Expertise
Specialized Regulatory Consulting
Cell & Gene Therapy
Regulatory and development strategy for AAV, CAR-T, CRISPR, epigenetic products, and other advanced therapy and regenerative medicine products.
Orphan Drug Development
Consilidated experience in orphan drug regulations, small trial sizes, and overcoming challenges faced in rare disease drug development.
Pediatric Therapeutics
Support for pediatric strategy and planning, PIPs, and rare pediatric disease programs.
Cell and Gene Therapy Consulting Services
A Team Of Experts Advising On Cellular, Tissue, And Gene Therapy Programs
At RareMoon Consulting, we are recognized leaders in cell and gene therapy consulting, with consolidated, hands-on experience guiding advanced therapy programs through every stage of development. Since 2016, our team has supported hundreds of cell and gene therapy (CGT) programs, navigating regulatory requirements for complex products such as CAR T-cell therapy, AAV gene therapies, and gene editing platforms.
RareMoon's consolidated cell and gene therapy (CGT) expertise is central to a powerhouse team of FDA and industry veterans with decades of hands-on experience advancing complex programs. Our senior regulatory scientists and clinical consultants bring insight from across the development lifecycle, including a pediatrician-geneticist and former FDA/CBER reviewer, an endocrinologist with experience in general medicine and both CBER and CDER, and a solid tumor oncologist who previously served at FDA/CBER. Our nonclinical team comprises senior pharmaceutical and toxicology experts from both the FDA and industry, while our CMC leadership includes gene therapy specialists and former EMA/CHMP/CAT regulators. Global strategic guidance is provided by senior EU regulatory consultants and a dedicated PIP expert with expertise in gene therapy and prior experience at the EMA/PDCO. Supported by a senior biostatistician with experience in rare disease and CGT trials, our team was purpose-built to guide advanced therapies through complex regulatory environments with precision, speed, and strategic clarity.
From IND-enabling strategy and regulatory submissions to interactions with the FDA, EMA, and global agencies, we help sponsors align innovation with regulatory expectations to accelerate timelines, reduce risk, and improve the likelihood of successful product approval.
Example of Therapeutic Areas We've Supported
- Rare genetic disorders (including ultra-rare conditions)
- Pediatric diseases and inherited metabolic disorders
- Hematologic diseases (e.g., hemophilia, sickle cell disease, beta-thalassemia)
- Neuromuscular disorders (e.g., Duchenne muscular dystrophy, SMA, Friedreich's ataxia)
- Central nervous system (CNS) disorders (e.g., Rett syndrome, epilepsy syndromes, leukodystrophies)
- Lysosomal storage disorders (e.g., Fabry, Pompe, MPS I–III)
- Ophthalmologic diseases (e.g., Stargardt disease, LCA, retinitis pigmentosa)
- Pulmonary diseases (e.g., cystic fibrosis)
- Oncology (solid tumors and hematologic malignancies, including pediatric cancers)
- Autoimmune disorders
- Endocrine and metabolic diseases (e.g., congenital adrenal hyperplasia)
- Dermatology (e.g., epidermolysis bullosa)
Examples of Products We've Support
- Cell therapies (e.g., CAR-T, gene-edited cell therapies)
- Gene therapies (e.g., AAV, dual-AAV, lentiviral vectors)
- Non-viral gene therapies (e.g., LNPs, electroporation-based delivery)
- Gene editing (e.g., CRISPR-Cas9, base editing, prime editing)
- Epigenetic therapies (e.g., CRISPR-based gene regulation platforms)
- RNA therapeutics (e.g., non-viral gene therapy)
- Tissue-Engineered Products (e.g., Regenerative medicine platforms)
Rare Disease and Orphan Drug Development Expertise
Strategy And Execution For Rare Disease Product Development And Regulatory Success
Supporting rare disease programs is the foundation of RareMoon. Since our inception in 2016, we've specialized in guiding orphan drug development across a range of modalities, regulatory pathways, and global markets. Our small, senior team works closely with your internal teams to develop submissions that withstand regulatory scrutiny while supporting your commercial and scientific objectives.
We partner with clients early to ensure regulatory strategies are aligned with both development goals and agency expectations, streamlining the path to designation and regulatory milestones. We bring deep expertise in orphan drug strategy, including global harmonization and tailored regulatory planning for programs with limited patient populations, as well as clinical trial design refined through decades of experience in supporting rare disease indications. From feasibility and designation strategy to regulatory submissions and agency engagement, RareMoon helps sponsors de-risk development, align with regulatory expectations, and bring life-changing therapies to the patients who need them most.
Examples of Therapeutic Modalities We've Supported
- Small molecules (e.g., genetic therapies)
- Repurposed drugs
- Antisense oligonucleotides (e.g., ASOs)
- RNA therapeutics (e.g., siRNA)
- Gene therapies and gene-editing technologies (e.g., viral and non-viral, CRISPR/Cas9)
- Monoclonal antibodies (mAbs)
- Peptide therapeutics (e.g., synthetic peptides)
- Cell therapies (e.g., CAR-T, autologous or allogeneic cell-based therapies)
- Immunotherapies (e.g., checkpoint inhibitors)
- Combination products
Examples of Therapeutic Areas We've Supported
- Rare genetic disorders (including ultra-rare and undiagnosed conditions)
- Pediatric diseases and inherited metabolic disorders
- Neuromuscular disorders (e.g., Duchenne muscular dystrophy)
- Central nervous system (CNS) disorders (e.g., epilepsy syndromes)
- Primary headache disorders (e.g., Trigeminal neuralgia)
- Lysosomal storage disorders (e.g., Fabry, Pompe, MPS I)
- Pulmonary diseases (e.g., cystic fibrosis)
- Oncology (solid tumors and hematologic malignancies, including pediatric cancers)
- Autoimmune disorders (e.g., dermatomyositis, Systemic Sclerosis)
- Gastrointestinal diseases (e.g., NEC)
- Dermatology (e.g., Netherton)
Pediatric Therapeutics Consulting
Expert Guidance For Pediatric Drug Development, Including Global Pediatric Regulatory Requirements
Pediatric development introduces additional complexity to any program, particularly in the context of rare diseases and advanced therapies. RareMoon supports sponsors in designing and justifying pediatric strategies that meet FDA and EMA expectations, including PIP submissions, pediatric study plans (PSP), and rare pediatric disease designations. Backed by a pediatrician and genetics expert, a PIP Expert, and a team with a decade of experience working together on rare and ultra-rare pediatric diseases and regulations, our team is available to support you at any stage of development.
We also advise on ethical and risk considerations specific to pediatric populations, including neonatal and adolescent studies. Our work aims to align your regulatory documentation, justification, and clinical trial plans with scientific standards and evolving global policy requirements.
Our Pediatric Services Include
- Development of Pediatric Investigation Plans (PIPs)
- PIP waivers and modifications
- Preparation of Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Requests
- Age-specific risk/benefit assessment and ethical strategy
- Advising on pediatric components of IND, CTA, and clinical trial protocols
- Development of FDA Pediatric Study Plan (PSP)
- Consultation on FDA Prospect of Direct Benefit (PDB) requirements