Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the best biotech stocks with high potential. H.C. Wainwright analyst Patrick Trucchio reiterated a Buy rating on Beam Therapeutics Inc. (NASDAQ:BEAM) on September 15, retaining the price target of $80. Is Beam Therapeutics Inc. (BEAM) the Best Gene-Editing Stock to Buy? The analyst supported the optimistic rating with the company’s strong momentum in its in vivo and ex vivo base editing programs. Similarly, in a report released on September 10, Kostas Bilio ...

CHARLESTOWN, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from its neuromuscular and cardiac programs at the World Muscle Society (WMS) 2025 Annual International Congress, October 7-11, 2025, in Vienna, Austria, and at the European Society of Gene & Cell Therapy (ESGCT) 2025 Annual Congress, October 7-12, 2025, in Seville, Spain. “Our presentations at WM ...

BOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, "enGene†or the "Company†), a clinical-stage, non-viral genetic medicine company, today announced that Hussein Sweiti, M.D., MSc, was appointed Chief Medical Officer, effective September 29, 2025. Dr. Sweiti is a surgical oncologist and physician-scientist with more than 15 years of experience spanning clinical practice, oncology clinical research, global drug development, regulatory submissions, and medical affairs. He mos. ...

Revised label allows treatment of DEB patients from birth VYJUVEK can now be applied by patients and caregivers PITTSBURGH, Sept. 15, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the United States Food and Drug Administration (FDA) approved a label update for VYJUVEK® (beremagene geperpavec-svdt) that expands the VYJUVEK eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and provides patients full flexibili ...

Core Insights - Beam Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-101, a genetically modified cell therapy aimed at treating sickle cell disease (SCD) [1][2] - The RMAT designation follows an earlier orphan drug designation and is expected to facilitate collaboration with the FDA as the company progresses towards a Biologics License Application (BLA) filing [2][4] - The BEACON Phase 1/2 trial has dosed 30 patients, with promising clinical data indicating robust increases in fetal hemoglobin and reductions in sickle hemoglobin [3][5] Company Overview - Beam Therapeutics is focused on developing precision genetic medicines using its proprietary base editing technology, which allows for precise genetic modifications without double-stranded breaks in DNA [7][8] - The company aims to create a fully integrated platform for gene editing, delivery, and manufacturing to provide long-term cures for serious diseases [7][8] Product Details - BEAM-101 is an investigational therapy that utilizes autologous CD34+ hematopoietic stem and progenitor cells, which are base-edited to enhance fetal hemoglobin production [5] - The therapy is designed to mimic naturally occurring variants that lead to increased fetal hemoglobin, potentially alleviating symptoms of SCD [5][6] Clinical Trial Insights - Updated clinical data from the BEACON trial presented at the EHA 2025 Congress showed durable increases in fetal hemoglobin and no vaso-occlusive crises reported post-engraftment [3][4] - The trial is ongoing, with additional data expected by the end of 2025, further supporting the potential of BEAM-101 as a transformative treatment for SCD [2][4]

Financial Data and Key Metrics Changes - Q2 net revenue was $96 million, marking a return to growth and bringing total net Visovac revenue since launch to over $525 million [6][27] - Net income for the quarter was $38.3 million, representing $1.33 per basic and $1.29 per diluted share, compared to $15.6 million in the prior year's second quarter [29] - Gross margin remained consistent at 93% in Q2 2025, with cost of goods sold at $7.2 million compared to $6 million in the prior year [28] Business Line Data and Key Metrics Changes - The return to growth in Q2 was attributed to patients resuming treatment and the ongoing expansion of the sales team [6] - Reimbursement approvals increased, with over 575 approvals secured by the end of Q2 [6] - Compliance while on drug was reported at 82%, although a decline is expected as the patient mix shifts [7] Market Data and Key Metrics Changes - The company is preparing for launches in Europe and Japan, with the European launch expected to begin this quarter [12][14] - Japan's Ministry of Health approved VYZUVAC, allowing for a broad label that includes all DEP patients from birth [11] - The identified patient pool in Germany and France exceeds 500 patients each, supported by expert centers [13] Company Strategy and Development Direction - The company aims for steady, multiyear growth in Europe and Japan, leveraging broad labels and flexible dosing options [16] - The focus remains on expanding the sales force and enhancing patient support to drive long-term sustainable growth [10] - The company is pursuing opportunities in other global markets through distributors and partners [17] Management's Comments on Operating Environment and Future Outlook - Management expects Q3 revenues to be below Q2 due to seasonal trends but anticipates a return to growth in Q4 [9] - The company is confident in the long-term growth trajectory, driven by a rich pipeline of clinical-stage programs [31] - Management highlighted the unpredictability of patient restarts and the need for ongoing monitoring of treatment patterns [8] Other Important Information - The company reported a strong balance sheet with over $820 million in cash and investments, positioning it well for upcoming launches and R&D objectives [30] - The R&D team is progressing with multiple clinical trials, including those in oncology and aesthetics [18][23] Q&A Session Summary Question: Was the revenue growth in Q2 impacted by the Salesforce expansion? - Management clarified that part of the increase was due to patients resuming treatment, and the full impact of new hires will be felt in the coming quarters [36] Question: Can you provide quantitative commentary on the slower start to Q3? - Management noted that it is early in the quarter and summer vacations typically lead to more treatment pauses, making it difficult to quantify [40] Question: How do drug holidays factor into the guidance for U.S. patients initiating VYJUVEC? - Management indicated they are on track but may be a quarter or two behind the original goal of reaching 720 patients [46] Question: What is the compliance rate among patients using different vial counts? - Management explained that compliance is defined based on the duration of drug use, with a range of 76-84% being consistent across calculations [102] Question: What are the timelines for capturing the remaining patient market in the U.S.? - Management emphasized that the goal is to target the entire 1,200 patient base, with a focus on achieving 60% market share within two years [92]

$96.0 million in 2Q VYJUVEK revenue and $525.4 million since launch in 3Q 2023 VYJUVEK approved in Japan for the treatment of DEB patients from birth Strong balance sheet, ending the quarter with $820.8 million in cash and investments PITTSBURGH, Aug. 04, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) today reported financial results for the second quarter ending June 30, 2025 and provided a business update. "With the approval of VYJUVEK in Europe and Japan, we are on the cusp ...

PITTSBURGH, July 29, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that it will report its second quarter 2025 financial results on Monday, August 4, 2025, prior to the open of U.S. markets. CONTACT Investors and Media: Stéphane Paquette, PhD Krystal Biotech spaquette@krystalbio.com The Company's management will also host a conference call and webcast at 8:30 am ET on Monday, August 4, 2025, to discuss the financial r ...

Core Insights - VYJUVEK has received marketing authorization in Japan for the treatment of dystrophic epidermolysis bullosa (DEB) starting from birth, marking it as the first genetic medicine approved for this condition in the country [1][5] - The approval allows for flexible administration options, including home dosing and administration by patients or their family members, which is a significant advancement in treatment accessibility [1][3] - The company anticipates launching VYJUVEK in Japan by the end of 2025, pending the completion of reimbursement procedures [4][7] Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs, with VYJUVEK being its first commercial product [8][9] - The company has a robust pipeline of investigational genetic medicines across various therapeutic areas, including respiratory, oncology, dermatology, ophthalmology, and aesthetics [8][9] Regulatory and Clinical Insights - The approval by Japan's Ministry of Health, Labour and Welfare (MHLW) was based on a comprehensive clinical dataset, including results from an open-label extension study in Japanese patients, which showed that all four patients achieved full wound closure at six months [5][6] - VYJUVEK was previously approved by the FDA in the United States in May 2023 and by the European Commission in April 2025, indicating a growing recognition of its therapeutic potential [7]

Clinical Pipeline and Regulatory Milestones - The company has 3 late-stage clinical programs in pivotal/Phase 3 trials for prevalent non-inherited indications[4,7,30,36,42,90] - Potential global filings are anticipated in 2025, 2026, and 2027[7,30,36,42,90] - The company has a deep pre-IND pipeline targeting conditions like ALS, MC4R obesity, and metabolic disease[7,8,32,38,45,93] Manufacturing and Technology - The company operates 2 GMP facilities at commercial scale[5,9,33,39,46,94] - Proprietary vectorization technology increases potency by 2-10x from the same promoter[5,9,34,40,47,95] - AI-driven improvements are based on over 20 vectors and more than 50 GMP runs[5,9,33,39,46,94] Partnerships and Financials - MeiraGTx will receive up to $415 million from Janssen through an asset purchase agreement[11] - Sanofi made a $30 million strategic investment through the sale of 4 million ordinary shares at $7.50 per share[11] AAV-AQP1 for Radiation Induced Xerostomia - There are 170,000 Grade 2/3 RIX patients in the US[13] - There are 15,000 new cases of grade 2/3 RIX annually in the US[13] AAV-GAD for Parkinson's Disease - There are 10 million Parkinson's patients worldwide[18,28] - The estimated economic burden of Parkinson's Disease in the US is $52 billion[19]