Bardet-Biedl Syndrome Pipeline 2025: Pioneering Clinical Developments by 8+ Global Leaders - DelveInsight | Featuring Rhythm Pharmaceuticals

DelveInsight's, "Bardet-Biedl Syndrome - Pipeline Insight, 2025," report provides comprehensive insights about 1+ companies and 1+ pipeline drugs in Bardet-Biedl 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.
With Bardet-Biedl Syndrome (BBS) increasingly impacting populations worldwide and contributing to comorbidities such as diabetes, cardiovascular disease, and certain cancers, the need for safer and more effective therapies is growing. DelveInsight reports that the BBS pipeline includes 8+ pharmaceutical and biotech companies developing 1+ therapeutic candidates for Bardet-Biedl Syndrome. These treatments are progressing through various stages of clinical and preclinical development, highlighting significant innovation and dedication to addressing this critical health challenge.

DelveInsight's "Bardet-Biedl Syndrome Pipeline Insight 2025" delivers a comprehensive analysis of the current R&D landscape, covering clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report is an essential resource for researchers, investors, and decision-makers seeking insights into the evolving Bardet-Biedl Syndrome therapeutics market and the breakthroughs shaping its future.

Explore the Cutting-Edge Landscape of Bardet-Biedl Syndrome Drug Development @ https://www.delveinsight.com/report-store/bardet-biedl-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

During the ERS Congress 2025 in September 2025, several biotechnology and pharmaceutical companies shared notable advancements in pulmonary and cardiopulmonary research:

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In September 2025, Diagonal Therapeutics presented new preclinical data highlighting the potential of its lead clustering antibody, DIAG723, as a disease-modifying therapy for pulmonary arterial hypertension (PAH).

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In September 2025, a study revealed that babies raised with dogs may have a reduced risk of developing childhood asthma.

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In September 2025, Savara shared data from the Phase 3 IMPALA-2 trial of Molgramostim Inhalation Solution for patients with autoimmune pulmonary alveolar proteinosis (aPAP).

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In September 2025, AllRock Bio, Inc. reported encouraging Phase 1 results for ROC-101, a first-in-class oral pan-ROCK inhibitor for cardiopulmonary and fibrotic diseases.

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In September 2025, Gossamer Bio, Inc., in collaboration with Chiesi Group, announced five scientific presentations on seralutinib for PAH and PH-ILD.

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In September 2025, Boehringer Ingelheim presented pooled analyses from the Phase III FIBRONEER Trademark program on nerandomilast, an investigational oral PDE4B inhibitor, showing a nominally significant reduction in mortality risk across IPF and PPF.

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In September 2025, Kaia Health launched a pilot program of Kaia Breathe, a digital pulmonary rehabilitation solution for COPD patients, in partnership with MedImprove.

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In September 2025, aTyr Pharma, Inc. shared additional findings from the Phase 3 EFZO-FIT Trademark study of efzofitimod in 268 patients with pulmonary sarcoidosis, a key form of interstitial lung disease.

Key Takeaways from the Bardet-Biedl Syndrome Pipeline Report

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DelveInsight's Bardet-Biedl Syndrome pipeline report highlights a dynamic landscape with 8+ active companies developing 1+ therapeutic candidates for the treatment of Bardet-Biedl Syndrome.

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In December 2024, the U.S. Food and Drug Administration (FDA) approved an expanded indication for Imcivree (setmelanotide), permitting its use for chronic weight management in adults and children aged 2 years and older with obesity caused by Bardet-Biedl Syndrome. This approval was supported by clinical trial results showing meaningful reductions in both weight and hunger among BBS patients.

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Key companies, including Rhythm Pharmaceuticals and others, are actively exploring new therapies to enhance the Bardet-Biedl Syndrome treatment landscape. Promising pipeline candidates include Setmelanotide and additional therapies in various stages of development.

Bardet-Biedl Syndrome Overview:

Bardet-Biedl syndrome (BBS) is a rare, autosomal recessive genetic disorder classified as a ciliopathy. It is associated with mutations in at least 20 genes (BBS1-BBS20) and exhibits an oligogenic inheritance pattern. BBS proteins, located in the centrosome, are essential for the formation and function of cilia. Dysfunction of these proteins impacts multiple organ systems that depend on ciliated cells, resulting in a broad spectrum of symptoms. Clinical signs of BBS usually develop gradually during the first decade of life, with most cases diagnosed in late childhood or early adulthood.

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Bardet-Biedl Syndrome Pipeline Analysis

The Bardet-Biedl Syndrome pipeline insights report 2025, provides insights into:

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Provides comprehensive insights into key companies developing therapies in the Bardet-Biedl Syndrome Market.

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Categorizes Bardet-Biedl Syndrome therapeutic companies by development stage: early, mid, and late-stage.

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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

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Reviews emerging Bardet-Biedl Syndrome drugs under development based on:

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Stage of development

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Bardet-Biedl Syndrome Route of administration

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Target receptor

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Monotherapy vs. combination therapy

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Bardet-Biedl Syndrome Mechanism of action

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Molecular type

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Offers detailed analysis of:

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Company-to-company and company-academia collaborations

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Bardet-Biedl Syndrome Licensing agreements

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Funding and investment activities supporting future Bardet-Biedl Syndrome market advancement.

Unlock key insights into emerging Bardet-Biedl Syndrome therapies and market strategies here: https://www.delveinsight.com/report-store/bardet-biedl-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Bardet-Biedl Syndrome Emerging Drugs

Setmelanotide: Rhythm Pharmaceuticals

Setmelanotide is an advanced investigational therapy that functions as a melanocortin-4 receptor (MC4R) agonist. It aims to restore the activity of the MC4R pathway, which can be disrupted by genetic mutations upstream of the receptor, thereby helping to curb excessive appetite and support weight loss. The U.S. FDA has granted Setmelanotide Breakthrough Therapy Designation for the treatment of Bardet-Biedl syndrome (BBS), and it has also received Orphan Drug Designation from both the FDA and the European Commission for this condition.

Bardet-Biedl Syndrome Pipeline Therapeutic Assessment

Bardet-Biedl Syndrome Assessment by Product Type

- Mono

- Combination

- Mono/Combination

Bardet-Biedl Syndrome By Stage

- 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

Bardet-Biedl Syndrome Assessment by Route of Administration

- Oral

- Parenteral

- Intravenous

- Subcutaneous

- Topical

Bardet-Biedl Syndrome Assessment by Molecule Type

- Recombinant fusion proteins

- Small molecule

- Monoclonal antibody

- Peptide

- Polymer

- Gene therapy

Download sample pages to get an in-depth assessment of the emerging Bardet-Biedl Syndrome therapies and key Bardet-Biedl Syndrome companies [https://www.delveinsight.com/sample-request/bardet-biedl-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Table of Contents

1. Report Introduction

2. Executive Summary

3. Bardet-Biedl Syndrome Current Treatment Patterns

8. Bardet-Biedl Syndrome - DelveInsight's Analytical Perspective

5. Therapeutic Assessment

1. Bardet-Biedl Syndrome Late-Stage Products (Phase-III)

7. Bardet-Biedl Syndrome Mid-Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

1. Inactive Products

11. Dormant Products

12. Bardet-Biedl Syndrome Discontinued Products

13. Bardet-Biedl Syndrome Product Profiles

18. Bardet-Biedl Syndrome Key Companies

15. Bardet-Biedl Syndrome Key Products

11. Dormant and Discontinued Products

17. Bardet-Biedl Syndrome Unmet Needs

18. Bardet-Biedl Syndrome Future Perspectives

19. Bardet-Biedl Syndrome Analyst Review

20. Appendix

21. Report Methodology

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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