Voretigene Neparvovec

Peter Marks' departure after a decade as the director of the FDA's Center for Biologics Evaluation and Research left some worried about the future of cell and gene therapies. The sudden resignation of Peter Marks, M.D., Ph.D., from the FDA branch that regulates biological products for humans laid bare the vaccine skepticism underpinning Robert F. Kennedy Jr.’s new leadership in the federal healthcare system.“If you’re going to be a biotech company right now, don’t be a vaccine company,” Leerink Partners analyst Mani Foroohar, M.D., said in a recent interview.But there’s another field of human medicines managed under Marks’ previous leadership that could face significant disruptions—cell and gene therapies. To many in this realm of the biopharma industry, Marks’ exit means losing a seasoned regulator and key ally.Marks played a pivotal role in ushering in the modern era of cell and gene therapies. A year after he became the director of the Center for Biologics Evaluation and Research (CBER) in 2016, his department approved the very first CAR-T cell therapy, Novartis’ Kymriah, opening up a drug class that offers the hope of a potential cure for patients with once difficult-to-treat blood cancers. That same year, the FDA cleared the U.S.’ first gene therapy, Roche’s Luxturna.In a landmark approval by Marks’ CBER at the end of 2023, Vertex and CRISPR Therapeutics’ Casgevy became the first treatment based on the Nobel Prize-winning CRISPR gene-editing system.  And last year, Marks oversaw the go-ahead of Iovance Biotherapeutics’ Amtagvi, the first cell therapy for solid tumors.Amid all of these developments, Marks wasn’t just a bystander but rather an outspoken champion of novel technologies and an advocate of regulatory flexibility to accelerate approvals.“We want to lean into accelerated approval whenever we can, particularly in the rare disease space, because if you have patients in need, getting something to them a year or two sooner can make the difference,” Marks said at the 2024 DIA Global Forum.The former CBER chief also worked to streamline the interactions between the agency and developers of cell and gene therapies. He introduced INTERACT meetings, which offer sponsors of advanced biological therapies the opportunity to gain early guidance from the FDA on a product development plan. He was also instrumental in the idea of a platform designation to significantly slash development timelines and regulatory work around follow-on therapies based on reproducible technology.Marks wasn’t the first senior FDA leader to exit under the new administration. His counterpart at the Center for Drug Evaluation and Research (CDER), Patrizia Cavazzoni, M.D., had left in January. But Marks’ departure was more poignantly felt by the industry, partly because of the duration of his leadership and “how he has really changed the character of—and approach at—CBER during his tenure,” Foroohar said.While small molecule drug regulations are well established, CBER requires more regulatory handholding because of the novelty and complexity of advanced technologies, Foroohar added.As Foroohar put it, Marks “was a driver of a lot of acceleration” for the cell and gene therapy space.Now, with Marks’ departure—plus a massive workforce reduction at the FDA—that trajectory looks to be under threat. Plus, any government-related hits would serve as a double whammy for an industry that’s already undergoing a serious crisis marked by pipeline discontinuations, layoff rounds, business closures, funding challenges, commercialization struggles and a Big Pharma retreat. Interpreting the impactFollowing news of Marks’ sudden departure, investors in vaccine and cell & gene therapy biotechs hit the exit, too.On Monday, besides the widely reported selloffs at Moderna and Novavax, CRISPR Therapeutics’ stock was down 8%, while share prices for base editing specialists Beam Therapeutics and Verve Therapeutics each dropped 12%. And allogeneic cell therapy leader Allogene Therapeutics also lost about 6% in market value.After the initial shock, industry watchers were left parsing through the implications for biopharma companies. Some within the industry are openly worried about the future of cell and gene therapies, even though Marks’ high-profile resignation was focused overwhelmingly on disagreements around vaccine regulations.“We MUST get Dr. Marks reinstated within the next 30 days,” Nicole Paulk, Ph.D., CEO of gene therapy startup Siren Biotechnology, posted on X on March 29.“We MUST protect Dr. Verdun at all costs,” she continued, referring to Nicole Verdun, M.D., director of CBER’s all-important Office of Therapeutic Products (OTP), which more directly evaluates cell and gene therapies. “What we do in the next 30 days may well dictate the future of #GeneTherapy #CellTherapy #Vaccines #GeneEditing for the next 100 years. We must act NOW!”Still, given how harshly Marks criticized RFK Jr. in his resignation letter (PDF), it seems “fairly unlikely” that Marks will be reinstated, Foroohar said. Besides Paulk, many others in the biopharma industry have expressed concern at Mark’s departure. Talking about the bigger biopharma sector, trade group Biotechnology Innovation Organization CEO John Crowley said in a Saturday statement: “We are deeply concerned that the loss of experienced leadership at the FDA will erode scientific standards and broadly impact the development of new, transformative therapies to fight diseases for the American people.”Some, however, are more optimistic.Stephen Majors, VP of global communications at the Alliance for Regenerative Medicine, is hopeful that cell and gene therapies won’t land in RFK Jr.’s crosshairs. Majors pointed to positive comments in the HHS secretary’s written testimony to Sen. Tim Scott during this confirmation hearing, as well as from Trump’s White House on the establishment of the Make America Healthy Again Commission, which brought up gene therapies for sickle cell disease as a noteworthy achievement.“I’ve spoken before on the future of gene therapies and the potential for developing technologies, like CRISPR-Cas9, to one day provide treatments for rare diseases like sickle cell,” RFK Jr. said in his response to Sen. Scott. “I am so excited that sickle cell disease patients now have two potential one-time cures, one developed with the CRISPR technology.”From Wall Street’s perspective, Citi analysts in a Monday note said they do not believe that Marks’ departure “will usher in a new period of regulatory uncertainty or questionable approvals.” Still, the analysts recognized Marks’ key role in regulatory policies toward cell and gene therapies.The analysts argued that it’s natural to have leadership turnover at federal agencies in a new administration, and that the FDA knows how to uphold continuity of approval standards.Similarly, a Jefferies team led by analyst Andrew Tsai also struck a positive note in a Saturday report to clients.Pointing to Marks’ comments in his resignation letter, Tsai concluded that “we do not see increased regulatory risk for sponsors currently working with CDER/CBER on some form an expedited pathway for rare diseases.”Marks highlighted the “unwavering commitment to public health priorities” that CBER has applied to “the development of cell and gene therapies to address both hereditary and acquired rare diseases.” CBER staffers are committed to “increased communication” with product developers and have been “exploring the dramatic transformation of our regulatory approach to expedite the delivery of directly administered genome editing products,” he wrote.However, whether those efforts will continue under Marks’ replacement—or face major disruptions—remains an open question. Two potential outcomesDepending on Marks’ potential successor, Foroohar envisioned the twin possibilities of relative stability or turmoil for cell and gene therapies. Until the agency provides comments, Foroohar is not favoring any scenario because “we don’t even have tea leaves yet.” That was on Monday.Tuesday afternoon, the FDA announced the appointment of Scott Steele, Ph.D., as acting director of CBER. Steele holds a Ph.D. in molecular biology from Princeton and has been a full-time senior adviser at CBER since late 2022. And outdated resume (PDF) of Steele’s on the FDA’s website shows he received predoctoral training in genetics at the National Institutes of Health until 1998.In Foroohar’s two scenarios, promoting an internal CBER leader would be “a strong indication that the status quo, philosophically, is going to continue for cell and gene therapy.”Despite the positive sign, Steele is at least for now only filling the role on an interim basis. Even among traditional institutionalists, Marks “had a more forceful view in favor of regulatory flexibility” and “a more high-touch regulatory approach with cell and gene therapy in particular,” Foroohar said. This suggests Marks’ stance toward these novel technologies appeared to be more favorable than many of his FDA colleagues.Marks brought to light his openness—particularly toward gene therapies—in his controversial decisions on Sarepta Therapeutics’ Duchenne muscular dystrophy treatment Elevidys. The former CBER chief stepped in to overrule objections from internal FDA reviewers twice for Elevidys’ initial accelerated approval and a traditional approval last year despite the failure of a confirmatory trial.“When we are on the leading-edge of science, we’re going to make mistakes sometimes, and we may not have the rock-solid evidence always,” Marks said during the Fierce Cell & Gene virtual event last fall. “But I think our commitment is that we will do our best to weigh the benefits, risks and uncertainty and wrap that up in terms of the other available potential therapies to make the best decision we can.”Elevidys may have been an isolated event, but it’s possible at a CBER without Marks that cell and gene therapies are forced to contend with more stringent rules, William Blair analysts said in a Monday note.The other scenario in Foroohar’s modeling could be more chaotic.“If we see someone whose background is much less technical than one would expect, more of a political appointee, doesn’t have direct regulatory experience, perhaps someone who doesn’t have a lot of direct scientific drug development experience, you would start to worry that that is a case where you’re going to see a lot more destruction,” Foroohar said.In this scenario, things could go bad for cell and gene therapies in two divergent ways, he figured.A political appointee could be more skeptical of the biopharmaceutical industry as a whole, adopting a stricter approach to accelerated approvals and making clinical development more difficult and costly.Alternatively, an FDA outsider could come with much greater regulatory flexibility than even Marks, falling closer to the “right to try” camp, Foroohar explained. There were advocates for that ideology under President Trump’s first term, he noted.That approach may come as a near-term positive for the cell and gene industry. But less scientific rigor would eventually be harmful to the health of the industry, Foroohar argued.“There’s a risk that if you have a very flexible regulatory profile, you start to have products that are approved with labeled indications and datasets that don’t clearly differentiate clinical data,” Foroohar said.As a result, payers would balk at reimbursement even for approved agents, which Foroohar views would be a “more profound long-term challenge to the industry.” A greater concernAlthough a trailblazer and a visible leader, Marks is just one person. To Foroohar, a potential bigger FDA issue threatening the growth of cell and gene field is the likely loss of talented staff at CBER.“The greater concern is that you’ll see a bleeding away either from headcount reductions or from […] voluntary departures of reviewers, CMC staff, etc., the boots on the ground […] as you see people struggle with working under a leadership […] in an agency where they feel like the mission is not respected or they don’t have support from the administration.”Lola Fashoyin-Aje, M.D., an FDA veteran who was tapped in early 2024 to lead CBER’s office of clinical evaluation focused on cell and gene therapies, left the agency a few weeks ago. Before CBER, Fashoyin-Aje worked at the FDA’s Oncology Center of Excellence, where she formed and served as the program lead of Project Equity, an initiative designed to promote racial diversity in clinical trials. Her departure came shortly after the Trump administration banned diversity programs at federal agencies.“There’s going to be a lot of departures behind Peter [Marks],” former FDA commissioner Scott Gottlieb, M.D., said Monday on CNBC’s “Squawk Box.”“If you pull bricks high up off the tower, it can be very obvious, and you can draw a lot of attention and also be unlikely to implode the entire structure,” Foroohar said, drawing an analogy between the FDA staffing situation and the game Jenga. “But if you start pulling all the blocks out from the middle and the bottom, you can pretty quickly collapse the infrastructure and make it wobbly very quickly.”As to the headcount reduction, RFK Jr.’s massive layoff round targeting 3,500 FDA employees—out of 10,000 for the entire HHS—appears to have affected industry-facing staffers even though core drug reviewers have been spared.Nearly the entire FDA team involved with negotiating industry user fees has been terminated, Politico’s AgencyIQ reported Tuesday, citing several sources close to the situation. The FDA and the biopharma industry need to agree on user fee programs, including the Prescription Drug User Fee Act (PDUFA) that governs the evaluation of drugs, to ensure that the agency has enough resources to provide predictable timelines for its review process.RFK Jr. has in the past expressed skepticism toward user fee programs—which are considered a bedrock of FDA operations—out of concerns of undue industry influence.“An FDA reviewer needs the support of others,” Leerink’s Foroohar said. “There’s all kinds of other infrastructure necessary for them to allow a reviewer to do their job.”And it’s not as if CBER is overstaffed, at least according to Marks and Foroohar. The FDA’s struggle to hire and retain talent to handle the influx of complicated cell and gene therapy programs in the past few years has been no secret among industry watchers.“I suspect we’re going to need more headcount in that area very soon,” Marks said last April during an Alliance for a Stronger FDA webinar. At that time, the agency was still hiring up and evaluating the resources necessary to “really make the transformation to a very high performing organization,” he said.“Don’t be surprised if, as we move through the next year or so, we come up with some asks,” Marks said during the webinar.   A regulatory retreat?Now, instead of bulking up, the FDA is slimming down.The current headcount reduction at the FDA is reportedly targeting communications, policy and operations functions. But some FDA operations that are critical to the industry beyond specific product reviews may be dependent on those staffers. “We’re advocating for OTP to be able to continue in its work, and part of that is maintaining the ability to have these early communications with companies,” Majors at the Alliance for Regenerative Medicine said. “Those early interactions are very, very important in terms of being able to move programs along quickly.”For example, on the policy side, the FDA in May 2024 laid out a draft guidance on a new platform designation, which aims to provide an expedited pathway for the development of novel therapies that utilize the same reproducible technology, such as a gene-editing system. The platform would save the FDA a figurative “three billion visits” by drug developers every time they try to use the same technology to edit a disease gene, Marks said at the American Society of Gene and Cell Therapy meeting last year.During the Fierce Cell & Gene event last year, Marks also said that his department was re-evaluating the class-wide boxed warnings about secondary cancers that it had previously slapped on existing commercial CAR-T therapies for blood cancers. Companies are closely following the FDA’s decision on the warning as they try to develop CAR-T products for more prevalent conditions, such as autoimmune diseases, which entail entirely different benefit-risk calculations from cancers.At the Alliance for a Stronger FDA webinar last year, Marks touted the Support for clinical Trials Advancing Rare disease Therapeutics (START) pilot program. It offers constant communication and clinical development guidance for companies developing treatments for rare disease to shorten their development timeline.“If we can show even a 10% decrease in time that translates into a sufficiently large number of dollars across different rare disease programs, that one could potentially easily justify staffing up and the number of reviewers that would need to be brought on board to make this a regular procedure,” Marks said of the pilot.Whether the personnel changes would delay the formal rollout of those initiatives remains to be seen.“Every signal that we have is that they are supportive of cell and gene therapy,” Majors said of the Trump administration. “We just want to make sure that […] even if CGT was not the target, that there isn’t collateral damage based on the actions they’re taking with vaccines, specifically, and then, more broadly, with everything they’re doing in the reorganization of FDA.”

LUXTURNA has significant market potential due to the high unmet need for effective treatments in rare genetic conditions. With its FDA approval for specific mutations in the RPE65 gene, it offers hope to patients who previously had limited options. The growing awareness and adoption of gene therapies in ophthalmology, along with advancements in personalized medicine, are expected to expand LUXTURNA's market reach. LAS VEGAS, March 26, 2025 /PRNewswire/ -- DelveInsight's " LUXTURNA Market Size, Forecast, and Market Insight Report" highlights the details around LUXTURNA, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of LUXTURNA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Spark Therapeutics/Novartis' LUXTURNA (AAV2-hRPE65v2; voretigene neparvovec) Overview LUXTURNA (voretigene neparvovec-rzyl, AAV2-hRPE65v2) is a one-time gene therapy designed to treat vision loss caused by mutations in both copies of the RPE65 gene. It works by delivering a functional copy of the RPE65 gene to replace the faulty one, helping to restore vision. The therapy is administered as a single subretinal injection beneath the retina in patients with confirmed RPE65 mutations and viable retinal cells. LUXTURNA was developed and commercialized in the US by Spark Therapeutics. Outside the US, Novartis holds the rights for development, registration, and commercialization under a licensing agreement. It is approved for treating vision loss caused by Leber's congenital amaurosis or retinitis pigmentosa due to confirmed biallelic RPE65 mutations. LUXTURNA uses an adeno-associated viral type 2 (AAV2) vector to deliver the RPE65 gene. The vector includes a cytomegalovirus (CMV) enhancer and a chicken beta-actin promoter to drive the expression of the normal human RPE65 protein. The parent AAV2 virus, which serves as the template, is a non-pathogenic, single-stranded DNA virus that depends on a helper virus for replication. Once inside retinal cells, the functional RPE65 protein enables the regeneration of 11-cis-retinal, which improves light detection and restores vision. In healthy individuals, RPE65 in retinal pigment epithelial cells converts all-trans-retinol into 11-cis-retinol, which then forms 11-cis-retinal—a critical step in the visual cycle that converts light into electrical signals. Mutations in the RPE65 gene disrupt this process, leading to vision loss. LUXTURNA restores the retinoid cycle by reintroducing functional RPE65 protein. LUXTURNA Dosage and Administration The recommended dose for each eye is 1.5 × 10¹¹ vector genomes (vg) in a 0.3 mL volume, administered via subretinal injection. Each eye should be treated on different days, with at least six days between injections. Oral corticosteroids equivalent to prednisone at 1 mg/kg/day (up to 40 mg/day) should be given for seven days, starting three days before injection, followed by a tapering dose over the next 10 days. LUXTURNA Dosage Forms and Strengths LUXTURNA is provided as a suspension for subretinal injection in a 0.5 mL extractable volume within a 2 mL single-dose vial. The concentration is 5 × 10¹² vg/mL, requiring a 1:10 dilution before use. The diluent is supplied in two single-use 2 mL vials. Learn more about LUXTURNA projected market size for retinitis pigmentosa @ LUXTURNA Market Potential Retinitis pigmentosa is a group of inherited retinal disorders that cause progressive degeneration of the retina and eventual blindness. It involves the gradual bilateral deterioration of rod and cone photoreceptors, leading to night blindness and progressive loss of peripheral vision, followed by a decline in central vision due to the loss of cone function. Retinitis pigmentosa is primarily linked to genetic mutations, with over 44 associated genes identified. In 2023, approximately 113,000 people in the US were affected by retinitis pigmentosa. Currently, available treatments are mainly off-label and do not address the underlying genetic causes of retinitis pigmentosa. Patients without the RPE65 mutation rely on supportive care, such as vitamin supplements, protection from sunlight, and visual aids. LUXTURNA is the only approved therapy for retinitis pigmentosa but is limited to patients with the RPE65 mutation. Gene and cell therapies are becoming key treatment strategies, targeting genes like RHO, USH2A, and RPGR. USH2A is a major focus, with two oligonucleotide candidates advancing in the RP pipeline. Optogenetics offers a novel gene therapy approach that works independently of specific gene mutations and shows promise in treating late-stage RP with severe photoreceptor loss. Several companies have progressed optogenetic gene therapies into clinical trials. According to DelveInsight, the retinitis pigmentosa gene therapy market is expected to grow due to increasing disease prevalence, improved awareness and treatment access, and a strong pipeline of therapies across the 7MM. Discover more about the retinitis pigmentosa market in detail @ Retinitis Pigmentosa Market Report Emerging Competitors of LUXTURNA Some of the drugs in the retinitis pigmentosa pipeline include Botaretigene sparoparvovec (Johnson & Johnson Innovative Medicine/MeiraGT), AGTC-501 (Beacon Therapeutics), MCO-010 (Nanoscope Therapeutics), GS030 (Gensight Biologics), CTx PDE6B (Coave Therapeutics), OCU 400 (Ocugen), jCell (jCyte), EA-2353 (Endogena Therapeutics), Ultevursen (ProQR Therapeutics), ADX‑2191 (Aldeyra Therapeutics), and other. In January 2025, Beacon Therapeutics Holdings Limited reported that the FDA has awarded Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for treating X-linked retinitis pigmentosa (XLRP). To know more about the number of competing drugs in development, visit @ LUXTURNA Market Positioning Compared to Other Drugs Key Milestones of LUXTURNA In October 2023, SpliceBio entered an exclusive collaboration and licensing agreement with Spark Therapeutics to utilize SpliceBio's proprietary Protein Splicing platform to develop a gene therapy for an undisclosed inherited retinal disease. In June 2023, Novartis Pharma obtained manufacturing and marketing approval for LUXTURNA in Japan as a gene therapy for IRD caused by mutations in both copies of the RPE65 gene. In November 2018, Novartis announced that the European Commission (EU) approved LUXTURNA for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene and who have enough viable retinal cells. The authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein, and Norway. In January 2018, Novartis entered into a licensing agreement with Spark Therapeutics covering development, registration, and commercialization rights to voretigene neparvovec in markets outside the US. Spark Therapeutics retains US rights for LUXTURNA. In December 2017, the FDA approved Spark Therapeutics' LUXTURNA for patients with confirmed Bi-allelic RPE65 Mutation-associated Retinal Dystrophy. In July 2017, Spark Therapeutics announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the US FDA designated LUXTURNA as a drug for a rare pediatric disease. In October 2016, LUXTURNA granted ODD for the treatment of inherited retinal dystrophy due to bi-allelic RPE65 mutation. In July 2015, LUXTURNA was designated as an orphan medicinal product for the treatment of retinitis pigmentosa. In September 2014, the US FDA granted LUXTURNA a Breakthrough Therapy Designation for Inherited retinal dystrophy. LUXTURNA Patent Details The company holds the following patents related to its AAV-based retinal gene therapy: US patent application relating to cell transfection improvements for AAV vector production. We expect that any patents issued from this application will expire in 2038, excluding any potential patent term extension or adjustment. A US patent application relating to improvements in AAV vector purification. It expects that any patent from this application will expire in 2038, excluding any potential patent term extension or adjustment. Patent for manufacturing gene therapy vector Spark Therapeutics holds exclusive licenses for three patent application families from CHOP, covering scalable methods for manufacturing high-purity gene therapy vectors. The first patent family pertains to the production of Spark's product candidates, including those under its collaboration with Pfizer. Patents from this family have been granted in the United States, Australia, and Mexico, with expiration dates in 2021, not accounting for any potential extensions or adjustments. Related patent applications are still under review in the United States, Brazil, Canada, China, Europe, Israel, India, and Japan. If granted, these patents are expected to expire in 2031, excluding any possible extensions or adjustments. Modified AAV vectors and gene delivery Spark Therapeutics is advancing technology across multiple areas to enhance and broaden its current product pipeline. This technology, exclusively licensed from CHOP, focuses on modifying gene therapy vectors, incorporating companion therapies or diagnostics, and developing new therapeutic genes. The licensed patent portfolio includes: Six US patent applications covering alternative or modified AAV vectors for gene delivery. Any resulting patents are expected to expire between 2028 and 2034, excluding possible patent term extensions or adjustments. Two pending US patent applications related to inhibiting immune responses to AAV vectors and detecting AAV-binding antibodies. Any patents granted from these applications are anticipated to expire between 2032 and 2034, excluding potential patent term extensions or adjustments. Discover how LUXTURNA is shaping the retinitis pigmentosa treatment landscape @ LUXTURNA Gene Therapy LUXTURNA Market Dynamics The market for LUXTURNA is primarily driven by the increasing prevalence of inherited retinal diseases (IRDs) and the growing acceptance of gene therapy as a viable treatment option. The high unmet medical need for effective treatments for rare genetic diseases has created a favorable environment for LUXTURNA. The therapy's ability to deliver long-term benefits from a single administration, reducing the burden of repeated treatments, has also contributed to its market acceptance. Furthermore, the strong backing from Roche, with its extensive commercialization and market access infrastructure, has helped expand LUXTURNA's reach across multiple markets. Favorable reimbursement policies in the U.S. and Europe have further supported market uptake. Despite its success, LUXTURNA faces significant market challenges. The high cost of treatment, which exceeds $850,000 for both eyes, poses a barrier to widespread adoption, even with reimbursement support. Access to treatment is also limited by the need for specialized surgical centers and trained ophthalmologists, which restricts the therapy's availability to select geographic regions. Moreover, as gene therapy technology evolves, the emergence of newer, potentially more effective or cost-efficient treatments could pose competitive pressure. Additionally, the complexity of gene therapy manufacturing and delivery, along with stringent regulatory requirements, adds to the operational challenges faced by Spark Therapeutics and Roche. The future market dynamics of LUXTURNA are expected to be influenced by ongoing advancements in gene therapy, increasing diagnostic capabilities for inherited retinal diseases, and expanding treatment infrastructure. Strategic partnerships and continued investment in patient access programs and healthcare provider training could enhance market penetration. Additionally, the successful clinical outcomes and long-term durability of LUXTURNA will play a crucial role in sustaining patient and physician confidence. As the gene therapy market matures, LUXTURNA's position as a first-mover in the inherited retinal disease space will likely provide it with a competitive edge, although it will need to navigate pricing pressures and emerging competition in the broader ophthalmology and gene therapy markets. Dive deeper to get more insight into LUXTURNA's strengths & weaknesses relative to competitors @ LUXTURNA Market Drug Report Table of Contents Related Reports Retinitis Pigmentosa Market Retinitis Pigmentosa Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key retinitis pigmentosa companies including Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic Sight, jCyte, Endogena Therapeutics, ProQR Therapeutics, Aldeyra Therapeutics, among others. Retinitis Pigmentosa Pipeline Retinitis Pigmentosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key retinitis pigmentosa companies, including Acucela Inc, Allergan Plc, Amarantus Bioscience Holdings Inc, Amgen Inc, Applied Genetic Technologies Corp, Asklepios BioPharmaceutical Inc, Astellas Pharma Inc, Caladrius Biosciences Inc, Dompe Farmaceutici SpA, GenSight Biologics SA, Grupo Ferrer Internacional SA, IDPharma Co Ltd, Janssen Pharmaceutical, MeiraGTx, Anabasis Pharma, Allegro Ophthalmics, Nacuity Pharmaceuticals, ReNeuron, jCyte, Editas Medicine, SanBio, NightstarTherapeutics, Biogen, OiDE OptoEye, among others. Cell and Gene Therapies in Rare Disorders Market Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cell and gene therapies in rare disorders companies including REGENXBIO, Coave Therapeutics, GenSight Biologics, Ultragenyx, Pfizer, Sangamo Therapeutics, Roche, 4D Molecular Therapeutics, Astellas Gene Therapie, Actus Therapeutics, Nanoscope Therapeutics, Ocugen, jCyte, Amicus Therapeutics, Capricor Therapeutics, Nippon Shinyaku, Brainstorm Cell Therapeutics, Editas Medicine, Abeona Therapeutics, Ishin Pharma, among others. Gene Therapy Competitive Landscape Gene Therapy Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gene therapy companies, including Novartis, Johnson & Johnson, Fibrocell Technologies, Pfizer, HELIXMITH Co., Ltd., Sarepta Therapeutics, REGENXBIO, Solid Biosciences Inc., Lexeo Therapeutics, Spark Therapeutics, Xalud Therapeutics, uniQure, Ultragenyx Pharmaceutical, Nanoscope Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . 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