Company Overview - Wave Life Sciences is a clinical-stage biotechnology company focused on developing stereopure oligonucleotide therapeutics for rare genetic diseases, utilizing its proprietary PRISM platform and strategic partnerships to enhance drug discovery and development across various therapeutic areas [1][10]. Financial Transactions - On December 8, 2025, Adrian Rawcliffe, a Director at Wave Life Sciences, exercised 42,000 options and sold the resulting shares in an open-market transaction valued at $630,000, with the exercise price set at $5.97 per share [3][5]. - The transaction was executed as part of a Rule 10b5-1 trading plan, which allows company insiders to sell shares on a predetermined schedule to mitigate the risk of insider trading [5]. Stock Performance - As of January 14, 2026, Wave Life Sciences' stock has increased nearly 28% year over year, outperforming the S&P 500's gain of 20% [6]. - The stock experienced a significant surge of 147% in a single trading session in December following the release of early clinical data for WVE-007, an RNA-targeted obesity therapy, indicating strong market interest in its innovative approach to obesity treatment [6][7]. Market Position - Wave Life Sciences is positioned as an innovator in the genetic medicines sector, particularly in the niche of RNA-targeting therapies, which are gaining traction in the pharmaceutical market [1][6]. - Despite the positive momentum, it is important to note that the clinical data released is still in the early stages, and further successful trials are necessary before commercialization can occur [7].

PresentationPaul BolnoPresident, CEO & Director Thank you, Tess, and thank you, everybody, for joining us today. Before we start, we'll obviously be making some forward-looking statements during this call, so I'd please ask you to refer to our SEC filings for updates. Taking a pause and thinking about entering 2026, we are energized now more than ever to unlock the broad potential of RNA medicines and open up the opportunity to transform human health. And what this really reflects back on as a journey on bu ...

Core Insights - The company is focused on unlocking the potential of RNA medicines to transform human health, emphasizing the importance of their proprietary RNA chemistry [1] Company Overview - The foundation of the company is its RNA chemistry, which differentiates it in the market and supports the rapid translation of genetic insights into impactful medicines [1] - The company has demonstrated a quick turnaround in developing therapies, with an obesity therapy taking only 18 months from generating mouse data to human clinical data, and about 24 months for further developments [1]

Wave Life Sciences (NasdaqGM:WVE) FY Conference January 13, 2026 05:15 PM ET Company ParticipantsPaul Bolno - President and CEOConference Call ParticipantsTessa Romero - VP and Analyst covering BiotechnologyTessa RomeroWelcome, everyone, to the 44th Annual J.P. Morgan Healthcare Conference. My name is Tess Romero, and I'm one of the Senior Biotech Analysts here at J.P. Morgan. Our next presenting company is Wave Life Sciences, and presenting on behalf of the company, we have President and CEO Paul Bolno. Pa ...

Wave Life Sciences J.P. Morgan Healthcare Conference January 13, 2026 Forward-looking statements Translating genetic insights into potentially best-in-class medicines This document contains forward-looking statements. All statements other than statements of historical facts contained in this document, including statements regarding possible or assumed future results of operations, preclinical and clinical studies, business strategies, research and development plans, collaborations and partnerships, regulato ...

Metabolic Health - The company demonstrated improvements in body composition by reducing fat, including visceral fat, while preserving lean mass[14]. - Increased visceral adiposity is associated with maintaining metabolic rate, which is linked to various diseases including cardiometabolic disorders[14]. - Silencing INHBE mRNA has potential to treat obesity and associated metabolic diseases, indicating a strategic focus on metabolic health[17]. - Higher circulating Activin E levels correlate with higher BMI, abdominal fat, and fasting insulin in non-diabetic adults, supporting the company's weight loss approach[19]. - The company is exploring the use of GalNAc-siRNA injections for potential treatment of obesity and metabolic diseases, indicating ongoing research and development efforts[20]. - The company aims to reduce abdominal adiposity to lower the risk of cardiovascular disease (CVD) and type 2 diabetes (T2D)[17]. - The company reported favorable traits in heterozygous INHBE carriers, including lower risk of abdominal obesity and higher HDL-c levels[16]. - The company is focused on developing strategies to enhance insulin sensitivity and reduce inflammatory risks associated with T2D[20]. - The company is committed to addressing metabolic dysfunction through innovative therapies and research initiatives[19]. - The company is actively pursuing market expansion in the metabolic health sector, leveraging its research on INHBE and Activin E[17]. - WVE-007 is expected to drive fat reduction and improve key measures of cardiometabolic health[21]. - A single dose of INHBE GalNAc-siRNA resulted in a 23% reduction in epididymal fat weight and a 40% reduction in body weight in DIO mice[22]. - WVE-007 shows potential for synergistic use with GLP-1s, leading to approximately 2x greater weight loss compared to GLP-1 alone[23]. - The INLIGHT clinical trial for WVE-007 is set to initiate in 1H 2026, targeting individuals with a BMI of 28-35 kg/m²[24]. - Improvements in body composition were observed three months after a single WVE-007 dose, with a 4% reduction in total fat mass and a 2.3% preservation of lean mass[25]. - A single dose of WVE-007 led to improvements in body composition similar to GLP-1 without muscle loss[27]. - The study indicates that reductions in serum Activin E are highly durable, supporting dosing once or twice per year[25]. - Key measurements in the INLIGHT trial will include safety, tolerability, pharmacokinetics, and body composition[24]. - Data from preclinical studies indicate that a 10 mg/kg dose of INHBE GalNAc-siRNA is effective in reducing body weight and fat mass[22]. - The company aims to address over one billion individuals globally with obesity through its innovative treatments[29]. - WVE-007 is expected to initiate new trials in 2026, focusing on fat loss while preserving muscle mass, with follow-up data anticipated in 1H 2026[30]. - The treatment strategy includes leveraging an orthogonal mechanism to incretins for enhanced efficacy in obesity management[29]. - The anticipated updates for WVE-007 include data from multiple dosing cohorts, which will inform future treatment protocols and market strategies[30]. Alpha-1 Antitrypsin Deficiency (AATD) - The treatment goals for Alpha-1 antitrypsin deficiency (AATD) include minimizing episodic exacerbations and decreasing liver damage, with approximately 200,000 individuals in the US and Europe affected by the severe form of the disease[33]. - RNA editing technology is being developed to restore physiological AAT production during acute phase responses, aiming to shift Pi*ZZ individuals to a biomarker profile consistent with Pi*MZ genotype[36]. - WVE-006 is positioned as a potential first-in-class therapy for AATD, addressing both liver and lung manifestations of the disease[35]. - The company anticipates significant improvements in metabolic safety and tolerability with its combination treatments for obesity[29]. - The follow-up data from the 240 mg and 400 mg single-dose cohorts of WVE-007 will provide insights into its efficacy in individuals with higher BMI and comorbidities[30]. - WVE-006 achieved total AAT levels exceeding 11 µM, with production of wild-type M-AAT greater than 50% during acute phase response[43]. - The RestorAATion-2 clinical trial is ongoing, with 400 mg MAD data expected in Q1 2026 and 600 mg SAD and MAD data expected in 2026[44]. - Total AAT reached 20.6 µM during an acute phase response, indicating a significant increase in AAT production[40]. - Serum AAT levels increased to 2.8 µM with a single 400 mg dose and 11.9 µM with a 200 mg multi-dose regimen[42]. - WVE-006 also reduces levels of Z-AAT, aiming to restore dynamic AAT physiology[41]. - The clinical trial objectives include safety, tolerability, pharmacokinetics, and serum M-AAT levels[44]. - The augmentation therapy has no impact on liver disease, while WVE-006 enables endogenous AAT production[41]. - Published data on CRP levels and AAT levels across different genotypes showed significant increases in one patient during an acute phase response[40]. PNPLA3 I148M Liver Disease - The RNA editing program WVE-008 targets PNPLA3 I148M liver disease, with a strong foundation in human genetics and durable RNA editing[47]. - Plasma AAT levels of approximately 13 µM were achieved, with 64% of patients reaching levels greater than 20 µM during acute phase response[43]. - The company reported a significant decrease in liver fat with PNPLA3 editing in the human HEPATOPAC® model, indicating effective treatment potential[52]. - WVE-008 is expected to be a first-in-class, disease-modifying therapy for PNPLA3 I148M-driven liver disease, with a Clinical Trial Application (CTA) anticipated to be filed in 2026[53]. - RNA editing is projected to restore PNPLA3 function, addressing liver diseases across various stages, with a focus on correcting the I148M mutation[51]. - Heterozygous carriers of PNPLA3 I148M have an 80% lower risk of liver-related death compared to homozygous carriers, highlighting the genetic impact on liver disease outcomes[49]. - The company aims to utilize a bifunctional modality to simultaneously knockdown and edit RNA, potentially unlocking complex indications requiring multiple target engagements[56]. - The editing approach is expected to restore full PNPLA3 activity, countering liver disease progression by improving lipid mobilization and reducing fat accumulation[51]. - Significant editing efficiency was achieved with AIMers, leading to a marked reduction in liver fat levels[52]. - The company is focusing on developing therapies that address the underlying genetic causes of liver diseases, particularly those related to PNPLA3 mutations[51]. - The RNA editing strategy is designed to mitigate inflammation and fibrosis associated with liver diseases, aiming for comprehensive treatment solutions[51]. - The company is committed to advancing its research and development efforts in RNA medicines to address complex liver disease indications[56]. Duchenne Muscular Dystrophy (DMD) - WVE-N531 demonstrated an average dystrophin expression of 7.8% between 24 and 48 weeks, with 88% of boys achieving greater than 5% dystrophin[62]. - The therapy has shown a mean exon skipping of 54% (95% CI: 46-63%) and a mean dystrophin expression of 7.8% (95% CI: 5.4-10.3%) at 48 weeks[64]. - Significant reductions in creatine kinase (CK) levels were observed, with approximately 50% reduction from baseline at 48 weeks[69]. - Evidence of reversal of fibrosis was noted, with fibrotic muscle declining by 28.6% at 48 weeks[72]. - WVE-N531 has received Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA, addressing a high unmet need in Duchenne muscular dystrophy (DMD) affecting ~1 in 5,000 newborn boys annually[61]. - The company plans to file for NDA with an accelerated approval strategy and monthly dosing planned for 2026[62]. - A significant decrease (~48%) in internalized nuclei was observed at 48 weeks, indicating a transition to healthier muscle[71]. - The therapy has shown statistically significant improvements in muscle health, including reduced inflammation and necrosis[65]. - WVE-N531 is the only DMD therapeutic to show uptake in myogenic stem cells, indicating its potential for muscle regeneration[70]. - The average tissue half-life of WVE-N531 supports a monthly dosing regimen, enhancing patient compliance[64]. - Wave DMD portfolio addresses a market opportunity of over $2.4 billion in the US, targeting 40-50% of the DMD population that remains untreated today[75]. - WVE-N531 shows significant improvements in treatment rates, with 8-10% for exon 51 and 11-13% for exon 45, compared to no available therapies for exon skipping[75]. Huntington's Disease - The company is advancing WVE-003, an allele-selective silencing treatment for Huntington's Disease, which affects over 200,000 patients across all disease states[79]. - Pre-symptomatic Huntington's Disease is estimated to affect around 160,000 individuals in the US and Europe, with symptomatic cases around 65,000[78]. - WVE-003 is characterized by its ability to penetrate the brain fully and address both loss-of-function and gain-of-function disruptions caused by mutant huntingtin protein[79]. - The company is focusing on expanding its market presence and product offerings, particularly in the exon skipping therapies segment[75]. - The minimal clinically important difference (MCID) for TTR is reported at 1.4 seconds, indicating significant clinical relevance in treatment outcomes[74]. - The company aims to leverage its unique portfolio to capture a significant share of the DMD and Huntington's Disease markets, enhancing patient outcomes[75]. - The potential for best-in-class exon skipping therapies is highlighted, with no current therapies available for certain exons[75]. - The company is committed to advancing its research and development efforts to address unmet medical needs in genetic diseases[79]. - WVE-003 achieved an industry-leading 46% reduction in mutant HTT protein levels in CSF with three doses, demonstrating significant efficacy[82]. - The treatment led to a 4.68% reduction in caudate atrophy compared to 5.10% in the placebo group, indicating a trend towards less atrophy[83]. - A 1% absolute reduction in the rate of caudate atrophy is associated with a delay of disability onset by at least 7.5 years, highlighting the clinical significance of the findings[84]. - The company anticipates delivering data from a 400 mg multi-dose cohort for WVE-006 in Q2 2026, indicating ongoing development efforts[89]. - Phase 2 trials for WVE-007 are set to initiate in H2 2026, focusing on obesity treatment, showcasing the company's expansion into new therapeutic areas[90]. - The company plans to submit an NDA for WVE-N531 to support accelerated approval with monthly dosing, reflecting strategic regulatory initiatives[89]. - WVE-003's efficacy in reducing mutant HTT levels correlates with functional benefits, suggesting potential for improved clinical outcomes[83]. - The company is poised for significant growth driven by RNAi and RNA editing technologies, indicating a strong pipeline of innovative products[88]. - The preservation of wild-type HTT levels throughout the study suggests a favorable safety profile for WVE-003[83]. - The company is evaluating new trials for WVE-007 as an add-on treatment for individuals with obesity, indicating a commitment to addressing comorbidities[90].

Wave expects to initiate a Phase 2a multidose portion of WVE-007 INLIGHT clinical trial in individuals living with obesity with higher BMI and comorbidities in 1H 2026, and initiate new trials of WVE-007 as an add-on to incretin and as post-incretin maintenance in 2026 Initial WVE-007 240 mg single-dose data reported in 2025 demonstrated improved body composition with fat loss similar to GLP-1 at three months with muscle preservation and the potential for once or twice-yearly dosing; higher dose and longer ...

Industry Overview - Genomics is a comprehensive study of genomes, which has gained significant interest from pharmaceutical and biotechnology companies for understanding diseases and developing therapies [2] - The distinction between genetics and genomics is crucial, with genomics focusing on the complete set of genes and their interactions within an organism [3] - Insights from genomic research are increasingly utilized to evaluate patient responses to drugs and support the development of targeted therapies, advancing personalized medicine [4] - The growth of genomics has also bolstered synthetic biology, which applies engineering principles to biology for various applications, including drug discovery and gene editing [5] - The rapid advancements in genomics are driven by significant reductions in the cost and time required for genome sequencing [6] Market Projections - The genomics market is projected to reach $80.17 billion by 2032 [9] - The global synthetic biology market was valued at $18.94 billion in 2025 and is expected to grow at a CAGR of 17.7% from 2026 to 2033 [9] Key Companies - **Pacific Biosciences of California (PACB)**: Specializes in advanced long-read sequencing systems for various applications, including healthcare and agriculture. The company has a Zacks Rank 2 (Buy) [12][14] - **Wave Life Sciences (WVE)**: A clinical-stage biotechnology company focusing on RNA medicines. The company reported positive interim data for its obesity treatment candidate WVE-007, which showed improvements in body composition [15][17] - **Sana Biotechnology (SANA)**: Developing cell engineering platforms for diseases like type 1 diabetes and B-cell cancers. The company has suspended its CAR T programs to focus on more promising candidates and has a Zacks Rank 3 [19][21]

CAMBRIDGE, Mass., Jan. 05, 2026 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced that Paul Bolno, MD, MBA, President and Chief Executive Officer, is scheduled to present at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Tuesday, January 13, 2026 at 2:15 p.m. PT / 5:15 p.m. ET. A live webcast of the presentation can be accessed by vis ...

Wave Life Sciences Ltd. (NASDAQ:WVE) ranks among the best stocks under $25 to buy now. On December 16, Oppenheimer boosted its price target for Wave Life Sciences Ltd. (NASDAQ:WVE) to $32 from $24, maintaining an Outperform rating on the company’s shares. The firm reported “highly encouraging” interim results for WVE-007, emphasizing the treatment’s clean risk profile at doses up to 600mg and apparent ease of translation from human genetics and preclinical research to clinical outcomes. According to Wave ...