Innovation in Early Clinical Research


Streamlining Clinical Development with Clinical and Regulatory Expertise

Early Clinical Research from Preclinical through Phase I and POC studies

We specialize in strategy and provide early clinical research services to enable informed, timely decision-making for our clients.

BBCR Consulting offers clinical, regulatory, translational, and biomarker consulting services that support our clients’ needs. Our process is designed to maximize time and cost efficiencies while mitigating risk.

Our mission is to support domestic and international pharma, biotech, and device companies by nurturing their products’ strengths while improving efficiency and safety.

To support innovative and repurposing treatments for orphan diseases

Developing innovative or repurposed drugs for orphan diseases can be rewarding, but navigating the challenges is not for the faint of heart.

Expert guidance is essential in an area where patients are few. A lack of previous studies may hamper progress as you mount an orphan petition and negotiate a clinical plan with the FDA.

Our regulatory and clinical research consultants are experts in biologics, cell and gene therapies, and small molecule developments. Identifying areas of need or economic interest can help sponsors find homes for treatments for orphan indications.

BBCR consultants have the experience to guide you through the Orphan clinical research process with a clinical plan and regulatory strategy for accelerated approval. Our team will empower you with medical insight, practical regulatory, translational medicine, and clinical research expertise, and streamlined clinical trials.

Rare Diseases Experience

  • Acute Myeloid Leukemia (AML)
  • Amyotrophic Lateral Sclerosis (ALS)
  • Biliary Atresia
  • CDKL5 Deficiency Disorder
  • Cholesteryl Ester Storage Disease (CESD)
  • Cystic Fibrosis
  • Cutaneous T cell Lymphoma
  • Duchenne Muscular Dystrophy (DMD)
  • Fabry Disease
  • Familial hypercholesterolemia (FH)
  • Gaucher’s Disease
  • Glycogen Storage Disease (GSD)
  • Head & Neck HNSCC
  • Hemorrhagic Cystitis
  • Hereditary Angioedema
  • IN1-Negative tumors
  • Kawasaki Disease
  • MP1-CDG
  • Multiple Myeloma
  • Niemann-Pick disease type C (NPC)
  • Neurotropic Keratitis
  • Ovarian Cancer
  • Pancreatic Cancer
  • Paraganglioma
  • Pemphigus
  • PGM1-CDG
  • Phenylketonuria (PKU)
  • Plexiform Neurophibromas
  • Prader-Willi Syndrome (PWS)
  • Primary Hyperoxaluria (PH)
  • Primary Immunodeficiency (WHIM)
  • Progressive familial intrahepatic cholestasis (PFIC)
  • Prader-Willi Syndrome (PWS)
  • Radiation Dermatitis
  • Recurrent Clostridium Difficile Infection
  • Renal Cell Carcinoma
  • Sickle Cell Disease
  • SLC35C1-CDG
  • Status epilepticus
  • STXBP1 Encephalopathy
  • Subtype Small Cell Lung Cancer (SCLC)
  • Synovial Sarcoma
  • Tuberous Sclerosis Complex


Strategy and Target Product Profile

A comprehensive development strategy provides a detailed roadmap for advancing a new compound from the lab through each stage of development. TPP facilitates communication among the sponsor, the FDA, and stakeholders in and outside the industry.

Translational Medicine and Epigenetics

A translational medicine program includes  adopting biomarkers to mitigate Phase I and II clinical risks, identifying patient subpopulations, facilitating decision-making, and accelerating  drug commercialization. BBCR’s epigenetic expert, Dr. Claudio Carini, has been a pioneer in leading major pharmaceutical companies’ efforts in epigenetics.

Biologics, Cell and Gene Therapy

Cell and gene therapy are the new frontiers in the fight against devastating diseases, including rare diseases and cancers.

Real World Evidence

Real-world evidence (RWE) is increasingly important in clinical trial design and drug approval. RWE is clinical evidence related to patient health status and treatment safety and efficacy generated in clinical practice.

Repurposing for New Indications

Drug repurposing offers your product shorter journeys to the clinics. It is especially crucial to rare and neglected diseases, cancers, and neurodegenerative diseases. The BBCR consultants, experienced in orphan, rare and ultra-rare diseases, have advised biotech and venture capitalists for new indications evaluation and prioritization.

Let’s Work Together

BBCR is committed to meeting the challenges of sponsors and investors seeking a clear path to market.

We invite you to reach out today to learn more.


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