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Our Pipeline

Advancing cell therapies for life-threatening pediatric and aging-related diseases

We are committed to advancing allogeneic cell therapies that have the potential to do more than improve symptoms – they aim to meaningfully change the course of serious diseases and potentially give patients and families more strength, time, and possibility.

Lomecel-B® (laromestrocel) - A pipeline in a product

Global Patient Population
Phase 1
Phase 2
Phase 3
 Milestones
Hypoplastic Left Heart Syndrome
> 30,0001
Phase 2
  • Ph2b enrollment completed, Top-line data anticipated Q3 2026
  • Rare Pediatric Disease, Orphan Drug, Fast Track Designations.
Pediatric Dilated Cardiomyopathy
100,0002
Phase 1
  • IND approved 2025
  • FDA confirmed that single Ph2b/3 study (no Ph1)
Alzheimer’s disease
> 32 Million3
Phase 2
  • Completed through Ph2a
  • RMAT & Fast Track Designations
  • Results published in Nature Medicine (‘25)
Aging-related Frailty
70-100 Million4
Phase 2
  • Completed Ph2b dose-finding trial
  • Results published in Cell Stem Cell ('26)

1 Global patient population estimated using prevalence in first world countries, incidence of 2.6 HLHS patients/10,000 live births and 130 million births per year. U.S. HLHS prevalence is ~1,000 patients per year. E.U., Japan, and China incidence rates range from ~2.1 to 2.8 per 10K live births. Sources: Bakker MK, et al. BMJ Open. 2019;9(7); POLI (GlobalData); U.N. Dept Economic and Social Affairs, 2015; ClearView Analysis, 2022.  2 Malinow I, et al. Front Pediatr. 2024;19(12).  3 Based on 2022 estimate of global persons >50 years of age with AD (32 million) and prodromal AD (69 million), not including preclinical AD, as reported in Gustavsson A, et al. Alzheimer’s Dement. 2023;19(2):658-670.  4 Estimate based calculation using global incidence of frailty among community-dwelling adults 60 years or older reported as 43.4 new cases per 1000 person-year, as reported in Ofori-Asenso R, et al. JAMA Network Open 2019;2(8), and global population size of 8.1 billion persons (Pew Research Center, 2025).

Therapeutic Areas

Hypoplastic Left Heart Syndrome (HLHS)

A rare and devastating congenital heart defect with limited long-term treatment options.

  • ~1,000 babies are born each year in the U.S. with HLHS
  • The left side of the heart fails to develop normally, leaving the right ventricle (RV) to support systemic circulation
  • Overall survival to adolescence is estimated at only 50–60%
  • Progressive deterioration of the systemic RV is a major driver of heart failure, transplant, and death
  • Even with surgical palliation, RV overload leads to increased mortality, delayed development, and long-term organ dysfunction

Selected sources: Newburger et al., Circulation (2018); Siffel et al., Pediatrics (2015); Stasik et al., J Thorac Cardiovasc Surg (2006); CDC; Lynch et al., JACC: Advances (2024).

Infant lying on a white bed and looking upward.

Young child sitting on a white blanket surrounded by toys and books.

Pediatric Dilated Cardiomyopathy (PDCM)

A rare, progressive heart disease with high mortality in children.

  • Affects an estimated 100,000 children worldwide
  • ~40% of children require heart transplant or die within two years of diagnosis
  • Current therapies focus on symptom management rather than disease modification
  • Disease is marked by cardiomyocyte loss and fibrotic remodeling
  • Preclinical and clinical evidence suggests MSCs may promote cardiomyogenesis, reduce inflammation and fibrosis, and support neovascularization

Selected sources: Lesizza et al. (2019); Lipshultz et al., Circulation (2018); Pittenger et al., NPJ Regenerative Medicine (2019).

Older adult smiling while receiving supportive care from a clinician.

Alzheimer’s disease (AD)

A progressive neurodegenerative disease with growing global impact.

  • The most common form of dementia, characterized by progressive loss of memory, cognition, and functional independence
  • Symptoms typically begin in the mid-60s and worsen over time
  • More than 6 million Americans aged 65+ are currently living with Alzheimer’s
  • Prevalence is expected to rise significantly as the population ages

Source: National Institute on Aging.

Older adult smiling during a conversation over coffee.

Aging-related Frailty (AF)

A chronic condition driven by systemic physiologic decline.

  • Characterized by reduced reserve and resilience across multiple organ systems
  • Common features include weakness, fatigue, weight loss, slowness, and reduced activity
  • Associated with higher risk of falls, fractures, infections, hospitalization, and mortality
  • No FDA-approved therapies currently exist
  • Estimated prevalence of ~15% in adults over 65 using CHS criteria
  • Aging-related Frailty does not yet have a consensus regulatory definition.

Source: Bandeen-Roche et al., J Gerontol A (2015).

Clinical Progress & Regulatory Momentum

Therapies for life-threatening pediatric and chronic aging-related conditions.

 

Positive Clinical Data
  • Positive initial results across 5 clinical trials
  • Clinical experience spanning 3 distinct indications
  • Well-established safety profile to date
Regulatory Pathways to BLAs
  • HLHS: Positive FDA Type C meeting; ongoing trial
  • Alzheimer’s disease: Positive FDA Type B meeting; planned single Phase 2/3 trial
FDA Designations (5 Total)
  • HLHS: Orphan Drug, Fast Track, Rare Pediatric Disease
  • Alzheimer’s disease: RMAT, Fast Track
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