Gene & Cell Editing

Andelyn Biosciences announced it is now manufacturing FAYUVI, Ultragenyx Pharmaceutical Inc.'s FDA-approved gene therapy for Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA, for commercial supply at its Columbus, Ohio facility. The move follows the U.S. Food and Drug Administration's approval of FAYUVI, making it the first FDA-approved gene therapy manufactured using the Andelyn AAV Curator Platform process. Sanfilippo syndrome type A is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginning in early childhood; it is estimated to affect 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years. Andelyn Chief Executive Officer Wade Macedone said the company is proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator Platform process, calling the milestone a reflection of the purpose behind Andelyn's founding. Andelyn, a full-service FDA-inspected cell and gene therapy commercial CDMO with more than 20 years of experience, has produced clinical and commercial material for more than 500 cGMP batches and 85 global clinical trials.

The U.S. Food and Drug Administration has approved FAYUVI, also known as UX111, a gene therapy developed by Ultragenyx Pharmaceutical Inc. for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare and progressive neurodegenerative lysosomal storage disorder. Abeona Therapeutics Inc. congratulated Ultragenyx on the approval, noting that the therapy originated from pioneering research by Drs. Haiyan Fu and Douglas McCarty at The Ohio State University and Nationwide Children's Hospital and was advanced through clinical development by Abeona as ABO-102 before Abeona out-licensed global development and commercialization rights to Ultragenyx in May 2022. Abeona Chief Executive Officer Vish Seshadri called the approval an extraordinary milestone for patients and families affected by a devastating disease that has lacked effective therapeutic options. The approval also represents a meaningful value-creation event for Abeona, which under its licensing agreement with Ultragenyx is eligible to receive certain commercial milestone payments and royalties tied to future product sales. FAYUVI is designed to deliver a functional copy of the SGSH gene to the central nervous system via an AAV9 viral vector, addressing the root genetic cause of MPS IIIA.

Eli Lilly has entered an exclusive research collaboration with QurCan Therapeutics to develop genetic medicines for nervous system diseases. The agreement centers on QurCan's polymer lipid nanoparticle delivery platform for central and peripheral nervous system targets, with Eli Lilly responsible for later development and commercialization. Lilly is also making a strategic investment in QurCan Therapeutics, tying capital directly to progress in genetic medicine delivery technology. The deal pushes Lilly further toward expansion in neurodegenerative and other specialty drugs alongside its well known GLP 1 portfolio, though pricing pressure and payer pushback still hang over Mounjaro, Zepbound and Foundayo. Investors will want to see a first concrete output such as a nominated development candidate or an announced IND timeline for at least one nervous system genetic therapy.

On September 18, the Ministry of Industry and Information Technology, the National Development and Reform Commission, and eight other ministries jointly released the 15th Five-Year Plan for the Development of the Pharmaceutical Industry, proposing that by 2030, biopharmaceutical R&D and application will rank among the world's leaders, and the biopharmaceutical industry will accelerate its rise as a national emerging pillar industry. The plan sets 10 expected indicators covering industrial scale and efficiency, innovative development, enterprise cultivation, and cluster development, including operating revenue of pharmaceutical industrial enterprises above designated size exceeding 3.5 trillion yuan, an average annual growth rate of the innovative drug industry scale exceeding 20 percent, more than 5 products with global annual sales exceeding 1 billion US dollars, first-in-class innovative drugs accounting for more than 25 percent of the global total, more than 200 innovative medical devices launched, 50 pharmaceutical industrial enterprises with annual operating revenue exceeding 10 billion yuan, and 20 pharmaceutical industrial parks at the 100-billion-yuan level. Boosted by this news, the three major stock indices all rose more than 1 percent in early trading that day. Huatian Technology hit the daily limit up, with turnover of 6.065 billion yuan, nearly 1.47 million lots of sealed buy orders on the limit-up board, and nearly 3.3 billion yuan of main capital rushing in, ranking first in the A-share market. Zhou Sicong, fund manager of Ping An Pharmaceutical Select, believes innovative drugs are likely to become an important growth theme, and CITIC Securities said China's innovative drugs have entered a stage of global value realization. According to statistics from Securities Times Data Treasure, since September, Chengdu Leading Pharmaceutical, Asymchem Laboratories, Porton Pharma Solutions, and Hunan Warrant Pharmaceutical have led gains, all rising more than 10 percent, with Chengdu Leading Pharmaceutical up a cumulative 16.87 percent.

The Faculty of Veterinary Science at Chulalongkorn University has succeeded in developing stem cell technology from dog adipose tissue to create insulin-producing cells that replace damaged pancreatic cells. Preliminary study results show a significant reduction in blood sugar levels. The research team is scaling up production to pilot scale and expects to begin testing in actual diabetic dogs within the next 2-3 years. Associate Professor Dr. Jenpop Sawangmak, Director of the Veterinary Stem Cell and Bioengineering Innovation Center (VSCBIC) and co-founder of Bioing Co., Ltd., stated that diabetes occurs in about 1 in 300 dogs and cats, particularly the type similar to type 1 diabetes in humans. The team uses mesenchymal stem cells (MSCs) derived from dog fat, which yield 500 times more cells than bone marrow and can be easily harvested during spay/neuter surgeries. Dr. Saranyu Ounthawee, a researcher, said they are using microfluidics technology to control the creation of millions of cell clusters for transplantation. The research project has been ongoing for over 8 years, has received 6 petty patents, and has established a spin-off company for commercial development. The team is also studying exosome therapy and plans to apply the knowledge to treat kidney, liver, and joint diseases in animals, as well as to exchange information with the human medical field. If the testing is successful, it will help reduce financial burdens and elevate Thailand's biomedical and veterinary standards to an international level.

Ultragenyx Pharmaceutical Inc. announced the publication of 96-week data from its Phase 3 study of GENGLYCOS AAV gene therapy for glycogen storage disease type Ia in The Journal of Inherited Metabolic Disease, showing that participants achieved a mean reduction in daily cornstarch intake of 61% while maintaining glycemic control. The study met its primary endpoint at Week 48, with DTX401-treated patients experiencing a 41% reduction in cornstarch compared to 10% for placebo, and by Week 96, 67% of participants in both the original and crossover groups eliminated at least one nighttime cornstarch dose, with 33% and 42% respectively eliminating nighttime dosing entirely. Patient-reported outcomes showed that 83% of DTX401-treated participants met or exceeded their own expectations for meaningful cornstarch reduction at Week 48, and the therapy was generally well tolerated with an acceptable safety profile. GENGLYCOS was recently approved by the FDA for patients ages eight and older with GSDIa.

Vertex Pharmaceuticals Incorporated stock has risen 15.4% in a month, driven by strong second-quarter results, higher 2026 guidance, and growing confidence in its post-cystic fibrosis growth story. The company reported second-quarter revenues of $3.33 billion, up 12% year over year, and raised its full-year revenue outlook to $13.1-$13.2 billion from $12.95-$13.1 billion previously. Earnings of $4.73 per share rose around 5% year over year. Vertex's CF products generated revenues of $6.1 billion in the first half of 2026, up 8.4% year over year, with Alyftrek sales of $573.6 million in the second quarter, up 35% sequentially. Non-CF products, including Journavx and Casgevy, are gaining traction, with combined second-quarter sales of $126 million, and the company expects non-CF revenues to exceed $500 million in 2026, up about 185% year over year. Vertex's renal pipeline, including povetacicept for IgAN, is advancing, with an FDA decision expected by Nov. 30, 2026. The stock trades at 27.52 forward earnings, above the industry's 19.44, and the Zacks Consensus Estimate for 2026 earnings has declined to $19.01 per share over the past 30 days. In July 2026, Vertex agreed to acquire Crinetics Pharmaceuticals for about $10 billion, adding rare endocrine diseases as a fifth pillar. Despite headwinds, Vertex remains a Zacks Rank #3 (Hold) stock, with long-term investors advised to retain it.

Abeona Therapeutics reported second-quarter results showing ZEVASKYN revenue of $11.4 million, a 31% increase from the first quarter, and secured a new technology add-on payment from the Centers for Medicare and Medicaid Services effective October 1, 2026. The company has treated 12 patients since launch, including five in the second quarter and three more in the third quarter to date, with its treatment center network expanding to seven activated sites. However, the net loss widened to $20.2 million, or $0.35 per share, from $17.1 million, or $0.30 per share, and one low-yield batch in the second quarter plus one out-of-specification batch in the third quarter generated no revenue despite patients being treated. Abeona also announced it will stop reporting leading indicators like scheduled biopsies and instead report only patients treated and revenue recognized each quarter.

Healios announced on the 19th that a patent has been registered in China for its next-generation iPS cell, the Universal Donor Cell, or UDC, created using gene-editing technology. The UDC is a low-immunogenic iPS cell made by removing polymorphic HLA through gene editing and introducing HLA-G, PD-L1, and PD-L2, giving it resistance to both adaptive and innate immunity. A suicide gene has also been introduced as a safety switch, and the company says the UDC is expected to serve as a next-generation technology platform for creating regenerative medicine products as a raw material for transplanted cells.

The US FDA granted accelerated approval to Ultragenyx Pharmaceutical's gene therapy Genglycos to treat glycogen storage disease type Ia in individuals eight years and older. The condition affects 1,500 to 2,500 patients in the US and 6,000 to 8,000 worldwide. Chief Medical Officer Eric Crombez noted that Genglycos is the first treatment to target the root cause of the disease. Accelerated approval was based on results from a phase 3 randomized, double-blind, placebo-controlled study in which those on Genglycos saw a reduction in cornstarch requirements compared to those in the placebo cohort. Shares are up about 5% in after-hours trading.

Biotech stocks reached their highest level since the height of the pandemic after Moderna and Merck announced that a late-stage trial testing their messenger RNA-based cancer therapy met its main goals. Healthcare became the best-performing sector in the S&P 500, with the State Street SPDR S&P Biotech ETF adding more than 4% to reach its highest level since February 2021. The individualized neoantigen therapy intismeran autogene, combined with Merck's Keytruda, improved recurrence-free survival with a statistically significant and clinically meaningful effect in the Phase 3 INTerpath-001 trial. Moderna shares more than doubled, Merck added over 12%, and rival vaccine makers BioNTech and Novavax also gained sharply. Gene editing companies including Prime Medicine, Intellia, CRISPR, Editas Medicine, and Beam Therapeutics were notable gainers, while AI-related biotechs Absci and Recursion Pharmaceuticals rallied after Anthropic selected Twist Bioscience as an independent evaluator.

Vertex Pharmaceuticals reported second quarter 2026 results, raising full year revenue guidance to US$13.1b to US$13.2b and completing a US$1.42b share repurchase program. The stock closed at $516.44, which Simply Wall St's narrative model frames as 7.6% undervalued relative to a fair value of $558.68. Vertex's current P/E of 29.7x sits above the US Biotechs industry average of 15.9x and above a fair ratio of 28.2x. The company's pipeline diversification includes programs in pain, kidney, and type 1 diabetes, leveraging genomic and gene-editing technologies.

MeiraGTx reported second quarter 2026 financial and operational results, highlighted by the completion of its acquisition of botaretigene sparoparvovec from Johnson & Johnson for $25 million and a strategic investment of up to $400 million from Oberland Capital. The company received FDA Breakthrough Therapy Designation for AAV2-hAQP1 and reported positive three-year data from its Phase 1 AQUAx clinical trial for radiation-induced xerostomia. MeiraGTx anticipates submitting global regulatory filings for bota-vec in 2026 and a potential BLA filing for AAV2-hAQP1 in mid-2027. Net income attributable to ordinary shareholders for the quarter was $160.7 million, or $1.76 basic and $1.71 diluted per share, compared to a net loss of $38.8 million in the prior year period. Cash, cash equivalents and restricted cash totaled $145.4 million as of June 30, 2026.

Intellia Therapeutics reported positive top-line results from the Phase III HALO trial for LONVOSI in hereditary angioedema, achieving an 87% reduction in mean monthly attacks versus placebo and hitting all key secondary endpoints. The company is advancing a rolling BLA submission with the FDA and expects to announce acceptance by the end of 2026, positioning for a potential U.S. launch in the first half of 2027 as the world's first in vivo gene editing product. Cash, equivalents, and marketable securities totaled $628.4 million as of June 30, 2026, up from $605.1 million at the end of 2025, with the runway expected to fund operations at least into 2028 excluding potential product revenues. Collaboration revenue fell to $7.7 million from $14.2 million a year earlier, while R&D expenses declined to $82.6 million from $97.0 million, and G&A expenses rose to $37.8 million from $27.2 million, resulting in a net loss of $106.6 million compared to $101.3 million in the prior-year quarter. Intellia also resumed enrollment in both Phase III trials for NEXI in ATTR after resolving clinical holds, and identified a specific HLA allele associated with higher-grade transaminase elevations, enabling new patient-screening strategies.

Regenxbio ended the second quarter of 2026 with cash, equivalents, and marketable securities of $106 million, and on a pro forma basis more than $310 million after receiving a $100 million milestone payment from AbbVie and approximately $108 million in net proceeds from a follow-on public offering, extending its cash runway into the fourth quarter of 2027. The company completed enrollment in the confirmatory study for RGX-202 ahead of schedule, with over 60 patients in pivotal and confirmatory trials, and the FDA confirmed that available data for RGX-121 is sufficient for review under accelerated approval with no additional studies required for BLA resubmission. Long-term data for sura-vec in wet AMD and diabetic retinopathy showed durable efficacy and safety, and the Phase 2B/3 NAVIGATE study for diabetic retinopathy was initiated. Top-line data for sura-vec in wet AMD is expected in the fourth quarter of 2026, while the BLA submission for RGX-202 is a multi-module process with the clinical module not expected to be complete until the first quarter of 2027. The US patent on Zolgensma has expired, reducing future royalty revenue from that product in the US, though coverage remains in about 20 countries outside the US and on Invisma worldwide.

MEDEZE Group Public Company Limited, or MEDEZE, is moving forward to reinforce its leadership in stem cells and regenerative medicine in Thailand by developing infrastructure that is certified both domestically and internationally. This covers a modern drug manufacturing license from the Food and Drug Administration, the country's first cell bank standard from the Ministry of Public Health, AABB and ISO accreditations, as well as participation in the Advanced Therapy Medicinal Products Regulatory Sandbox to help drive the development of advanced medical products in the country. Chief Executive Officer Dr. Veerapol Khemarangsan revealed that the company is committed to building confidence among the public, investors, and the medical community by developing comprehensive stem cell and advanced medical infrastructure under standards recognized both in Thailand and internationally. It is also continuously extending research, collaboration with medical institutions, and innovation in regenerative medicine to elevate Thailand's potential toward becoming a regional hub for regenerative medicine and medical innovation.

Shape Therapeutics and the Rett Syndrome Research Trust have announced a partnership to advance SHP-401, an investigational one-time gene therapy for Rett syndrome. Under the agreement, RSRT will fund translational studies to evaluate Shape’s RNAfix guide RNA, designed to correct the MECP2 R168X mutation—the most common causal point mutation in Rett patients, accounting for approximately 10% of cases—paired with a proprietary AAV5-derived capsid for delivery to the central nervous system. The studies will be conducted in non-human primates to characterize biodistribution and on-target editing, with the goal of generating data to support advancement toward IND-enabling studies. In a recent preclinical study, a single intravenous dose of the therapy achieved approximately 70% RNA editing of the R168X mutation throughout the brain in a mouse model, restoring full-length MeCP2 protein, substantially improving Rett-like phenotypes, and extending median lifespan from 50 days to as long as 174 days while reducing the risk of death by 88 to 93 percent. The collaboration aims to build on these results and move the program closer to clinical translation.

Lexeo Therapeutics has received Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration for LX2020, its investigational gene therapy for PKP2-associated arrhythmogenic cardiomyopathy. The designation was granted based on recent interim clinical data from the ongoing HEROIC-PKP2 Phase I/II clinical trial. LX2020 now holds RMAT, Orphan Drug, and Fast Track designations, which provide enhanced opportunities for FDA interaction and may enable accelerated approval pathways. The company’s Chief Medical Officer, Narinder Bhalla, stated that the milestone underscores the potential of LX2020 to address the underlying genetic cause of the disease, for which no approved disease-modifying treatments currently exist. Lexeo Therapeutics expects to share additional clinical and regulatory updates before the end of the year.

Beam Therapeutics has dosed the first patient in the global pivotal cohort of its Phase 1/2 trial evaluating BEAM-302 for alpha-1 antitrypsin deficiency. The company also completed dosing for all adult and adolescent patients in the Phase 1/2 BEACON trial of risto-cel in sickle cell disease, with a biologics license application submission expected as early as year-end 2026. Updated BEAM-302 clinical data were selected for a late-breaking oral presentation at the European Respiratory Society Congress in September 2026. Beam received FDA clearance of its investigational new drug application for BEAM-304 in phenylketonuria and has initiated clinical start-up activities. The company ended the second quarter of 2026 with $1.2 billion in cash, cash equivalents and marketable securities, and expects its cash runway to support operating plans into mid-2029.

CRISPR Therapeutics reported a narrower net loss of $91.2 million for the second quarter of 2026, down from $208.5 million a year earlier, and outlined multiple clinical updates expected in the second half of the year. The company held cash, cash equivalents, and marketable securities of $2,364.4 million as of June 30, 2026. Regulatory submissions have been completed for its gene-edited cell therapy CASGEVY in Saudi Arabia and the United Kingdom for children aged 5 to 11 with sickle cell disease and transfusion-dependent beta-thalassemia, adding to existing approvals in 39 countries. Pipeline updates anticipated in the second half include data on zugocabtagene geleucel for autoimmune disease and hematologic malignancies, a Phase 1b readout for CTX310 targeting ANGPTL3, and a Phase 2 update for CTX611, a long-acting siRNA therapy for thromboembolic and clotting-related indications being developed with Sirius Therapeutics. The company also initiated Phase 1 trials for CTX340 in refractory hypertension and CTX460 in alpha-1 antitrypsin deficiency.

Haitai Biological's investee company Zhongmou Medical has received clinical trial approval from the National Medical Products Administration for its independently developed ZM-02 ophthalmic injection, intended for the treatment of advanced retinitis pigmentosa. The drug is a Class 1 therapeutic biological product and represents a new-generation optogenetic gene therapy. It delivers a novel light-sensitive protein gene through a single intravitreal injection and can be broadly applied to both inherited and acquired retinal degenerative diseases. The drug previously obtained orphan drug designation from the U.S. FDA in October 2024. The company cautions that drug development involves high investment and high risk and will not affect near-term financial performance.

Taysha Gene Therapies and Catalent have signed a commercial supply agreement for TSHA-102, Taysha’s investigational gene therapy for Rett syndrome, under which Catalent will serve as the primary commercial manufacturer following potential FDA approval. The agreement builds on a partnership that has supported TSHA-102 development since 2020 and secures long-term manufacturing capacity at Catalent’s FDA-licensed gene therapy facility in Harmans, Maryland. Catalent will provide GMP manufacturing and commercial supply, leveraging its experience across more than 90 gene therapy programs. Taysha’s Chairman and CEO Sean P. Nolan stated that establishing long-term commercial manufacturing capacity is a critical component of the company’s launch readiness strategy, with BLA-enabling Process Performance Qualification activities already underway. Rett syndrome, caused by mutations in the MECP2 gene, affects an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K., and currently has no approved disease-modifying therapies.

Red Star Capital Bureau reports that gene editing therapies have already been approved for market overseas, with Casgevy priced at 2.2 million US dollars. In December 2023, the US FDA approved Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy, as well as Bluebird Bio's Lyfgenia, both for treating sickle cell disease patients aged 12 and above. In China, the main players in gene editing therapy are emerging biotech companies such as BonYao Bio and Boya Jiyin, with BonYao Bio's BRL-101 having entered Phase II clinical trials. Over 20 A-share listed companies are involved in gene editing technology, but most say they are not engaged in gene therapy research, instead using gene editing technology to support related new drug development. These include BioMap, Obio Technology, Shanghai Model Organisms, GenScript Biotech, Porton Pharma Solutions, and BGI Genomics.

Five biotech stocks featured on RTTNews have delivered substantial gains, with upcoming catalysts that could drive further upside. uniQure N.V. surged 186% since August 2025, driven by positive data for its Huntington's disease gene therapy AMT-130, and is on track to submit regulatory filings in the third quarter of 2026. CareDx Inc. gained 138% since June 2025 after a series of strong earnings reports, raised guidance, and the acquisition of Naveris, with the commercial launch of the NavDx liquid biopsy test as a key catalyst. Adaptive Biotechnologies Corp. rose 130% since May 2025, supported by growing adoption of its clonoSEQ MRD test and multiple guidance raises, with the planned separation of its Immune Medicine business as a potential catalyst. Enliven Therapeutics Inc. jumped 207% since November 2025 on positive Phase 1 data for its chronic myeloid leukemia drug ELVN-001, and expects to initiate a Phase 3 trial in the second half of 2026. Amylyx Pharmaceuticals Inc. climbed 217% since June 2025, with a pivotal Phase 3 data readout for its post-bariatric hypoglycemia drug Avexitide anticipated in the third quarter of 2026.

Morgan Stanley analyst Terence Flynn nearly doubled his price target for CRISPR Therapeutics to $60 from $33 and upgraded the stock to hold. The new target implies a potential upside of 26% from current levels. CRISPR Therapeutics, a gene-editing specialist, recently won a label expansion for its approved medicine Casgevy, developed with Vertex Pharmaceuticals, to treat patients as young as two with certain rare blood disorders. The company also expects important data readouts by year-end for pipeline candidates including CTX310, a potential one-and-done therapy for high LDL cholesterol and triglycerides, and CTX611, an investigational anticoagulant designed to reduce bleeding risk.