22q11.2 Deletion Syndrome - Pipeline Insight, 2024

This “22q11.2 Deletion Syndrome - Pipeline Insight, 2024” report provides comprehensive insights about 2+ companies and 2+ pipeline drugs in 22q11.2 Deletion Syndrome 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

22q11.2 Deletion Syndrome: Understanding

22q11.2 Deletion Syndrome: Overview

22q11.2 Deletion Syndrome, also known as DiGeorge Syndrome, is a rare genetic condition caused by a missing piece of chromosome 22. This syndrome can manifest in various ways, affecting different parts of the body and leading to a range of symptoms. The deletion of a segment on chromosome 22 can result in congenital malformations such as cardiac defects, palatal anomalies, facial dysmorphism, developmental delays, and immune deficiencies. It is characterized by a 3 million base pair deletion on chromosome 22, affecting 30 to 40 genes. This condition is considered a contiguous gene deletion syndrome, with the loss of multiple genes close together contributing to its diverse features. The deletion often includes the TBX1 gene, which plays a crucial role in heart, parathyroid, thymus, and facial structure development.

Symptoms of 22q11.2 Deletion Syndrome can vary from mild to severe and may include congenital heart defects, immune system abnormalities, distinctive facial features, learning disabilities, behavioral issues, and delayed growth and development. Common physical symptoms can involve heart abnormalities, underdeveloped thymus, and decreased calcium levels. Additionally, affected individuals may exhibit unique facial characteristics and may experience issues with speech, hearing, and immune function.

Diagnosing 22q11.2 Deletion Syndrome involves clinical examination and genetic testing to confirm the presence of the deletion. Tests such as fluorescence in situ hybridization (FISH), multiplex ligation-dependent probe amplification (MLPA), or array comparative genomic hybridization (aCGH) are used to detect the deletion. Prenatal diagnosis is possible in familial cases through chorionic villus sampling or amniocentesis.

Management of 22q11.2 Deletion Syndrome is symptom-based and requires a multidisciplinary approach. Treatment may include surgeries to address heart defects or palate issues, feeding support, calcium supplementation, occupational, physical, and speech therapy, as well as educational and behavioral interventions. Regular monitoring of calcium levels, thyroid function, and immune function is essential. Treatment focuses on addressing specific symptoms to improve the quality of life for individuals with this syndrome.

"22q11.2 Deletion Syndrome- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the 22q11.2 Deletion Syndrome pipeline landscape is provided which includes the disease overview and 22q11.2 Deletion Syndrome treatment guidelines. The assessment part of the report embraces, in depth 22q11.2 Deletion Syndrome 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, 22q11.2 Deletion Syndrome 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 22q11.2 Deletion Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve 22q11.2 Deletion Syndrome.

22q11.2 Deletion Syndrome Emerging Drugs Chapters

This segment of the 22q11.2 Deletion Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.

22q11.2 Deletion Syndrome Emerging Drugs

NB-001: Nobias Therapeutics

NB-001 is believed to be a non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs) and is dosed orally. It would be a first-in-class treatment for patients with 22q11DS and certain comorbid psychiatric conditions, including anxiety, attention-deficit/hyperactivity disorder, and autism. Results from the multi-center, randomized, placebo-controlled crossover trial demonstrate the safety and tolerability of NB-001 Currently, the drug is in Phase II stage of its clinical trial for the treatment of 22q11DS.

22q11.2 Deletion Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different 22q11.2 Deletion Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in 22q11.2 Deletion Syndrome

• There are approx. 2+ key companies which are developing the therapies for 22q11.2 Deletion Syndrome. The companies which have their 22q11.2 Deletion Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Nobias Therapeutics.

Phases

DelveInsight’s report covers around 2+ 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

22q11.2 Deletion Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

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

22q11.2 Deletion Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses 22q11.2 Deletion Syndrome 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 22q11.2 Deletion Syndrome drugs.

22q11.2 Deletion Syndrome Report Insights

• 22q11.2 Deletion Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

22q11.2 Deletion Syndrome 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 22q11.2 Deletion Syndrome drugs?
• How many 22q11.2 Deletion Syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of 22q11.2 Deletion Syndrome?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the 22q11.2 Deletion Syndrome 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 22q11.2 Deletion Syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Nobias Therapeutics
• Herophilus

Key Products

• NB-001

Research programme: neuropsychological disorders therapeutics

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