PTC Therapeutics, Inc.

Rising prevalence of genetic disorders, supportive regulatory frameworks, and advancements in gene and RNA-based therapies are fueling market expansion. WILMINGTON, DE, UNITED STATES, September 5, 2025 / EINPresswire.com / -- The global nucleic acid therapeutics market is entering a phase of accelerated growth, driven by strong innovation pipelines and favorable policy support. Valued at US$ 8.8 Bn in 2024, the market is projected to grow at a robust CAGR of 14.7% between 2025 and 2035, reaching US$ 44.5 Bn by 2035. Growing awareness of genetic diseases, widespread clinical trials, and rapid progress in RNA-based platforms are reshaping the future of precision medicine. Market Introduction Nucleic acid therapeutics represent one of the most transformative innovations in modern biotechnology. These therapies leverage engineered DNA and RNA molecules to correct or silence defective genes, introduce beneficial genetic material, or modulate protein expression. Core modalities include gene therapies, antisense oligonucleotides (ASOs), RNA interference (RNAi), aptamers, and messenger RNA (mRNA)-based therapeutics. Their therapeutic applications extend across neuromuscular disorders, oncology, viral infections, ophthalmological diseases, metabolic syndromes, and autoimmune conditions, providing high specificity, targeted action, and the potential for one-time curative treatments. Unlike traditional pharmaceuticals, nucleic acid therapeutics address the root cause of diseases at the genetic level, minimizing systemic toxicity and enabling personalized medicine. Dive Deeper into Data: Get Your In-Depth Sample Now - https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=86333 Analyst Viewpoint Analysts at Transparency Market Research note that the nucleic acid therapeutics market is on the cusp of becoming a mainstream segment in the biopharmaceutical industry. Key growth catalysts include: Rising prevalence of rare and inherited disorders such as Duchenne muscular dystrophy, cystic fibrosis, and sickle cell anemia, which remain underserved by conventional treatments. Regulatory acceleration through FDA Fast Track, Breakthrough Therapy, and EMA Priority Medicines (PRIME) designations, which shorten development timelines for critical therapies. Challenges remain in terms of manufacturing scalability, delivery mechanisms, and cost-effectiveness, particularly for therapies requiring complex viral or lipid nanoparticle vectors. However, the success of mRNA vaccines during the COVID-19 pandemic has proven the scalability and adaptability of RNA-based platforms, catalyzing confidence among investors and policymakers. The increasing number of clinical trials, coupled with strategic collaborations, points to a market that is set to transform global healthcare by 2035. Key Market Drivers 1. Rising Prevalence of Genetic Disorders Genetic diseases affect millions worldwide, with a growing number being diagnosed through advancements in genomic sequencing and prenatal screening. This improved detection rate is expanding the eligible patient pool for nucleic acid therapeutics. As these therapies directly correct or silence defective genes, their adoption is expected to increase significantly in the coming years. 2. Regulatory Approvals and Expedited Pathways Regulatory agencies such as the U.S. FDA and the European Medicines Agency (EMA) are creating streamlined approval frameworks for nucleic acid therapeutics, particularly in the area of rare and orphan diseases. Expedited review processes reduce time-to-market, encouraging pharmaceutical and biotech companies to increase investment in clinical development. 3. Advancements in RNA-based and Gene Therapies Breakthroughs in antisense oligonucleotides, siRNA, and mRNA platforms are enabling therapies that target diseases previously considered untreatable. The integration of artificial intelligence, nanotechnology-driven delivery systems, and improved chemical modifications has enhanced drug stability, precision targeting, and reduced off-target effects, making RNA-based treatments commercially viable. Segment Analysis By Therapy Type Antisense Oligonucleotides (ASOs): Largest segment due to their efficacy in modulating gene expression; widely used in neuromuscular and rare genetic disorders. Small Interfering RNA (siRNA): Emerging as a strong segment with applications in oncology and metabolic diseases. Gene Therapies: High growth potential owing to curative benefits in monogenic disorders. Aptamers: Gaining momentum in targeted oncology treatments. Others (including mRNA therapies): Strong future growth expected, particularly post-COVID-19 success. By Delivery Method Viral Vector-based Systems: Widely used but face challenges in immunogenicity and manufacturing scalability. Non-viral Delivery Systems: Lipid nanoparticles and polymer-based carriers showing promising safety and efficiency. By Route of Administration Intravenous: Dominant due to systemic distribution. Subcutaneous: Preferred for ease of administration and chronic therapies. Others (Intrathecal, etc.): Specialized routes for targeted disease conditions. By Therapeutic Area Neuromuscular Disorders (DMD, SMA) Metabolic Disorders Cardiovascular Disorders Ophthalmological Disorders Oncological Disorders Others (Infectious, Autoimmune, etc.) By End-user Hospitals (largest share due to advanced care infrastructure) Academic and Research Institutes (driving clinical trials and innovation) Specialty Centers (focusing on genetic and rare disorders) Regional Insights North America: Leads the global market due to strong biotech infrastructure, presence of leading companies, and early adoption of innovative therapies. Favorable reimbursement policies and active regulatory support enhance growth prospects. Europe: Rapid adoption supported by EMA’s proactive stance on genetic therapies. Strong research ecosystems in Germany, the UK, and France are advancing innovation. Asia Pacific: Poised for fastest growth with rising healthcare investments, supportive government initiatives, and a growing patient pool in China, India, and Japan. Latin America & Middle East & Africa: Emerging regions where adoption is gradually increasing, though affordability and access remain challenges. Partnerships with global biotech firms are expected to enhance growth. Key Players Novartis AG Pfizer, Inc. Sanofi Novo Nordisk A/S AstraZeneca plc Alnylam Pharmaceuticals, Inc. Amgen Inc. Sarepta Therapeutics, Inc. Bluebird Bio, Inc. CSL Behring LLC Ferring Pharmaceuticals Inc. Krystal Biotech, Inc. PTC Therapeutics, Inc. Jazz Pharmaceuticals plc Astellas Pharma Inc. Recent Developments Novartis (Nov 2024): Acquired Kate Therapeutics, expanding its AAV-based gene therapy pipeline for neuromuscular diseases in a deal valued up to US$ 1.1 Bn. Sarepta Therapeutics (Nov 2024): Entered into a licensing agreement with Arrowhead Pharmaceuticals for siRNA programs in muscle and rare pulmonary disorders, valued at US$ 825 Mn. Market Trends Accelerating investments in RNA-based and gene-editing platforms. Rising demand for personalized and precision medicine approaches. Continuous technological innovations in viral and non-viral delivery platforms. Increasing collaborations between pharma companies, biotech firms, and academic institutions to fast-track clinical pipelines. Future Outlook The nucleic acid therapeutics market is expected to remain one of the fastest-growing sectors within biotechnology and pharmaceuticals through 2035. Factors such as increasing prevalence of genetic disorders, regulatory acceleration, advancements in RNA and gene therapy platforms, and strong demand for personalized medicine will drive market expansion. With a projected CAGR of 14.7%, the sector is set to deliver groundbreaking treatments that could redefine modern healthcare. Why Buy This Report? Reliable market size forecasts and CAGR projections through 2035 In-depth assessment of market drivers, restraints, and opportunities Comprehensive segmentation by therapy type, delivery method, therapeutic area, and region Competitive landscape with profiles of leading companies and recent strategic developments Analysis of emerging technologies and trends shaping future growth Browse More Trending Research Reports: Nucleic Acid Extraction Instruments & Reagents Market - https://www.transparencymarketresearch.com/nucleic-acid-extraction-instruments-reagents-market.html Oligonucleotide Synthesis Market - https://www.transparencymarketresearch.com/oligonucleotide-synthesis-market.html Aptamers Market - https://www.transparencymarketresearch.com/aptamers-market.html RNA Therapeutics Market - https://www.transparencymarketresearch.com/rna-therapeutics-market.html Small Interfering RNA (siRNA) Therapeutics Market - https://www.transparencymarketresearch.com/small-interfering-rna-therapeutics-market-report.html Dark Genome Therapeutics Market - https://www.transparencymarketresearch.com/dark-genome-therapeutics-market.html Nucleic Acid Aptamers Market - https://www.transparencymarketresearch.com/nucleic-acid-aptamers.html Ligase Enzymes Market - https://www.transparencymarketresearch.com/ligase-enzymes-market.html Thermal Cycler Market - https://www.transparencymarketresearch.com/thermal-cycler-market.html Electroporation Instruments Market - https://www.transparencymarketresearch.com/electroporation-instruments-market.html Legionella Testing Market - https://www.transparencymarketresearch.com/legionella-testing-market.html Cell Analysis Market - https://www.transparencymarketresearch.com/cell-analysis-market.html Molecular Diagnostics Market - https://www.transparencymarketresearch.com/molecular-diagnostics-industry.html Biobanking Market - https://www.transparencymarketresearch.com/biobanking-market.html Genetic Testing Services Market - https://www.transparencymarketresearch.com/genetic-testing-services-market.html Array Market - https://www.transparencymarketresearch.com/global-array-market.html Oligonucleotides Market - https://www.transparencymarketresearch.com/oligonucleotides-market.html Digital PCR & Quantitative PCR Market - https://www.transparencymarketresearch.com/digital-pcr-quantitative-pcr-market.html About Transparency Market Research Transparency Market Research, a global market research company registered at Wilmington, Delaware, United States, provides custom research and consulting services. Our exclusive blend of quantitative forecasting and trends analysis provides forward-looking insights for thousands of decision makers. Our experienced team of Analysts, Researchers, and Consultants use proprietary data sources and various tools & techniques to gather and analyses information. Our data repository is continuously updated and revised by a team of research experts, so that it always reflects the latest trends and information. With a broad research and analysis capability, Transparency Market Research employs rigorous primary and secondary research techniques in developing distinctive data sets and research material for business reports. Contact: Transparency Market Research Inc. CORPORATE HEADQUARTER DOWNTOWN, 1000 N. 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PITTSBURGH, Sept. 2, 2025 /PRNewswire/ -- RareMed Solutions®, the nation's leader in patient support services for complex therapies, is proud to announce an expansion of its relationship with PTC Therapeutics™ with the launch of non-commercial pharmacy dispensing services for PTC's therapy, Sephience™. The recent approval of Sephience by the FDA marks a significant milestone in the treatment of phenylketonuria (PKU) for both adult and pediatric patients. This new therapy, developed by PTC, has the potential to transform the lives of those living with PKU by offering an innovative treatment option that addresses their unique needs. Support for Patients and Providers RareMed will be providing non-commercial pharmacy dispensing services to support patients in challenging circumstances. These services are designed to provide both patients and healthcare providers with appropriate assistance. To provide seamless access and support, RareMed RareSupport® Team is expanding. A larger team of liaisons will play a crucial role in providing outreach and coordination efforts across PTC's supported therapies so that every patient may receive appropriate, personalized care and attention, while leadership will provide guidance, oversight, and compliance support. Commitment to Care RareMed's commitment to providing care extends beyond just the launch of new therapies. The program's support framework is designed to assist patients and providers alike in navigating the treatment landscape with confidence. By expanding the team, RareMed aims to facilitate a smoother, more coordinated experience for all stakeholders involved in the treatment process. As RareMed continues to grow and adapt to the evolving needs of its patients, the company's focus remains steadfast on delivering compassionate, effective, and timely care. About Phenylketonuria Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe). If left untreated or poorly managed, Phe—an essential amino acid found in all proteins and most foods—can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally. About RareMed RareMed Solutions is the nation's only concierge patient services provider, delivering award-winning care to patients living with complex conditions. RareMed partners with biopharma to transform the lives of patients afflicted with devastating complex conditions by accelerating access to biomedical breakthroughs. RareMed offers case management, co-pay, coupon and financial assistance programs, reimbursement support, nursing support, healthcare professional education, patient adherence & education, and non-commercial pharmacy dispensing services to all fifty states from its headquarters in Pittsburgh, Pennsylvania and bases across the country. RareMed's advanced AI-integrated hub technology offerings, including the revolutionary RarePath® platform, provide unparalleled management and analytics capabilities to biopharma across the nation. The company has a breadth of experience developing, transitioning, and maintaining therapy-specific solutions that ensure unparalleled manufacturer & patient satisfaction across a variety of patient services models. RareMed's undivided complex disease focus, high caliber associates, fully dedicated teams, and sophisticated proprietary technology enable it to address the unique needs of complex disease patients. RareMed has been consistently recognized as a top workplace in the nation by USA Today and Newsweek and continually raises the benchmark for patient experience according to MMIT patient satisfaction surveys, in which the team recently achieved a perfect 100-point net promoter score. Visit RareMed's website at . SOURCE RareMed Solutions WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Pictured: FDA Headquarters, iStock, Grandbrothers iStock, Grandbrothers Aside from the rare disease market, Novo Nordisk also scored a key regulatory win last month for its blockbuster GLP-1 drug Wegovy, which can now be used to treat patients with metabolic dysfunction-associated steatohepatitis. August was a big month for the rare disease space, which saw four history-making approvals—though the streak was marred by one rejection. Also walking away with a win last month was Novo Nordisk, which secured a critical label expansion that, according to analysts, could help its blockbuster GLP-1 medication gain access to nearly $2 billion in added peak sales. Read below for more. Four Firsts for Rare Diseases Company: Jazz Pharmaceuticals Drug: Modeyso Indication: Glioma The first rare disease nod came on Aug. 6 for Jazz Pharmaceuticals’ dordaviprone, which was approved for patients aged 1 year and above with diffuse midline glioma with an H3 K27M mutation who have progressed after prior treatments. The drug, which will carry the brand name Modeyso, is the first systemic therapy for this specific type of glioma, according to the FDA’s announcement of the approval. Modeyso was approved under the FDA’s accelerated pathway, supported by overall response (OR) data from a pair of Phase I and Phase II studies. According to Jazz’s news release announcing the approval, Modeyso achieved an OR of 22% in 50 adult and pediatric patients. Among responders, median duration was 10.3 months. To maintain Modeyso’s approval, Jazz is running a confirmatory Phase III trial that is expected to be complete in August 2026. Company: Insmed Drug: Brinsupri Indication: Non-cystic fibrosis bronchiectasis Not a week after Jazz, Insmed secured an FDA nod for brensocatib—now sold as Brinsupri—for the rare respiratory disorser non-cystic fibrosis bronchiectasis. Brinsupri, taken orally once daily, is indicated for patients aged 12 years and up. Insmed has set its price at $88,000 per year—“a little higher” than what analysts were expecting, Guggenheim Partners wrote at the time. Brinsupri marks two firsts for the biopharma industry: Not only is it the first drug for bronchiectasis to reach the market, but it is also the first approved treatment that works by blocking DPP1, an enzyme that plays a role in activating the inflammatory response in airways. Brinsupri is backed by data from the Phase III ASPEN trial. Data published in The New England Journal of Medicine in April showed that Brinsupri lowered the rate of pulmonary exacerbations by around 20% versus placebo. The Phase II WILLOW trial also supported Brinsupri’s approval, demonstrating a roughly 40% reduction in the risk of exacerbations relative to placebo. Company: Precigen Drug: Papzimeos Indication: Recurrent respiratory papillomatosis Then, on Aug. 14, Precigen won the FDA’s go-ahead for zopapogene imadenovec-drba, now named Papzimeos, for the treatment of recurrent respiratory papillomatosis (RRP). In its announcement of the approval, the FDA said Papzimeos is a “first-of-its-kind” non-replicating immunotherapy for this disease. With around 1,000 new cases in the U.S. annually, RRP is a rare disease that manifests as benign tumors in the airways, leading to difficulties in swallowing and breathing. If left unchecked, RRP can lead to death. Papzimeos works by helping the body mount an immune response against cells infected by HPV 6 and HPV 11, both associated with RRP. Pivotal Phase I/II data supported Papzimeos’ approval, showing that the biologic elicited a complete response rate of more than 50% in treated patients, while over 85% needed fewer surgical interventions in the year after treatment. Company: Ionis Pharmaceuticals Drug: Dawnzera Indication: Hereditary angioedema Capping off the rare disease rally this month is Ionis Pharmaceuticals. On Aug. 22, the California biotech’s antisense oligonucleotide donidalorsen, now branded Dawnzera, became the industry’s first RNA-targeting prophylactic for hereditary angioedema (HAE). Patients 12 years and older can receive the therapy, which is given via a subcutaneous injection every four weeks. Dawnzera targets the prekallikrein mRNA, causing its destruction. This, in turn, reduces overall levels of PKK, a protein that plays a role in swelling and pain attacks in HAE. Data from the OASIS-HAE study backed Thursday’s approval, demonstrating an 81% reduction in HAE attack rate versus placebo over 24 weeks of observation. Results were published May 2024 in the New England Journal of Medicine and additionally showed a significant improvement in patients’ quality of life. Wegovy Win For Novo On Aug. 15, the FDA gave the go-ahead for Novo Nordisk’s blockbuster injection Wegovy to be used in adults with metabolic dysfunction-associated steatohepatitis. The GLP-1 drug, indicated for patients with moderate to advanced liver scarring but without cirrhosis, should be used in conjunction with a reduced-calorie diet and higher physical activity. Wegovy’s approval in MASH is a “step in the right direction” for Novo, analysts at BMO Capital Markets wrote in an Aug. 17 note to investors, adding that breaking into the MASH market “could start to help shift the momentum” for Wegovy, which in the first half of 2025 has been hit hard by the rise of compounders. BMO anticipates peak MASH sales of $1.9 billion for Wegovy. Data from the Phase III ESSENCE trial, which supported the label expansion, showed that Wegovy improved liver fibrosis without worsening steatohepatitis in 37% of treated patients at 72 weeks, versus 22.5% in placebo comparitors. At the same time, Wegovy resolved steatohepatitis without worsening fibrosis in 62.9% of patients, as compared with 34.1% of placebo participants. Wegovy has “clear efficacy in MASH,” the BMO analysts wrote, adding that its “clean safety pro broad benefits across metabolic disease” could help establish the drug “as a backbone treatment for MASH.” Wegovy joins Madrigal Pharmaceuticals’ Rezdiffra in the MASH space. Approved in March 2024 , Rezdiffra made $180.1 million last year. Three COVID-19 Vaccines Cleared, With Restrictions The FDA on Aug. 27 approved updated COVID-19 vaccines from Pfizer, Moderna and Novavax, but with key limitations: The shots can only be used in adults 65 years and older and younger people who are at elevated risk of severe outcomes. There are some minor differences across the three, particularly as it pertains to use in the at-risk younger population. Novavax’s Nuvaxovid, a protein-based shot, is authorized for individuals 12 through 64 years. Pfizer and BioNTech’s mRNA-based Comirnaty can be given to children as young as 5 years. Moderna’s mNEXSPIKE is indicated for people 12 through 64 years, while Spikevax is indicated for individuals 6 months through 64 years. Both vaccines are mRNA-based. These approvals come after Health Secretary Robert F. Kennedy Jr. in May removed routine COVID-19 vaccination for healthy children and healthy pregnant women from CDC recommendations. PTC Hit With Rejection in Friedreich’s Ataxia The FDA on Aug. 19 turned down PTC Therapeutics’ vatiquinone, which the company has proposed for the treatment of Friedreich’s ataxia in children and adults. In its complete response letter, the FDA stated that “substantial evidence of efficacy was not demonstrated” and that PTC would need an additional “adequate and well-controlled study” to support a resubmission. Matthew Klein, the company’s CEO, said in a statement at the time that PTC is planning to meet with the FDA to discuss potential next steps. Vataquinone is a small molecule that blocks some of the cellular pathways that go awry in patients with frataxin mutations, the underlying cause of Friedreich’s ataxia. Patients with this rare neuromuscular disease suffer from loss of coordination and muscle strength, as well as difficulty speaking, swallowing and breathing. Around 25,000 patients have been diagnosed worldwide, according to PTC. Vataquinone missed the mark in a Phase III, registration-directed trial back in 2023, failing to meet its primary endpoint of improving gait, stability and limb function after 72 weeks. PTC sought registration with the FDA based on secondary outcomes such as stability.