Cystinosis - Pipeline Insight, 2024

This “Cystinosis- Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Cystinosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Cystinosis: Understanding

Cystinosis: Overview

Cystinosis is a rare, multisystem genetic disorder characterized by the accumulation of an amino acid called cystine in different tissues and organs of the body including the kidneys, eyes, muscles, liver, pancreas and brain. Generally, cystinosis is broken down into three different forms known as nephropathic cystinosis, intermediate cystinosis and non-nephropathic (or ocular) cystinosis. The age of onset, symptoms, and severity of cystinosis can vary greatly from one person to another. Nephropathic cystinosis presents in infancy and is the most common and severe form. Early detection and prompt treatment are critical in slowing the development and progression of symptoms associated with cystinosis. The kidneys and eyes are the two organs most often affected. Individuals with nephropathic or intermediate cystinosis ultimately require a kidney transplant. Non-nephropathic cystinosis only affects the corneas of the eyes. Cystinosis is caused by mutations of the CTNS gene and is inherited as an autosomal recessive disease. At one time, nephropathic cystinosis was fatal at a very young age. However, the development of a medication known as cysteamine (which lowers the levels of cystine in the cells of the body) and improvements in kidney transplantation have transformed cystinosis from a fatal kidney disorder to a chronic, multisystem disorder with a life expectancy well into adulthood and even beyond 50 years of age. The specific symptoms and severity of cystinosis vary greatly from one person to another based upon several factors including age of onset and whether the disorder is promptly diagnosed and treated. The progression of the disorder can be slowed by early diagnosis and treatment. Eventually, cystinosis can affect all tissues of the body. The age of onset for different symptoms varies greatly. All types of cystinosis are caused by mutations of the CTNS gene. The disease is inherited in an autosomal recessive fashion. Recessive genetic disorders occur when an individual inherits a mutation in the same gene from each parent. If an individual receives one normal gene and one gene for the disease, the person will be a carrier for the disease, but usually will not show symptoms. Carriers for cystinosis (e.g., parents of affected individuals) never have symptoms of the disease. The risk for two carrier parents to both pass the defective gene and, therefore, have an affected child is 25 percent with each pregnancy. The risk to have a child who is a carrier like the parents is 50 percent with each pregnancy. The chance for a child to receive normal genes from both parents and be genetically normal for that particular trait is 25 percent. The risk is the same for males and females. Cystinosis affects males and females in equal numbers. The disorder is estimated to occur in 1 in 100,000-200,000 people in the general population. Cystinosis has been reported worldwide, in all ethnic groups.

Cystinosis- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cystinosis pipeline landscape is provided which includes the disease overview and Cystinosis treatment guidelines. The assessment part of the report embraces, in depth Cystinosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Cystinosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Cystinosis R&D. The therapies under development are focused on novel approaches to treat/improve Cystinosis.

Cystinosis Emerging Drugs Chapters

This segment of the Cystinosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Cystinosis Emerging Drugs

AVR RD 04: AVROBIOAVROBIO’s investigational gene therapy for cystinosis is being studied in a Phase 1/2 investigational trial sponsored by the University of California, San Diego. The first patient was dosed in October 2019. The single-arm trial is expected to enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis.

NP-3: Nacuity Pharmaceuticals Nacuity has developed an oral dosage form containing a small molecule antioxidant for the treatment of nephropathic cystinosis. In vitro POC, chronic oral toxicology studies and a Phase 1 clinical trial in healthy volunteers have been successfully completed. Clinical development program is planned. This program has been granted orphan and rare pediatric disease designations; thus, an eligible new drug application would result in Nacuity receiving seven years of U.S. FDA regulatory exclusivity and potentially a priority review voucher.

Cystinosis: Therapeutic Assessment

This segment of the report provides insights about the different Cystinosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Cystinosis

There are approx. 5+ key companies which are developing the therapies for Cystinosis. The companies which have their Cystinosis drug candidates in the most advanced stage, i.e. phase I/II include, AVROBIO.

Phases

This report covers around 5+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Cystinosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Cystinosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cystinosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Cystinosis drugs.

Cystinosis Report Insights

• Cystinosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Cystinosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Cystinosis drugs?
• How many Cystinosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cystinosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cystinosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Cystinosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• AVROBIO
• Birch Therapeutics
• Nacuity Pharmaceuticals

Key Products

• AVR RD 04
• BRH101
• NP-3

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