RegulationFast Track (United States), PRIME (European Union), Regenerative Medicine Advanced Therapy (United States), Innovative Licensing and Access Pathway (United Kingdom)

Structure/Sequence

Sequence Code 1183790423

Source: *****

Clinical Trials associated with Ixoberogene soroparvovec

NCT06856577

/ RecruitingPhase 3

A Multi-Center, Randomized, Double-Masked, Active-Comparator-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (ARTEMIS)

This is a multi-center, randomized, double-masked, active-comparator-controlled, Phase 3 study in a broad participant population (treatment-naïve and treatment-experienced) with neovascular (wet) age-related macular degeneration (nAMD). The study will evaluate a single intravitreal (IVT) injection of Ixo-vec compared to an active comparator. The primary endpoint of this study is the mean change in best corrected visual acuity (BCVA) of Ixo-vec compared to an active comparator measured at an average of Weeks 52 and 56.
Safety, tolerability, and efficacy will be evaluated throughout the study.

Start Date28 Feb 2025

Sponsor / Collaborator

Adverum Biotechnologies, Inc.

NCT05536973

/ Active, not recruitingPhase 2

A Multi-Center, Randomized, Double-Masked Phase 2 Study to Assess Safety and Efficacy of ADVM-022 (AAV.7m8-aflibercept) in Anti-VEGF Treatment-Experienced Patients With Neovascular (Wet) Age-related Macular Degeneration (nAMD) [LUNA]

Neovascular or wet age-related macular degeneration (nAMD) is a degenerative ocular disease associated with the infiltration of abnormal blood vessels in the retina from the underlying choroid layer and is a leading cause of blindness in patients over 65 years of age. The abnormal angiogenic process in nAMD is stimulated and modulated by vascular endothelial growth factor (VEGF). Treatment of nAMD requires frequent intravitreal (IVT) injections of VEGF inhibitors (anti-VEGF) administered every 4-16 weeks. ADVM-022 (AAV.7m8-aflibercept) is a gene therapy product being developed for the treatment of nAMD and offers the potential for sustained intraocular expression of aflibercept following a single IVT injection. ADVM-022 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice.

Start Date23 Aug 2022

Sponsor / Collaborator

Adverum Biotechnologies, Inc.

NCT05607810

/ Active, not recruitingNot Applicable

A Long-Term Follow-Up Study of ADVM-022 in Diabetic Macular Edema- INFINITY Extension

This is a prospective, observational study designed to evaluate the long-term safety and tolerability of ADVM-022 in participants with diabetic macular edema (DME). Participants who previously participated in the INFINITY parent study and received a single unilateral intravitreal dose of ADVM-022 are eligible for enrollment upon completion of the end of study visit in the parent study.

Start Date10 Aug 2022

Sponsor / Collaborator

Adverum Biotechnologies, Inc.

100 Clinical Results associated with Ixoberogene soroparvovec

100 Translational Medicine associated with Ixoberogene soroparvovec

100 Patents (Medical) associated with Ixoberogene soroparvovec

Literatures (Medical) associated with Ixoberogene soroparvovec

04 Aug 2025·MOLECULAR PHARMACEUTICS

A Semimechanistic Ocular Pharmacokinetic Model for ADVM-022 Gene Therapy Describing the Dose-Exposure Relationship in Monkeys and the Scaling to Human

Article

Author: Hugi, Florian ; Renaud, Lionel ; Vollmer, Jannik ; Machacek, Matthias

Gene therapies are emerging as a new treatment modality. Due to their novelty, general pharmacological properties have yet to be established. For example, the translation from animal models to humans for first-in-human dose selection and the dose-exposure relationship remain poorly characterized. A mechanistic and quantitative framework would improve preclinical program design, enable more robust first-in-human dose predictions, and support more rigorous dose adjustments during clinical development. This study establishes a semimechanistic mathematical model for aflibercept expression and pharmacokinetics (PK) following intravitreal (IVT) ADVM-022 administration in monkeys and humans, drawing on the preclinical and clinical data presently available. ADVM-022 is an AAV2.7m8-based viral vector that delivers the gene encoding aflibercept, an antivascular endothelial growth factor (VEGF) fusion protein. It was developed as a gene therapy for treating wet age-related macular degeneration (wAMD) and is administered through a single IVT injection. The proposed model incorporates established ocular PK for intravitreally administered proteins, along with an expression component that links AAV dose to aflibercept production. Based on pooled PK data from monkey studies, the model suggests that transduction occurs not only in the retina but also in other ocular tissues bordering the vitreous, contributing to the observed intraocular aflibercept levels. Increasing doses within the lower range of preclinical studies (3 × 10¹⁰-2 × 10¹³ vg/eye) lead to increased transduction and expression, plateauing at upper limits of approximately 12.7 μg/day·cm³ for the retina, and 0.785 μg/day for extra-retinal tissues at higher doses. Assuming similar transduction efficiency between humans and monkeys, with adjustments for anatomical differences, the model provided predictions of ocular aflibercept concentrations that aligned with observations from the two dose groups in the phase 1 OPTIC clinical trial, supporting the utility of this approach.

01 Aug 2025·Graefe's Archive for Clinical and Experimental Ophthalmology

Gene therapy in neovascular age related macular degeneration: an update

Review

Author: Quesada, Erika ; Campos, Xiomara ; Rojas, Sofía ; Wu, Lihteh

Neovascular age-related macular degeneration (NV-AMD) is a leading cause of preventable blindness in the elderly. Intravitreal injections of anti-VEGF agents are currently the treatment of choice for NV-AMD. However this treatment is burdensome and fosters non-compliance which leads to inferior visual outcomes. Gene therapy has emerged as a promising therapeutic option for NV-AMD that may improve these outcomes. Potential risks of gene therapy include a potential immune response that may be elicited by the vector, accidental activation of oncogenes or inactivation of tumor suppresor genes leading to malignant transformation via insertational mutagenesis and integration of the viral DNA inserts into the host's DNA. The main strategy of current gene therapy for NV-AMD has focused on delivering transgenes that express anti-angiogenic proteins that directly or indirectly inhibit the VEGF pathway. Ixoberogene soroparvovec, RGX-314 and 4D-150 are the leading NV-AMD genetic treatment programs. Pre-clinical models suggest that genome surgery with clustered regularly interspaced short palindromic repeats (CRISPR) may be another option in the future.

01 Jul 2025·International ophthalmology clinics

Gene Therapy in Age-related Macular Degeneration

Review

Author: Zhou, Henry W ; Kim, Leo A

Recent advances in gene therapy and salient features of the current AMD therapeutic landscape have led to increased interest in applying gene therapy approaches to AMD. This review will discuss approaches to drug administration, viral and non-viral delivery vectors, and current trials in gene therapy for both wet and dry AMD. Drug administration routes include subretinal, intravitreal, and suprachoroidal approaches. Viral vectors include adenoviral, lentiviral, and adeno-associated viral (AAV) vectors. Non-viral vectors include lipid nanoparticle (LNP) and polymer-based vectors. Current trials in wet AMD include ADVM-022 and RGX-314. Current trials in dry AMD include GT-005 and JNJ-1887.

105

News (Medical) associated with Ixoberogene soroparvovec

27 Oct 2025

·www.pharmaceutical-technology.com

Eli Lilly buys Adverum in eye disease gene therapy punt

As part of the deal, Eli Lilly will acquire Adverum’s lead candidate ixo-vec, an intravitreal gene therapy for wAMD treatment. Credit: Tada Images via Shutterstock.com. Eli Lilly has agreed to acquire eye disease specialist Adverum Biotechnologies, bucking a recent trend of big pharma companies deciding to steer clear of the cell and gene therapy sector. Eli Lilly has offered Adverum $3.56 per share in cash, including an additional $8.91 in milestone payments. The latter depends on US approval of the biotech’s lead gene therapy candidate, ixo-vec, within seven years and achieving more than $1bn in annual global sales within ten years. This brings the total consideration to $12.47 a share, valuing the deal at a possible $261.7m. The share offer agreed on 24 October reflects a nearly 15% discount from the $4.18 closing price on 23 October. For Adverum, the potential buyout from Eli Lilly provides financial respite. The biotech has been struggling for cash in recent times – holding $44.4m to its name in July 2025. The lack of capital had increased jeopardy for ixo-vec, an intravitreal gene therapy that advanced into a Phase III trial (NCT06856577) for the treatment of wet age-related macular degeneration (wAMD) in March 2025. Indeed, Eli Lilly stated that without a $65m loan given to Adverum to continue ongoing clinical trials, the biotech would only be able to finance itself through October before having to wind down operations. Despite having to help fund ixo-vec’s development, which has been granted fast track and regenerative medicine advanced therapy (RMAT) designations by the US Food and Drug Administration (FDA), Eli Lilly could use the candidate to enter the lucrative wAMD market. The AMD sector, which also includes the dry form, is expected to reach $27.5bn across 7MM by 2031 (7MM: US, France, Germany, Italy, Spain, UK, and Japan), according to GlobalData analysis . GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence There is no gene therapy approved with a wAMD indication, with current treatments working via the anti–vascular endothelial growth factor (VEGF) mechanism, such as Regeneron’s blockbuster Eylea (aflibercept). The therapy is administered every four weeks for the first five months, followed by a single injection every two months. For Eli Lilly’s soon-to-be acquired ixo-vec, this could offer patients a one-and-done treatment. Lilly molecule discovery group vice-president Andrew Adams said: “Ixo-vec has the potential to transform wAMD treatment from a paradigm of chronic care with repeated intravitreal injections to a convenient one-time therapy.” Adverum CEO Laurent Fischer: “[Lilly’s] scientific depth and global reach offer the opportunity to accelerate our vision to deliver a transformative one-and-done therapy that can potentially restore and preserve vision for millions of patients living with wAMD.” Lilly bucks big pharma trend This is not the first time in 2025 that Eli Lilly has swooped in to rescue a cash-strapped biotech specialising in gene therapies. In April, the big pharma signed a licensing deal worth up to $1.4bn for Sangamo Therapeutics’ neurology-targeting gene therapy. However, Lilly’s recent deals, which includes a $1.3bn acquisition of RNA-based gene therapy developer Rznomics in May 2025, goes against the grain of big pharma generally opting to retreat from the cell and gene therapy sector. Earlier this month, Galapagos wound down its cell and gene therapy division after failing to sell the unit. Japanese pharma Takeda also abandoned its cell therapy research , pivoting instead towards small molecules, biologics and antibody-drug conjugates (ADCs). In addition, Gilead Sciences’ Kite Pharma terminated its cell therapy collaboration with Shoreline in September 2025, ending a research partnership valued at $2.3bn. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free eBook Navigating cell therapy process development The cell therapy manufacturing sector is rapidly evolving, driven by the need to balance innovative process development with rigorous GMP standards. This eBook, brought to you by industry leaders Cytiva, explores how biotech firms can address common challenges — including minimizing contamination risks from manual processes, overcoming resource limitations, and creating scalable, robust designs. Industry experts discuss strategies including automation, strategic outsourcing, and flexible equipment selection to streamline processes and reduce regulatory risks. To unlock expert insights on optimizing your cell therapy pathway from clinical development to commercialization, fill in your details now. Thank you. You will receive an email shortly. Please check your inbox to download the eBook. By Cytiva Thematic You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy . Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

AcquisitionGene TherapyPhase 3License out/inCell Therapy

24 Oct 2025

·www.fiercebiotech.com

Eli Lilly buys cash-strapped Adverum for its phase 3-stage eye disease gene therapy

Andrew Adams, Ph.D., Eli Lilly group vice president, molecule discovery, said ixo-vec \"has the potential to transform wAMD treatment.\"\n Just as Adverum’s cash reserves were running dry, Eli Lilly has swooped in to buy the biotech and its phase 3-stage eye disease gene therapy.The prize for Lilly is ixo-vec, an adeno-associated-virus-based gene therapy Adverum entered into a phase 3 study for wet age-related macular degeneration (wAMD) back in March. The biotech has been touting the intravitreal gene therapy as a potential one-and-done treatment that could “preserve sight for life.”But Adverum’s big plans for ixo-vec looked in doubt as the biotech faced its remaining $44.4 million in cash reserves held in July running dry this month. In fact, Lilly and Adverum explained in this morning’s release that if the deal falls through, Adverum’s remaining funds are only enough to finance the biotech for the final days of October and then wind down the business.Andrew Adams, Ph.D., Lilly group vice president, molecule discovery, said ixo-vec “has the potential to transform wAMD treatment from a paradigm of chronic care with repeated intravitreal injections to a convenient one-time therapy.”Under the proposed deal, Adverum’s stockholders will receive $3.56 per share of the biotech—slightly below Adverum’s closing price Thursday of $4.18—along with a nontransferable contingent value right (CVR). The CVR entitles the holder to receive up to a total of $8.91 cash if ixo-vec is approved within seven years and annual sales hit $1 billion within a decade.With an estimated 20.9 million Adverum shares outstanding, Fierce calculates that Lilly is spending around $74 million for Adverum, rising to $260 million if the CVRs are paid out in full.It’s not the first time Lilly has come to the rescue of a cash-strapped gene therapy company. Back in April, the Big Pharma threw struggling Sangamo Therapeutics an $18 million lifeline in return for using the biotech\'s neurotropic AAV capsid to develop a gene therapy for a central nervous system disease.“We are excited about the potential to join Lilly, with a proven track record in the discovery, development, and commercialization of innovative medicines for chronic and age-related conditions,” Adverum’s CEO Laurent Fischer, M.D., said in the Oct. 24 release. “We share Lilly\'s commitment to healthy aging and genetic medicines innovation,” Fischer added. “Their scientific depth and global reach offer the opportunity to accelerate our vision to deliver a transformative one-and-done therapy that can potentially restore and preserve vision for millions of patients living with wAMD.”Adverum had previously assessed the intravitreal injection in diabetic macular edema, an effort that was eventually discontinued after a patient went blind in the treated eye.It’s been a busy year for eye-related gene therapies. While Johnson & Johnson’s $130 million bet on an X-linked retinitis pigmentosa candidate suffered a phase 3 setback in May, the likes of Opus Genetics, MeiraGTx, Beacon Therapeutics and 4D Molecular Therapeutics have seen better news in the clinic.Meanwhile, the $135 million series B and Big Pharma backing secured by Stargardt disease-focused SpliceBio in June suggests investors can still be enthused by the potential for gene therapies to treat rare ocular diseases.

Phase 3Gene Therapy

24 Oct 2025

·endpoints.news

Lilly expands gene therapy work with Adverum acquisition

Eli Lilly is bolstering its gene therapy portfolio at a time when other large drugmakers are reassessing their work in the complex and commercially challenging field. The Indianapolis-based drug giant said Friday morning that it plans to acquire Adverum Biotechnologies and its Phase 3 candidate ixo-vec in a milestone-heavy deal. Perhaps in a sign of the gene therapy field’s recent struggles, Lilly is getting Adverum at a discount. Lilly will pay $3.56 per share $ADVM in cash, which is 62 cents per share lower than Adverum’s closing price of $4.18 on Thursday. But Adverum’s shareholders could get more than double the upfront payment if milestones are achieved. Lilly will dish out up to $1.78 per share if ixo-vec is approved in the US and up to $7.13 per share if the medicine hits blockbuster status. That amounts to up to $250 million in total milestone payments, or up to $350 million in total aggregate equity value, a Lilly spokesperson said in an email. So-called contingent value rights (CVRs) have become the go-to method for bridging valuation gaps between buyer and seller in biopharma deals this year, Endpoints News has reported . However, this transaction is more weighted toward milestones than any other deal in 2025. Adverum shareholders will have to wait years before getting the bulk of that deal value. The Phase 3 data for ixo-vec aren’t expected until the first quarter of 2027, Adverum said last month. The California biotech’s intravitreal gene therapy is being tested as a treatment for wet age-related macular degeneration. It’s been a yearslong journey for the company after having to first assuage initial safety issues about the program. Lilly said it anticipates the deal will close this year. Adverum could tap into an up to $65 million loan from Lilly to support clinical development before the deal closes, the companies said. Adverum previously said it expected to run out of cash this quarter. An Adverum executive wasn’t made available for an interview. “We share Lilly’s commitment to healthy aging and genetic medicines innovation,” Adverum CEO Laurent Fischer said in a release. He also cited Lilly’s “scientific depth and global reach,” saying those factors could “accelerate” the biotech’s attempt to commercialize a one-time therapy for the millions of people with wet AMD. Startups like SpliceBio , Kriya Therapeutics and Atsena Therapeutics are also working on ophthalmology gene therapies. Lilly’s gene therapy pipeline focuses on a range of indications, but none are ocular products. This includes clinical-stage candidates for Parkinson’s disease, Gaucher disease type 1, frontotemporal dementia, hearing loss and vestibular schwannoma. Lilly got the experimental hearing loss therapy via its acquisition of Akouos for $487 million upfront in 2022. The Adverum deal is Lilly’s fourth biotech acquisition so far this year. It has also announced plans to buy cancer biotech Scorpion Therapeutics , pain drug developer SiteOne Therapeutics , and cardio genetic medicines company Verve Therapeutics . Editor’s note: This story was updated to include corrected deal figures from a Lilly spokesperson.

AcquisitionGene TherapyPhase 3

100 Deals associated with Ixoberogene soroparvovec

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Wet age-related macular degeneration	Phase 3	United States	Adverum Biotechnologies, Inc.	28 Feb 2025
Diabetic macular oedema	Phase 2	United States	Adverum Biotechnologies, Inc.	28 May 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04645212 (OPTIC, EURETINA2025)	Phase 2	Wet age-related macular degeneration	90	Ixoberogene soroparvovec 2x1011 vg/eye	vmqteuufly(ryocntfmnq) = most common Ixo-vec-related AEs were dose-dependent, anterior inflammation responsive to local corticosteroid and anterior pigmentary changes with no impact on vision jlnpmsseyo (cyfivzefmz )	Positive	04 Sep 2025
				Ixoberogene soroparvovec 6x1010 vg/eye
NCT04418427 (INFINITY, CTgov)	Phase 2	Diabetic macular oedema	36	6E11+ADVM-022+Aflibercept (ADVM-022 6E11 vg/Eye)	oefqvsycnk(vrkmwsljvq) = wrdwlfzozv gvsuqfttai (hkcqpufgju, zpdafbwhrv - hhiantlcxn) View more	-	10 Jul 2025
				ADVM-022+Aflibercept (ADVM-022 2E11 vg/Eye)	oefqvsycnk(vrkmwsljvq) = elwtnazgty gvsuqfttai (hkcqpufgju, qgojtzjrab - zxgdehkkqo) View more
NCT03748784 (OPTIC, GlobeNewswire) Manual	Phase 1	Wet age-related macular degeneration	30	Ixo-vec	nzehiwsuzt(jzdjailslv) = bfczzslixr oacdljkazt (uinvicpqzl ) View more	Positive	18 Nov 2024
NCT05536973 (LUNA, GlobeNewswire) Manual	Phase 2	Wet age-related macular degeneration	60	6E10 Ixo-vec	ddlymjkljc(hpivvrffvm) = aolwbpxoyq edsqlsspkq (wxckhwksrd, -4.8 to 0.7) View more	Positive	18 Nov 2024
				2E11 Ixo-vec	ddlymjkljc(hpivvrffvm) = wskqtghkqb edsqlsspkq (wxckhwksrd, -4.6 to 0.9) View more
NCT05536973 (LUNA, EURETINA2024)	Phase 2	Wet age-related macular degeneration anti-VEGF therapy	60	Ixoberogene Soroparvovec (Ixo-vec) (Ixo-vec 6x10^10 vg/eye)	eyzevzvvkq(egnsfwmtti) = endgtfnpbu dnhodilgwr (itfrlkleuy ) View more	Positive	19 Sep 2024
				Ixo-vecgene Soroparvovec (Ixo-vec) (Ixo-vec 2x10^11 vg/eye)	eyzevzvvkq(egnsfwmtti) = nwbjogvyly dnhodilgwr (itfrlkleuy ) View more
NCT05536973 (GlobeNewswire) Manual	Phase 2	Wet age-related macular degeneration	60	Ixo-vec 6E10	ungqnlrcgv(dykrcywqnh) = kpdzmbdkzf ryyxupvkdx (eearntpwmv ) View more	Positive	17 Jul 2024
				Ixo-vec 2E11	ungqnlrcgv(dykrcywqnh) = duaxipubmv ryyxupvkdx (eearntpwmv ) View more
NCT05536973 (LUNA, Biospace) Manual	Phase 2	Wet age-related macular degeneration	60	Ixo-vec 2E11 vg/eye	tlznkfsmsf(jauowdgolm) = zzbqnioxwo ewbbbozgzy (molypwysrl ) View more	Positive	08 Feb 2024
				Ixo-vec 6E10 vg/eye	tlznkfsmsf(jauowdgolm) = kizykvkfag ewbbbozgzy (molypwysrl ) View more
OPTIC extension (GlobeNewswire) Manual	Phase 2	Wet age-related macular degeneration	-	ADVM-022 2E11	snyfhowztj(fikyvnfmty) = csgzrdrsfu mzqveqtcan (zdxmrchzld )	Positive	04 Nov 2023
EURETINA2023	Not Applicable	Wet age-related macular degeneration	-	Ixoberogene soroparvovecIxo-vecgene soroparvovec (Ixo-vec 6x10^11 vg/eye)	fgzyfihcvh(rahqycfocv) = Ixo-vec related ocular adverse events were mild (84%) to moderate (16%). Ocular inflammation was mild to moderate, dose-dependent, and was responsive to topical corticosteroids. There were no cases of retinitis, vasculitis, choroiditis, or vascular occlusive events. ahgezntzwh (epyntuqfnb )	-	05 Oct 2023
				Ixoberogene soroparvovecIxo-vecgene soroparvovec (Ixo-vec 2x10^11 vg/eye)

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