterol Degrading Platform with Genevant’s LNP technology. Genevant is eligible for up to $107 million per product, plus royalties in the mid to high single digits.
Pantherna Therapeutics research partnership with Bayer – September 2024
Pantherna and Bayer partnered to develop advanced LNP technology for mRNA therapies targeting organs beyond the liver. The collaboration aims to improve therapeutic efficacy and scalability. Financials were not disclosed.
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 Gene Therapy & Vectors Venture Activity
Venture activity in gene therapy and vectors surged in Q3 2024, with 6 funding rounds raising $273 million. A sharp rise from Q2 2024, which saw just 2 rounds raising $64 million. The sector has raised $337 million across 8 funding rounds over the past two quarters.
Top 5 Gene Therapy & Vectors Venture – Q3 2024
Beacon Therapeutics – Series B – $170M – July 2024
Beacon Therapeutics raised $170 million in series B, led by Forbion, with participation from Syncona, Oxford Science Enterprises, TCGX, and Advent Life Sciences. The funds will advance AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and support Phase 1/2 trials for Dry Age-related Macular Degeneration (dAMD).
Genespire – Series B – $52M – September 2024
Genespire raised $52 million (€46.6 million) in series B funding to launch a Phase I/II trial for GENE202, targeting methylmalonic acidemia, and support additional gene therapy research. Investors include Sofinnova Partners, XGEN Venture, and CDP Venture Capital.
Vironexis – Seed – $26M – September 2024
Vironexis Biotherapeutics raised $26 million in seed funding, led by Drive Capital and Future Ventures. The funds will advance a gene therapy that has FDA fast-track and rare pediatric disease tags. The company has a pipeline of over 10 preclinical candidates targeting cancers.
Amlogenyx – Seed – $14M – August 2024
Amlogenyx Inc., a subsidiary of Ultragenyx Pharmaceutical, has completed a $14 million seed funding round. The company is focused on developing innovative gene therapies for Alzheimer’s disease and other amyloid-related disorders. This funding will support Amlogenyx’s research efforts to advance treatments targeting these challenging diseases.
Bio-Sourcing – Private – $11.1M – September 2024
Belgian biotech company Bio-Sourcing secured €12.5 million ($11 million) from the European Innovation Council (EIC) Accelerator. The funding, consisting of €2.5 million in grants and €10 million in equity, will support the development of Bio-Sourcing’s BioMilk platform. This innovative platform uses goats to produce monoclonal antibodies in their milk, offering a more cost-effective and eco-friendlier alternative to traditional methods.
Also make sure to also check out Gene Therapy R&D Partnerships, M&A, and Ventures – H1 2024