Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations KB407 transduction confirmed in all six patients with successful bronchoscopies irrespective of modulator-status; percentage of conducting airway cells transduced in each patient ranged from 29.4% to 42.1% Registrational repeat dosing CORAL-3 study design submitted to FDA in late December; anticipating enrollment in study to start in 1H 2026 following alignment with the FDA Investor call to be he ...

Investor call to be held January 8 at 4:30 pm ET to discuss data update and next stepsPITTSBURGH, Jan. 07, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced that it will host an investor conference call and webcast tomorrow, Thursday, January 8, 2026, at 4:30 pm ET, to disclose an interim clinical update from CORAL-1, the Company’s multi-center, dose escalation Phase 1 study evaluating KB407 in patients with cystic fibrosis. The interim clinical update will focus ...

CHARLESTOWN, Mass., Jan. 06, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, will present at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 5:15 p.m. PT (8:15 p.m. ET). A live webcast of the presentation will be available on the Events page of the Investors section of the Company ...

Prime Medicine, Inc. (NASDAQ:PRME) is among the 12 Best Genomics Stocks to Invest In. Prime Medicine Announces NEJM Publication Of PM359 Data Prime Medicine, Inc. (NASDAQ:PRME) stated on December 7, 2025, that the New England Journal of Medicine has published Phase 1/2 clinical results for PM359, its experimental autologous hematopoietic stem cell therapy for p47phox chronic granulomatous condition. Prime Editing for p47-phox Chronic Granulomatous Disease is a publication that presents preliminary data f ...

NEW YORK, Dec. 22, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today announced that R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics, will present at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 7:30 AM PT in San Francisco, California. The event will be webcast live under the News & Events tab in the Investors section of ...

CHARLESTOWN, Mass., Dec. 16, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today shared that the U.S. Department of Health and Human Services (HHS) officially added Duchenne muscular dystrophy (Duchenne) to the Recommended Uniform Screening Panel (RUSP), the list of conditions recommended for universal newborn screening across the United States. For nearly a dec ...

Core Insights - Lexeo Therapeutics, Inc. is hosting a virtual event focused on managing PKP2-associated arrhythmogenic cardiomyopathy on December 9, 2025 [1][2] - The event will feature Dr. Victoria Parikh, an expert in cardiovascular medicine, who will discuss holistic management approaches for PKP2-ACM [2] - Lexeo is a clinical stage genetic medicine company aiming to innovate treatments for cardiovascular diseases, with a focus on genetic causes [3] Company Overview - Lexeo Therapeutics is based in New York City and is dedicated to reshaping heart health through pioneering science [3] - The company is advancing a portfolio of therapeutic candidates targeting genetic causes of various conditions, including LX2006 for Friedreich ataxia and LX2020 for PKP2 arrhythmogenic cardiomyopathy [3]

Core Insights - Beam Therapeutics Inc. is set to present updated safety and efficacy data for BEAM-101, a gene therapy for sickle cell disease (SCD), at the 67th ASH Annual Meeting in December 2025 [1][2] - BEAM-101 aims to provide a one-time, disease-modifying treatment for patients suffering from severe vaso-occlusive crises associated with SCD [2][3] Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines, utilizing a proprietary base editing technology to develop therapies [5][6] - The company has established a fully integrated platform for gene editing, delivery, and manufacturing capabilities [5] Product Details - BEAM-101 is an investigational therapy that modifies autologous CD34+ hematopoietic stem and progenitor cells to increase fetal hemoglobin production, potentially alleviating symptoms of SCD [3] - The ongoing BEACON Phase 1/2 study is evaluating the safety and efficacy of BEAM-101 in adult patients with severe SCD [3] Disease Context - Sickle cell disease affects approximately eight million people globally and is characterized by severe complications including anemia, pain crises, and organ failure [2][4] - In the U.S., SCD is the most common inherited blood disorder, impacting an estimated 100,000 individuals [4]

Core Insights - Billionaire Daniel Sundheim's hedge fund, D1 Capital Partners, is capitalizing on strong market momentum, with a reported 11.8% gain for the year as of April, despite broader market challenges due to US tariffs [3][6] - Analysts at Goldman Sachs predict the S&P 500 will surpass the 6,600 level by year-end, with an expected 7% earnings-per-share growth for the index this year and next [2] - Sundheim emphasizes the opportunity to invest in high-quality businesses on non-US exchanges as the equity market rises amid the US Federal Reserve's easing cycle [7] Company Performance - D1 Capital Partners experienced a significant recovery after a 30.5% decline in 2022, achieving a 44% return in 2024, driven by strategic investments, particularly in European markets [6] - Lexeo Therapeutics (NASDAQ:LXEO) is highlighted as a stock with a 95.14% upside potential, with a recent capital raise of $135 million to strengthen its financial position ahead of clinical trials [11][12][14] - Affirm Holdings, Inc. (NASDAQ:AFRM) shows a 30% upside potential, with recent partnerships expanding its funding and payment solutions, reflecting a growing trend in consumer finance [15][16][18]

Summary of Avidity Biosciences FY Conference Call Industry Overview - The focus of the conference was on muscular dystrophies, particularly Duchenne Muscular Dystrophy (DMD) and Facioscapulohumeral Muscular Dystrophy (FSHD) [1][24] - DMD is a multibillion-dollar market projected to grow to $3 billion by 2033 [1] - Other muscular dystrophies like DM1 and FSHD currently lack approved therapies, but promising treatments are in development [1] Key Points on DMD Treatments - Current DMD treatments include first-generation PMO exon-skipping therapies, new gene therapies, and steroids, but significant unmet needs remain, especially for non-ambulatory patients [2][3] - The FDA's approval of the first PMO exon-skipping therapy is seen as a watershed moment that opened investment in the DMD space [3][4] - The approval set a low regulatory bar, leading to increased investment and development of new therapies [3][5] - New technologies, such as Antibody Oligonucleotide Conjugates (AOCs), are being developed to improve dystrophin production and functional benefits [9][10] Clinical Data and Efficacy - AOCs have shown promising results, with 40% exon skipping and a 25% increase in dystrophin levels [10][11] - Creatine kinase (CK) levels, a measure of muscle damage, have returned to normal for many patients, indicating functional improvement [11][14] - The FDA's low bar for approval has allowed for a variety of therapies to enter the market, but safety and efficacy remain critical [16][19] Gene Therapy Insights - Sarepta Therapeutics' Elevidys gene therapy faced scrutiny but initially showed strong uptake due to the unmet need in DMD [16] - Avidity's approach focuses on optimizing the gene therapy construct and manufacturing processes to improve safety and efficacy [18][19] - The company has developed a microdystrophin that closely resembles natural dystrophin, aiming for higher expression and longer half-life [17][18] FSHD Developments - FSHD is characterized by the aberrant expression of the DUX4 transcription factor, which is toxic to muscle cells [26][27] - Avidity is using AOC technology to deliver silencing RNA to inhibit DUX4 expression, showing early signs of clinical benefit [27][30] - The identification of a circulating biomarker (C-DUX) for DUX4 may facilitate accelerated approval processes [29][32] Market Dynamics and Future Outlook - The DMD market is expected to see multiple effective therapies, with opportunities for various players due to the large unmet need [41][42] - The potential for combination therapies is highlighted, with patients and physicians open to using multiple modalities for better outcomes [47][48] - The FDA's accelerated approval pathway is anticipated to remain in place to encourage ongoing innovation in the muscular dystrophy space [49] Conclusion - The conference highlighted the significant advancements in genetic medicine for muscular dystrophies, the importance of regulatory pathways, and the potential for new therapies to address unmet needs in DMD and FSHD [1][41][49]