uBriGene offers full regulatory support, from pre-IND to BLA, DMFs, and expedited programs, with expertise across the US, China, EU, and more.
uBriGene’s experienced regulatory affairs team supports the entire lifecycle of advanced therapy product development, from pre-IND to BLA, as well as DMFs in various countries, including the United States, Canada, China, the European Union, and Australia.
As a leading ATMP CDMO provider, uBriGene offers regulatory services for CMC sections as part of its CDMO service package.
From pre-IND to BLA, DMFs, and expedited programs
Expertise across the US, China, EU, Canada, Australian, and more
With extensive experience across international regulatory authorities in advanced therapies, we help you navigate the complexities of the regulatory landscape. Our regulatory services support submissions from pre-IND and IND to pre-BLA, BLA, and beyond.
Key services include:
Risk assessment and management
Preparation and submission of expedited program and designation applications (ODD, FTD, BTD, RMAT, PRIME) to US FDA, China NMPA, and EMA
Regulatory support for CMC sections of pre-IND, IND, pre-BLA, and BLA submissions, including IMPDs
CTD/eCTD-based submissions to US FDA, China NMPA, Health Canada, and EMA
DMF applications
Amendments and supplements for CMC changes, covering clinical trials (especially pivotal trials) and post-marketing approvals.
Provide guidance through the complex, ever-evolving regulatory landscape
Ensure compliance with regional regulatory guidelines and legislation
Mitigate regulatory risks
Reduce potential costs in the later stages of CMC development
Accelerate development and bring therapies to market faster
In the competitive pharmaceutical industry, everyday counts—especially once your product has demonstrated efficacy in clinical studies. A one-month delay in obtaining marketing licensure can mean the difference between being first-in-class or second-in-class.
uBriGene’s regulatory team can help assess the feasibility of obtaining Fast Track Designation (FTD) and Regenerative Medicine Advanced Therapy (RMAT) from the US FDA, Breakthrough Therapy Designation from both the China NMPA and the FDA, and Priority Medicines (PRIME) status from the EMA.
We assist in submitting expedited program applications and liaising with health authorities to secure these designations. These programs offer more frequent interactions with health authorities during development and allow for rolling submissions of your BLA.
Investigator-initiated trials (IITs) are clinical studies designed, conducted, and managed by independent investigators—typically physicians or researchers—using GMP-like materials. These studies offer valuable clinical data on the safety and efficacy of a potential therapeutic program. IITs are often the fastest and most cost-effective route to achieving clinical proof-of-concept, at a fraction of the cost of traditional clinical trials.
uBriGene can support IIT studies, with the resulting data serving as a key factor in determining whether to continue developing your investigational drug. GCP-compliant IIT data can also help reduce the sample size needed for future IND trials, accelerating the development process.
Request a ConsultationSeveral critical raw materials from uBriGene (lentiviral helper plasmids, AAV helper plasmids, 293XS cell banks, and HEK293TH cell banks) have DMF on file with the FDA. These DMF numbers can be directly referenced, reducing the review and evaluation process for related drug projects.
| Item | Application | DMF record |
|---|---|---|
| GMP grade lentiviral packaging plasmids | GMP Lentivirus production | DMF 27777 |
| GMP grade AAV packaging plasmids | GMP AAV Manufacturing | DMF 28890 |
| 293XS cell banks | GMP AAV Manufacturing | DMF 28844 |
| HEK293TH cell banks | GMP Lentivirus production | DMF 29108 |
| GMP grade Ubrinuclease | Remove nucleic acid removal during GMP production | DMF 29414 |
| GMP grade Cas9 proteins, mRNA | Gene editing | DMF 29884 |
| 293TH Cell Banks | GMP Lentivirus production | DMF 31873 |
| Human Mesenchymal Stem Cell Exosomes | GMP MSC Manufacturing | DMF 31873 |
| Control of plasmids and plasmid bank | GMP Plasmid Manufacturing | DMF 30703 |
| Control of AAV and AAV critical intermediates | GMP AAV Manufacturing | DMF 30700 |
| AAV packing RC plasmid | GMP AAV Manufacturing | DMF 32370 |
| uBriGene (Germantown site) GMP Cell and Gene Facility | GMP Cell and Gene Manufacturing | DMF 31266 |
*Outsourced testing
Learn about CAR-T production process & regulatory guidelines.
Discover Lentivirus (LVV) production processes and testing.
Learn more details of our RNA-LNP manufacturing and capabilities.
Discover how uBriGene helped our client to receive FDA IND clearance in just nine months.
Yes, we offer regulatory affairs services at any stage of your product development, regardless of whether we manufacture your products.
The timeline depends on product complexity and data availability. uBriGene can begin preparing the pre-IND and IND packages alongside CMC, nonclinical, and clinical studies. A first draft can include placeholders while data are pending, and a near-final version can be completed within two weeks once all required data are available. This is the document preparation timeline, not the regulatory review or approval timeline.
Data requirements vary by designation and regulatory authority. uBriGene supports applications for programs including FDA Fast Track, Breakthrough Therapy, and RMAT designations, as well as China NMPA Breakthrough Therapy and EMA PRIME. Eligibility and supporting evidence are assessed on a case-by-case basis; clinical data requirements depend on the specific program.
An investigator-initiated trial (IIT) is designed and conducted by an independent investigator or institution, while a sponsor-led IND trial is conducted under a sponsor's regulatory responsibilities. Both require appropriate ethical, safety, and regulatory oversight. Product quality, nonclinical evidence, and submission requirements depend on the jurisdiction, product, and study design; an IIT does not automatically have lower requirements.
uBriGene provides regulatory affairs support for cell and gene therapy development, including pre-IND consultation, IND and BLA submissions, CMC documentation, DMF preparation, CTD/eCTD dossier support, and regulatory authority interactions. Services can be provided independently of uBriGene’s manufacturing services.
Yes. uBriGene supports Common Technical Document (CTD) and electronic CTD (eCTD) preparation for regulatory submissions. Support includes organizing technical documentation and coordinating relevant CMC, nonclinical, and clinical information according to the applicable regulatory requirements.
uBriGene supports applications for expedited development programs, including FDA Fast Track and Breakthrough Therapy designations, Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., China NMPA Breakthrough Therapy designation, and the European Medicines Agency’s PRIME scheme. Eligibility criteria and evidence requirements vary by program and product.
CMC manufacturing & testing
Our experienced RA team provides comprehensive regulatory filing support, offering guidance from preclinical stages to commercialization.
