Gene & Cell Editing

Akamai Technologies surged 21.3% in premarket trading after announcing a seven-year, $11.6 billion cloud services commitment from AI company Anthropic, designed to support Anthropic's growing CPU workload demands across Akamai Cloud's distributed infrastructure. The deal includes provisions allowing the relationship to expand by up to an additional $9 billion, potentially taking the total commitment to about $20 billion, and Akamai also disclosed a hardware supply agreement with Lenovo and authorized contract manufacturer Jabil to procure about $1.7 billion in memory components. People Incorporated rose 6.5% in premarket trading after The Wall Street Journal reported that MGM Resorts International is weighing a potential takeover bid for Barry Diller's media conglomerate, with an offer potentially coming within days, just one day after People Incorporated withdrew its own $48.30-per-share cash proposal to acquire MGM's remaining public shares. Select Water Solutions rose 5.5% after announcing a definitive agreement to acquire Pilot Water Solutions for $700 million, plus up to $15 million in potential contingent consideration, in a transaction including $600 million in cash and $100 million in Class A common stock. Prime Medicine climbed 11.7% in after-hours trading to $3.49 after the U.S. Food and Drug Administration cleared its Investigational New Drug application for PM647, an investigational in vivo Prime Editor designed to correct the E342K mutation in the SERPINA1 gene, the root cause of Alpha-1 Antitrypsin Deficiency, paving the way for a global first-in-human Phase 1/2 clinical trial. On the downside, Comcast slipped 1.6% in premarket trading to $21.77 after KeyBanc downgraded the stock to Underweight from Sector Weight with an $18 price target, Twilio fell 3.7% after HSBC downgraded it to Reduce from Hold with a $211 price target, Zscaler fell 3.6% after naming Ross Tackett Chief Revenue Officer effective Oct. 1, succeeding Mike Rich, and Scholastic tumbled 11% after reporting fiscal first-quarter 2027 results that missed expectations, with revenue down 3.9% year over year to $216.8 million and an adjusted loss per share of $3.63.

Prime Medicine shares jumped 8% Friday after the FDA cleared its IND application for PM647, an experimental gene-editing therapy for Alpha-1 Antitrypsin Deficiency. The clearance allows Prime Medicine to begin a Phase 1/2 trial evaluating PM647 in adults with the inherited disorder, with initial clinical data expected in 2027. PM647 is designed to fix the genetic mutation that causes Alpha-1 Antitrypsin Deficiency, and Prime Medicine said the treatment could restore the normal protein and potentially help treat both the lung and liver problems caused by the disease.

REGENXBIO Inc. announced positive three-year long-term follow-up data from the Phase II ALTITUDE study of investigational surabgene lomparvovec, also known as sura-vec or ABBV-RGX-314, in non-proliferative diabetic retinopathy using suprachoroidal delivery, presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles. In data as of August 17, 2026, 60% of all Dose Level 3 participants with three-year visits, or 6 of 10, achieved a greater than 2-step improvement on the Diabetic Retinopathy Severity Scale without additional treatment for diabetic retinopathy, and these participants experienced no vision-threatening events. Additionally, the majority of participants, 3 of 4, who achieved a 1-step DRSS improvement at one year without supplemental anti-VEGF injections went on to achieve a greater than 2-step DRSS improvement by three years without additional treatment. No new sura-vec-related safety signals and no intraocular inflammation were observed through three years in 17 participants receiving short-course prophylactic topical steroids. Dose Level 3 is being evaluated in the Phase IIb/III NAAVIGATE trial of sura-vec in NPDR, and REGENXBIO is developing sura-vec in collaboration with AbbVie.

The global autologous hematopoietic stem cell gene therapy market is projected to grow from $5.23 billion in 2025 to $6.30 billion in 2026, a compound annual growth rate of 20.6%, and to reach $13.44 billion by 2030 at a 20.8% CAGR, according to the Autologous Hematopoietic Stem Cell Gene Therapy Market Global Report 2026 added to ResearchAndMarkets.com. Growth drivers include commercialization of gene-editing therapies, rising regulatory approvals, wider adoption of personalized medicine and expanding investment in cell and gene therapy infrastructure, alongside next-generation non-viral delivery platforms. In February 2024, Vertex Pharmaceuticals received conditional marketing authorization from the European Commission for CASGEVY, developed with CRISPR Therapeutics, for patients aged 12 and older with severe sickle cell disease and transfusion-dependent beta thalassemia. In October 2025, AGC Biologics partnered with Rarity Public Benefit Corporation to support development and Good Manufacturing Practice manufacturing of RDP-101 for adenosine deaminase severe combined immunodeficiency. North America was the largest regional market in 2025, while Asia-Pacific is expected to be the fastest-growing region; featured companies include Novartis AG, Vertex Pharmaceuticals, CSL Behring, Orchard Therapeutics, CRISPR Therapeutics, Rocket Pharmaceuticals, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics and Prime Medicine.

PTC Therapeutics has completed its previously announced agreement with Sangamo Therapeutics to acquire ST-920, a BLA-stage, one-time administered AAV gene therapy for Fabry disease. A rolling BLA submission to the FDA for accelerated approval of ST-920 is expected to be completed in the fourth quarter of 2026. Chief Executive Officer Matthew B. Klein said the company looks forward to completing the submission and potentially bringing a one-time administered, safe and effective durable disease treatment to the Fabry community. ST-920, also known as isaralgagene civaparvovec, has received Orphan Drug, Fast Track, and RMAT designations from the FDA, along with Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency. In clinical studies, the therapy enabled long-term production of the deficient alpha-galactosidase A enzyme and significant reduction in globotriaosylceramide levels.

Beijing has unveiled a five-year plan aimed at making China a global leader in drug innovation, with a target of developing 25% of the world's first-in-class drugs by 2030. The plan, issued in a joint notice from multiple government ministries, lays out 25 priority tasks spanning innovation capacity, research and technology, including expanded R&D, the use of AI and quantum computing in drug discovery, and the building of pharmaceutical ecosystems beyond pipelines. The government aims for the industry to reach 3.5tn yuan, or $520bn, in annual revenue by 2030, with 50 companies exceeding $1.5bn in annual revenue, and at least five Chinese drugs achieving global annual sales above $1bn. The notice singled out next-generation therapies including antibody-drug conjugates, cell and gene therapies, and CAR-T, along with vaccines and newer platforms such as mRNA, and included a section on industrialising traditional Chinese medicine. Citi analysts said the plan signals a shift from scale-driven growth toward innovation-driven, quality-oriented development, with a broad whole-value-chain policy push covering innovative drugs, medical devices, AI-driven drug discovery, and CDMO/CRO. The biomedical plan was released alongside counterparts for other strategic sectors, including integrated circuits, aerospace, the low-altitude economy, new energy storage and intelligent robotics.

On September 18, the Ministry of Industry and Information Technology, the National Development and Reform Commission and eight other departments jointly issued the 15th Five-Year Plan for the development of the pharmaceutical industry, laying out 25 key tasks in eight areas. The plan proposes that by 2030, operating revenue of Chinese pharmaceutical enterprises above designated size will exceed 3.5 trillion yuan, the innovative drug industry will grow at an average annual rate of more than 20 percent, China's share of first-in-class innovative drugs globally will exceed 25 percent, more than 200 innovative medical devices will be launched, and the number of pharmaceutical industry parks with output of at least 100 billion yuan will reach 20. Boosted by the news, innovative drug and CRO stocks rose on September 21, with Nanhua Bio gaining for a third straight day, while Harbin Pharmaceutical Group and Shandong Xinhua Pharmaceutical hit their daily limits. The same day, PCB-related shares advanced, as research reports from multiple institutions noted that upstream raw material prices have kept climbing and PCB makers have begun repricing, with the third quarter of 2026 expected to mark the start of an earnings recovery for the sector. MLCC-related shares were active, as AI-driven demand has caused shortages and sharp price surges in high-capacity models, with some spot prices soaring several-fold. Humanoid robot stocks rebounded, after drone footage suggested that the main structure of Tesla Optimus's dedicated production line in Texas may be nearing completion; the line is scheduled to begin production in 2027 with an annual capacity target of 10 million units.

Ultragenyx Pharmaceutical received full U.S. FDA approval in September 2026 for FAYUVI, also known as UX111, the first-ever treatment for pediatric patients with Sanfilippo syndrome Type A, a previously untreated ultra-rare and fatal neurodegenerative disease. The approval came with a Priority Review Voucher and expands Ultragenyx's commercial gene therapy portfolio and U.S. manufacturing footprint. The FAYUVI clearance follows the accelerated FDA approval of GENGLYCOS, also known as DTX401, for GSDIa in August 2026, giving the company two new gene therapies approved within weeks, each generating its own Priority Review Voucher. Ultragenyx's investment narrative projects $1.2 billion in revenue and $43.8 million in earnings by 2029, with a $27.00 fair value estimate implying 86% upside to the current price. The company's biggest immediate risk remains high cash burn and potential dilution if launches underperform or costs do not come down, with adoption, reimbursement and scaling of the new therapies shaping whether it can move closer to its 2027 profitability goal.

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.

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.