Editas Medicine Announces EDIT-401 Oral Presentation at ESGCT Congress and Participation in Investor Conferences

Analysis reveals significant industry trends and economic implications

Release Date

2025-10-07

Category

Clinical Trial Event

Reference

Source

Breakthrough Clinical Results

Editas Medicine announced an oral presentation on EDIT-401, a CRISPR gene editing medicine for lowering LDL-cholesterol by upregulating LDLR, at the European Society of Gene and Cell Therapy (ESGCT) Congress. The presentation will cover in vivo data from studies in mice and non-human primates. Additionally, management will participate in upcoming investor conferences in October to discuss genetic medicines and genome editing technologies.

Key Highlights

  • Editas Medicine to present on EDIT-401 at the ESGCT Congress.
  • EDIT-401 is a CRISPR gene editing medicine for lowering LDL-cholesterol.
  • The presentation will include in vivo data from mice and non-human primates.
  • Editas Medicine management will participate in upcoming investor conferences.

Incidence and Prevalence

Global Estimates of High Cholesterol Prevalence

According to the Global Burden of Disease 2021 study, hypercholesterolemia ranks among the five common metabolic diseases presenting a significant global health challenge. The burden of hypercholesterolemia has increased 1.6-fold to 3-fold over the past three decades (1990-2021), though the rate of increase is slowing with an annual percentage change of -0.33%.

The highest absolute burden of hypercholesterolemia is found in the most populous countries: India, China, and the United States, while the highest relative burden is concentrated in Oceania Island states.

Recent studies reveal varying prevalence rates across different regions:

In China, a 2020 study found a high prevalence of dyslipidemia (42.84%) among people aged 45 years and above, with low HDL-C being the most common type. The awareness, treatment, and control rates among dyslipidemic subjects were 20.27%, 14.41%, and 4.94% respectively. A 2018 study of Chinese coal miners reported an even higher overall prevalence of 68.28%. By 2010, hypercholesterolemia caused 281,000 deaths and 5.912 million disability-adjusted life years (DALYs) in China, accounting for 3.4% of total deaths.

In Brazil, the prevalence of self-reported high cholesterol increased from 12.5% in 2013 to 14.6% in 2019 among adults.

A 2024 study reported dyslipidemia prevalence of 72.39% in a population aged 35-65 years, with significantly higher rates in women than men.

In Japan, a 2018 study found hypercholesterolemia prevalence of 21.5% in men and 31.0% in women, with over half not receiving medication.

Among people living with HIV/AIDS on second-line antiretroviral therapy in central China, hyperlipidemia prevalence was 33.9% in 2018, with female patients showing a higher rate of 37.0%.

A 2018 study in Iran found borderline cholesterol prevalence of 39.8% in males and 46.3% in females, with high cholesterol affecting 13.1% of men and 18.0% of women.

In Senegal, a 2015 study reported isolated hypercholesterolemia at 60.91%, with higher rates in women (66.22%) than men (52.01%).

A 2012 study in Kuwait showed hypercholesterolemia prevalence peaked in 2006-2007 (56.0% in men; 53.6% in women) before declining significantly in 2008-2009 (33.7% in men; 30.6% in women).

In Tunisia, a 2010 study reported hypercholesterolemia prevalence of 8.4% with borderline high cholesterol at 17%.

Demographic patterns show women have approximately 60% higher probability of high cholesterol diagnosis than men, with frequency increasing with age up to 59 years. A 2014 study noted that almost 25% of men and 42% of women older than 65 years have total cholesterol levels greater than 240 mg/dL.

Despite rising obesity rates, some countries like Australia have reported declining mean cholesterol levels over time.

Key Unmet Needs and Target Populations for High Cholesterol Management

High-Risk Patient Populations

Familial Hypercholesterolemia (FH) remains a significant unmet need, with FH patients being at very high risk of cardiovascular disease. FH-Phenotype is associated with increased all-cause and atherosclerotic cardiovascular disease (ASCVD) mortality, with hazard ratios of 1.74 for all-cause death and 2.18 for ASCVD. Women under 50 years with FH-Phenotype face particularly high risk (hazard ratio: 5.27 for all-cause death).

Despite optimal statin therapy, a significant residual ASCVD risk persists, creating an unmet clinical need for novel lipid-lowering agents that can target low-density lipoprotein cholesterol (LDL-C) and other atherogenic particles. Desired values of LDL cholesterol have become progressively lower in recent decades, yet conventional therapies often fail to achieve these targets in many patients.

Specific Target Populations

  • Patients with ASCVD or heterozygous FH whose LDL-C levels remain high despite maximally tolerated lipid-modifying therapies
  • Homozygous FH patients requiring specialized center referral
  • High-risk patients with elevated TG-rich lipoproteins and low HDL-C levels who exhibit persistent cardiometabolic risk despite controlled LDL-C
  • Patients with statin intolerance, poor adherence, or inadequate treatment efficacy
  • Patients with atherogenic dyslipidemia characterized by high triglycerides, low HDL-C, elevated LDL-C, and high apoB:apoA1 ratios
  • Cancer patients receiving immune checkpoint inhibitors (ICIs) who develop dyslipidemia, with no specific guidelines currently available
  • Patients with metabolic syndrome (MetS) lacking optimal pharmacotherapy for their multifaceted risk factors
  • Ultra-rare populations like those with glycogen storage disease type 1b (GSD1b) who present with hyperlipidemia

Age-Specific Considerations

For elderly patients (65+ years), the rationale for treating hypercholesterolemia is less clear than in middle-aged patients, with weaker associations between hypercholesterolemia and cardiovascular risk as age increases. While statins have shown efficacy in elderly populations, convincing data for subjects over 80 are lacking.

For pediatric patients, recent guidelines recommend screening children aged 8-10 years to increase FH diagnosis. However, long-term safety data on statin use in young children are not available, and benefits are based on proxy measures rather than reduction in cardiovascular events.

Access and Diagnostic Challenges

Familial hypercholesterolemia remains vastly underdiagnosed worldwide. Barriers to cascade testing of relatives persist despite improvements in gene technology and decreased costs of genetic testing.

For rural populations, decreased access to specialty care potentially limits appropriate treatment of severe hypercholesterolemia. Studies show patients seen by cardiology were more likely to receive appropriate treatment (statins, PCSK9 inhibitors) and achieve better LDL-C control compared to those without specialty care access.

The COVID-19 pandemic created additional challenges in managing FH patients, with potential impaired access to routine FH services, highlighting the importance of continuing lipid-lowering therapy during infections.

Company drugs in pipeline

Editas Medicine Drug Pipeline Indications

I don't have current information about Editas Medicine's drug pipeline indications. To get accurate and up-to-date information about Editas Medicine's pipeline, I recommend:

  1. Visiting the official Editas Medicine website (www.editasmedicine.com), which typically maintains a current "Pipeline" or "Programs" section detailing their drug candidates and targeted indications

  2. Reviewing their latest investor presentations and quarterly reports, which often contain the most recent updates on clinical programs

  3. Checking clinical trial databases like ClinicalTrials.gov where you can search for trials sponsored by Editas Medicine

  4. Reading recent press releases from the company announcing clinical trial initiations, results, or regulatory updates

  5. Consulting financial analyst reports that cover Editas Medicine (NASDAQ: EDIT)

As a gene editing company utilizing CRISPR technology, Editas Medicine typically focuses on developing treatments for genetic diseases and potentially oncology indications, but the specific programs and their development stages would need to be verified through the sources mentioned above.

For the most accurate information about which indications Editas Medicine currently has drugs in pipeline for, including the clinical trial status of each candidate and their therapeutic areas, please consult these official sources.