Latest news with #SCD
Yahoo
a day ago
- Business
- Yahoo
The 2 Smartest Beaten-Down Biotech Stocks to Buy on the Dip
CRISPR Therapeutics has the potential to generate significant breakthroughs in gene editing. Regeneron's biggest growth driver is still performing well, and it is developing newer medicines. 10 stocks we like better than CRISPR Therapeutics › Even though broader equities have been highly volatile this year, it's still a good idea to invest in stocks for a straightforward reason. Holding shares of top companies for five years and beyond will usually allow anyone to earn superior returns. That's not a secret, but it's easy to forget when dealing with an uncertain near term. Even turbulent times don't change this fact. For investors still seeking companiies with attractive long-term prospects, let's consider two in the biotech industry: CRISPR Therapeutics (NASDAQ: CRSP) and Regeneron Pharmaceuticals (NASDAQ: REGN). These drugmakers have faced issues recently, and their shares have declined significantly over the trailing-12-month period. However, there may be considerable upside here for patient investors. Skepticism about CRISPR Therapeutics' prospects may be warranted. Though it has a product on the market, a gene editing therapy called Casgevy it developed with Vertex Pharmaceuticals, it is not yet generating much revenue despite first being approved in late 2023. Administering Casgevy to patients with sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT) -- two rare blood-related disorders -- is a complex and lengthy process. That's one of the issues with the company's gene-editing medicines. CRISPR also remains unprofitable, a significant concern for many investors given the challenging and uncertain economy we face. Even so, the company might be worth serious consideration. Though it is taking time to ramp up sales, Casgevy's potential is vast even without potential label expansions. There is also very little competition to speak of for the medicine. There were no one-time curative treatments for SCD and TDT before the advent of gene-editing therapies. Casgevy should eventually generate well over $1 billion in annual sales. Furthermore, despite its disadvantages, the field of gene-editing has the potential to lead to major breakthroughs. Casgevy was an example. CRISPR's pipeline features other potential gems. CTX-112 is being tested in B-cell malignancies, CTX-131 in solid tumors, and CTX-211 in type 1 diabetes. Also, CTX-310 and CTX-320 target high levels of cholesterol. Of these, CTX-112 earned the Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration, which means it has demonstrated promising signs of efficacy for treatment of a serious condition for which there is an unmet clinical need. CRISPR Therapeutics is somewhat on the riskier side, but the biotech company could soar in the next five years as it continues to make substantial clinical and regulatory progress. Shares have declined by 32% over the past year. Now may be a good time to initiate a small position on the dip. Regeneron is facing biosimilar competition for Eylea, one of its biggest growth drivers. In the first quarter, the company's revenue decreased by 4% year over year to $3 billion. But there is some good news. Regeneron has long been planning for the Eylea patent cliff, and if not for the approval of a new high-dose (HD) formulation of the medicine in late 2023, it would be in a much worse position. U.S. sales of Eylea HD increased by 54% year over year to $307 million in the period. This version of Eylea should continue to steal some patients away from the old formulation due to the former's better dosing schedule. Furthermore, Eylea HD could potentially earn label expansions, helping to mitigate Eylea-related losses. Meanwhile, Regeneron's other main growth driver, eczema treatment Dupixent, is still performing well. Regeneron co-markets Dupixent with Sanofi. The medicine's total sales in the first quarter grew 19% year over year to $3.67 billion. Dupixent hasn't peaked yet. Recent label expansions, including in treating chronic obstructive pulmonary disease (COPD), which it earned in the U.S. in October, should allow it to continue growing its revenue for a while. Elsewhere, Regeneron is developing newer medicines. It has been making strides in its oncology business lately. Within the next year, it could earn several brand-new approvals or label expansions in this field. These will also help it overcome the Eylea-related troubles. Lastly, Regeneron has a robust share-buyback program and offers a dividend it initiated this year. The sell-off it experienced over the last 12 months might have been justified. The company tried to fend off Eylea biosimilars in court, but that didn't work, leading to a significant drop in its share price, which was justifiable. But at current levels -- down by 38% over the trailing-12-month period -- Regeneron's shares look attractive. Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and CRISPR Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $651,049!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $828,224!* Now, it's worth noting Stock Advisor's total average return is 979% — a market-crushing outperformance compared to 171% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 19, 2025 Prosper Junior Bakiny has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics, Regeneron Pharmaceuticals, and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. The 2 Smartest Beaten-Down Biotech Stocks to Buy on the Dip was originally published by The Motley Fool
Yahoo
5 days ago
- Business
- Yahoo
Fulcrum Therapeutics to Present at Upcoming Medical Meetings
CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (the 'Company') (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced upcoming presentations at the 5th Global Congress on Sickle Cell Disease and the 2025 European Hematology Association Congress. The Company's presentations are listed below. Following congress publication, copies of the presentations will be available in the "Publications & Presentations" section of the Company's website at 5th Global Congress on Sickle Cell Disease, Abuja, Nigeria, June 3-6, 2025: Poster Presentation: Preclinical and Early Clinical Results of Pociredir, a Novel, Oral, Potent Fetal Hemoglobin Inducer for the Treatment of Sickle Cell Disease (SCD) (Presentation ID: 103, Tuesday, June 4) Poster Presentation: A Phase 1b, Open-Label, Multiple-Dose Study Evaluating Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Pociredir in Patients with Sickle Cell Disease (SCD): Trial Design (Presentation ID: 101, Tuesday, June 4) 2025 European Hematology Association Congress, Milan, Italy, June 12-15, 2025 Poster Presentation: Pharmacokinetics (PK), Pharmacodynamics (PD), and Safety of the Novel Oral Fetal Hemoglobin (HBF) Inducer Pociredir in Healthy Adults in a Phase 1 study (PF1170 – Friday, June 13) Poster Presentation: Pociredir, a Potent and Selective EED Inhibitor for the Treatment of Sickle Cell Disease, Induces Target Engagement and Gene Expression Changes that are Specific and Reversible in Wild-Type Mice (PS2175 – Saturday, June 14) About Fulcrum TherapeuticsFulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's lead clinical program is pociredir, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease (SCD). Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. For more information, visit and follow us on Twitter/X (@FulcrumTx) and LinkedIn. About PociredirPociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED) that was discovered using Fulcrum's proprietary discovery technology. Inhibition of EED leads to potent downregulation of key fetal globin repressors, including BCL11A, thereby causing an increase in fetal hemoglobin (HbF). Pociredir is being developed for the treatment of SCD. Initial data in SCD demonstrated proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. In clinical trials conducted prior to the clinical hold, which was lifted by the FDA in August 2023, pociredir was generally well-tolerated in people with SCD with up to three months of exposure, with no serious treatment-related adverse events reported. Pociredir has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD. To learn more about these trials please visit About Sickle Cell DiseaseSickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. People with SCD typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease, and reduced life expectancy. Contact:Kevin GardnerLifeSci Advisors, LLCkgardner@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
NDTV
6 days ago
- Health
- NDTV
All You Need To Know About Sickle Cell Disease
Sickle Cell Disease (SCD) is a genetic blood disorder that affects millions globally, especially in India's tribal and rural populations. According to the World Health Organisation (WHO), it is one of the most prevalent inherited diseases worldwide, caused by a mutation in the haemoglobin gene. This mutation results in the production of abnormally shaped red blood cells that resemble a sickle, hence the name. These misshapen cells can block blood flow, leading to severe pain, organ damage, and even stroke. Early diagnosis and proper management are key to improving quality of life for those living with this lifelong condition. What causes sickle cell disease? SCD is an inherited condition passed on when both parents carry the sickle cell gene. Instead of round, flexible red blood cells, affected individuals produce crescent-shaped cells that are rigid and sticky. These cells die early and clog blood vessels, leading to chronic anaemia and painful episodes known as sickle cell crises. Common symptoms and signs of sickle cell disease Symptoms usually appear around 5-6 months of age and can vary in severity throughout life. 1. Anaemia Chronic fatigue, weakness, and pale skin due to reduced red blood cells 2. Pain crises Sudden, severe pain in the chest, back, joints, or limbs 3. Swelling in hands and feet Often the first sign in infants 4. Frequent infections Damaged spleen makes patients more prone to bacterial infections 5. Delayed growth or puberty Due to reduced oxygen and nutrients 6. Vision problems Blocked blood vessels can damage the retina Diagnosis of sickle cell disease SCD can be diagnosed through a simple blood test. Early diagnosis is vital to begin preventive care and reduce complications. 1. Newborn screening Mandatory in many Indian states, helps detect SCD early 2. Haemoglobin electrophoresis Confirms the presence of abnormal haemoglobin 3. Prenatal testing Available for expecting parents with a family history Health complications associated with SCD Sickle cell disease can lead to life-threatening complications if not managed well. 1. Stroke Caused by blocked blood vessels in the brain 2. Acute chest syndrome Similar to pneumonia, caused by trapped sickle cells 3. Organ damage Long-term oxygen deprivation affects the liver, kidney, and heart 4. Leg ulcers and bone damage 5. Psychological stress Chronic illness often affects mental well-being Treatment and management There is no universal cure for SCD yet, but treatment options focus on reducing symptoms and preventing complications. According to the Centres for Disease Control and Prevention (CDC), comprehensive care including regular health check-ups can significantly improve the life expectancy of patients. 1. Hydroxyurea A medication that reduces frequency of pain episodes and the need for blood transfusions 2. Blood transfusions Help manage severe anaemia and prevent stroke 3. Pain management With anti-inflammatory drugs and sometimes opioids 4. Bone marrow transplant The only potential cure, mostly in children with severe symptoms 5. Vaccinations and antibiotics To prevent infections Living with sickle cell disease Lifestyle modifications play a crucial role in managing SCD. a. Stay hydrated to prevent sickling of red blood cells b. Avoid extreme temperatures, high altitudes, and strenuous activities c. Regular exercise, gentle physical activity can improve circulation d. Balanced diet, rich in iron, folate, and vitamin D e. Mental health support, counselling and support groups are beneficial Government initiatives in India India launched the National Sickle Cell Anaemia Elimination Mission (2023) to eliminate the disease by 2047. The focus is on widespread screening in tribal regions, genetic counselling, prenatal testing, and free treatment and awareness programmes. This is a critical step toward reducing the disease burden in states like Chhattisgarh, Odisha, Gujarat, and Maharashtra. Sickle Cell Disease may be lifelong, but it doesn't have to define one's life. With early diagnosis, regular treatment, and supportive care, individuals with SCD can lead full and productive lives. Increased awareness, especially in high-risk communities, and government-led efforts can together help combat this inherited condition and ensure a healthier future for generations to come. Disclaimer: This content including advice provides generic information only. It is in no way a substitute for a qualified medical opinion. Always consult a specialist or your own doctor for more information. NDTV does not claim responsibility for this information.
The Hindu
27-05-2025
- Health
- The Hindu
Study on stigma faced by people with sickle cell disease
A study co-authored by Deepa Bhat, professor in Anatomy and certified genetic counsellor, JSS Medical College, Mysuru has been published in The Lancet Regional Health – Southeast Asia, marking a national breakthrough in stigma research related to sickle cell disease, said the JSS Academy of Higher Education and Research (JSS AHER), Mysuru. 'It (the study) is a milestone in public health and genetic research,' the JSS AHER said in a release here. JSS Medical College is the constituent college of JSS AHER. The release said the study introduces the ICMR-SCD Stigma Scale for India (ISSSI) – 'the first scientifically validated tool from India to assess the multi-dimensional stigma faced by the people living with SCD and their caregivers.' Developed under the leadership of Dr. Bonta V. Babu and funded by the ICMR National Task Force Project under the Socio-Behavioural Research Division, the ISSSI is a culturally attuned and linguistically adaptable scale, tailored to the Indian context. The multi-centric study involved fieldwork across six tribal regions in collaboration with the Central Tribal University, Vizianagaram, Bodoland University, Assam, Parul University, Vadodara, ICMR-RMRC, Bhubaneshwar and JSS Medical College of JSS AHER, Mysuru. 'The scale captures stigma across key domains – familial, illness burden, interpersonal relationships, healthcare interactions and social disclosure – and is now positioned as a vital tool for the National Sickle Cell Anaemia Elimination Mission,' the release stated. The ISSSI tool is now available for use in clinical settings, community outreach and health systems research, enabling policymakers, clinicians and researchers to address stigma with precision and empathy, according to the university.
The Hindu
24-05-2025
- Health
- The Hindu
Study validates India's first ICMR-SCD Stigma Scale for sickle cell disease
India accounts for the second-highest prevalence of sickle cell disease (SCD) globally, following sub-Saharan Africa. However, not much research has been done, and no stigma measurement tool was available to India before the Indian Council of Medical Research (ICMR) recently developed India's first ICMR-SCD Stigma Scale for India (ISSSI). The three scales available globally were unsuitable due to India's diversity. India's stigma scale was developed following global standards and a recently published study has validated the ISSSI patient scale (ISSSI-Pt) and ISSSI caregiver scale (ISSSI-Cg) according to an article published in the Lancet (regional health South-East Asia) this month. ISSSI is the first in India and the fourth globally, available in clinical and research settings. People with SCD often suffer from severe pain episodes, chronic fatigue, swelling, infections, and potential organ damage, significantly impacting their quality of life. 'This study explicitly developed and validated the ISSSI. The finalized scales capture multidimensional aspects of stigma, including familial and reproductive, social disclosure, illness burden, interpersonal, and healthcare interaction challenges. The findings underscore the scales' psychometric robustness and utility in clinical and research settings,' said the article. It added that the methodological rigour employed in the scale development makes it a robust tool for understanding SCD-related stigma among Indian SCD patients and their caregivers in clinical and research contexts. 'The ISSSI will be available to the mission and researchers immediately,'' said the article. Meanwhile, the study was conducted across six SCD-endemic districts in India -- Alluri Seetharama Raju in Andhra Pradesh, Anuppur in Madhya Pradesh, Chhoteudepur in Gujarat, Kandhamal in Odisha, Mysuru in Karnataka, and Udalguri in Assam state. These sites were selected to capture a wide range of socio-cultural and geographical contexts. Each district presents unique demographic and socioeconomic characteristics, including variations in access to healthcare and awareness of SCD. Sickle cell disease is caused by the inheritance of two mutated β-globin genes, one from each parent, leading to the production of abnormal haemoglobin, known as sickle haemoglobin. SCD is a significant public health problem in many regions, impacting populations in several countries. The new scales for India measures multidimensional aspects of stigma, including familial and reproductive, social disclosure, illness burden, interpersonal, and healthcare interaction challenges. The development of the ISSSI represents a significant advancement in understanding and addressing the multifaceted impact of SCD, including stigma. India has now announced the national sickle cell anaemia elimination mission to eliminate SCD as a public health problem, implementing various SCD management interventions and assessing their impact is inevitable. In India, SCD represents a pressing public health concern, particularly within tribal communities, in which the prevalence is considerably high when compared with the general population. Limited healthcare access, socioeconomic challenges, widespread misconceptions, and inadequate knowledge about the disease compound the burden of SCD in India.
