DealForma’s review of gene therapy shows that during 2024 gene therapy attracted a significant share across R&D partnerships, M&A, and venture funding, with a focus on CNS, oncology, ophthalmology, renal, and rare diseases. Key R&D deals included AviadoBio’s October option deal with Astellas for AVB-101 in FTD ($2.2 billion), ImmuneOnco’s August bispecific antibody licensing to Instil Bio for solid tumors ($2.1 billion), and Sangamo’s December AAV capsid licensing to Astellas for neurological targets ($1.3 billion).
On the M&A front, Novartis acquired Kate Therapeutics in November (up to $1.1 billion) for muscular dystrophy and cardiomyopathy assets; Ocuphire merged with Opus Genetics in October (pro forma cash: $37 million) for a seven-program ophthalmic pipeline; and NAYA Biosciences acquired Florida Biotechnologies in January (up to $25 million in stock) for a Phase II LHON therapy.
Venture activity included Beacon’s $170 million series B (July) for XLRP and dAMD, Purespring’s $105 million (July) for kidney gene therapies, and Interius BioTherapeutics’ $67 million to advance its in vivo CAR-T program for B-cell malignancies.
Gene Therapy R&D Partnerships
In 2024, gene therapy R&D partnerships declined, with 13 deals totaling $6 billion and just $100 million in upfront payments, down from 20 deals worth $7.9 billion and $400 million upfront in 2023. Across 2 years, 33 deals closed $13.9 billion.
Â
Top Gene Therapy R&D Partnerships in 2024
AviadoBio license option deal with Astellas – October 2024
AviadoBio granted Astellas an exclusive global option to develop and commercialize AVB-101, an AAV-based gene therapy targeting frontotemporal dementia (FTD) with progranulin mutations. Signed at Phase I, following initial patient dosing in the ASPIRE-FTD trial, the deal includes $30 million in upfront cash and $20 million in equity. AviadoBio is eligible for up to $2.2 billion in potential milestone and license payments, plus royalties.
ImmuneOnco development and commercialization deal with Instil Bio – August 2024
ImmuneOnco granted Instil Bio exclusive global rights (excluding Greater China) to develop and commercialize two cancer therapies: IMM-2510, a PD-L1xVEGF bispecific antibody, and IMM-27M, an anti-CTLA-4 antibody, both targeting solid tumors. The agreement was signed at the Phase I stage. ImmuneOnco will receive $10 million upfront and is eligible for up to $40 million in near-term milestones, $270 million in development and regulatory milestones, and up to $1.8 billion in commercial milestones, along with royalties.
Sangamo development and commercialization deal with Astellas – December 2024
Sangamo granted Astellas exclusive global rights to develop and commercialize gene therapies for neurological diseases using its STAC-BBB neurotropic AAV capsid platform. The deal, signed at the discovery stage, includes technology transfer by Sangamo, while Astellas will lead all R&D and commercialization efforts. Astellas also holds the option to add up to four more targets. Sangamo will receive $20 million upfront and is eligible for up to $1.3 billion in milestone and target fees, plus tiered royalties.
Gene Therapy M&A
2024 gene therapy M&A activity decelerated, with 5 deals worth $1.2 billion (including contingents) and just $100 million in upfront cash. This contrasts with 2023’s 9 deals totaling $2.1 billion, including $1.6 billion in non-contingent payments. Over 2 years, 14 deals reached $3.3 billion in value, with $1.7 billion in upfront cash.
Top Gene Therapy M&A in 2024
Novartis acquiring Kate Therapeutics – November 2024
Novartis announced the acquisition of Kate Therapeutics, a preclinical-stage company developing AAV-based gene therapies for neuromuscular and cardiovascular disorders. Kate’s pipeline includes KT-430 for X-linked myotubular myopathy, KT-809 for Duchenne muscular dystrophy, and four discovery programs targeting various forms of muscular dystrophy and cardiomyopathy. The deal includes two proprietary platforms, Deliver and Cargo, and is valued at up to $1.1 billion, comprising upfront cash and milestone payments.
Ocuphire Pharma acquiring Opus Genetics – October 2024
Ocuphire Pharma acquired Opus Genetics in an all-stock deal and will rename the combined entity Opus Genetics, trading on Nasdaq as IRD, from October. The deal adds seven gene therapy programs focused on inherited retinal diseases, including two Phase I/II candidates—OPGx-LCA5 (Leber congenital amaurosis; data expected Q3 2025) and OPGx-BEST1 (Best disease), and five preclinical assets for retinitis pigmentosa and related conditions. Opus will receive 5,237,063 common shares and 14,145 series A preferred shares (convertible to 14.1M common shares), with some subject to a 180-day lockup. Post-deal ownership: 58% Ocuphire, 42% Opus. Pro forma cash: $37 million. Deal value is based on disclosed equity terms; no milestones included.
NAYA Biosciences acquiring Florida Biotechnologies – January 2024
NAYA Biosciences acquired Florida Biotechnologies, gaining a Phase II gene therapy candidate for Leber’s Hereditary Optic Neuropathy (LHON). Florida will receive $20 million upfront in NAYA stock (4 million shares at $5/share) and is eligible for up to $5 million more (1 million shares) tied to milestone achievements.
Gene Therapy Ventures
In 2024, gene therapy ventures raised $773 million across 20 funding rounds, marking a decline compared to 2023’s $1.4 billion from 21 rounds. Altogether, these two years saw 41 rounds and $2.2 billion raised in venture capital, underlining fewer deals and lower funding.
Top Gene Therapy Venture Funding in 2024
Beacon Therapeutics – Series B – $170M – July 2024
Beacon Therapeutics raised $170 million in a series B led by Forbion, with participation from Syncona, Oxford Science Enterprises, TCGX, and Advent Life Sciences. The funding will advance clinical development of its lead gene therapy candidate, laru-zova (AGTC-501), for X-linked Retinitis Pigmentosa (XLRP), and support early-stage trials for its Dry Age-related Macular Degeneration (dAMD) program. Beacon also announced new board appointments as part of the financing.
Purespring Therapeutics – Series B – $105M – October 2024
Purespring Therapeutics raised £80 million ($105 million) in an oversubscribed series B led by Sofinnova Partners, with participation from Gilde Healthcare, Forbion, British Patient Capital, and Syncona. The funds will advance its AAV-based gene therapy pipeline targeting kidney diseases, including lead candidate PS-002 for IgA Nephropathy (IgAN), set to enter Phase I/II trials. Purespring’s platform targets podocytes cells involved in 60% of renal diseases, offering a novel, disease-modifying approach for conditions like nephrotic syndrome and other glomerular diseases.
Interius BioTherapeutics – Series B – $67M – August 2024
Interius BioTherapeutics, a Penn Medicine spinout, raised $67 million to advance its lead in vivo gene therapy candidate, INT2104. The first patient was dosed in the Phase I INVISE trial, evaluating INT2104, a first-in-class therapy that delivers a CAR transgene directly in vivo to generate CAR-T and CAR-NK cells targeting CD20-positive B-cell malignancies. This approach may eliminate the need for ex vivo cell engineering or chemotherapy.
Also check out Autoimmune R&D Partnerships, M&A, Ventures, and IPOs in 2024
