Aro Biotherapeutics’ Post

The EMA recently approved Casgevy, the first CRISPR- Cas9 medicine for beta-thalassemia and sickle cell disease.    ⬇ Read more in our executive summary of the approval ⬇   We cover:  1️⃣  An overview of the Casgevy approval by the European Medicines Agency. 2️⃣ A summary of the pivotal Casgevy trials. 3️⃣ The specific regulatory support Casgevy received from the EMA. 4️⃣ The wider regulatory considerations for gene-editing medicines. #regulatoryaffairs #drugdevelopment #orphandrugs #celltherapy #ATMP https://lnkd.in/gmrvp-2u

Massive news in the #genomics theme! Last Friday, the FDA approved the ground-breaking gene-editing therapy, Casgevy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals. This therapy marks a significant advancement as the first gene-editing treatment in the US for severe Sickle Cell Disease (SCD), impacting around 100,000 people in the US and 20 million worldwide. This approval sets the stage for potential advancements in addressing other rare blood diseases, with a PDUFA meeting on March 30, 2024, discussing Casgevy's approval for beta-thalassemia (TDT). A promising step forward in the pursuit of curing genetic disorders. Considering the recent challenges faced by genomics stocks over the past few years, is it now opportune for investors to contemplate allocation? https://lnkd.in/eZCEaXJ3 ARK Investment Management LLC Rize ETF #HealthcareInnovation #GeneEditing #FDAApproval

We’re cheering on the recently announced participants in the FDA's START Pilot Program! Aiming to accelerate novel drug and biologic development in the rare disease space, the FDA reviewed applications earlier this year and accepted the following companies into the Program: Larimar Therapeutics Inc. (Friedreich’s Ataxia) Grace Science, LLC (NGLY1 Deficiency) Myrtelle (Canavan Disease) Calico Life Sciences (Vanishing White Matter Disease) Denali Therapeutics (Sanfilippo Syndrome Type A) Neurogene Inc. (Rett Syndrome) The START Pilot Program was designed to enhance communication with FDA staff around clinical study design, control groups, patient population, nonclinical information, and product characterization. This is one of several programs announced by the FDA in recent months including: Rare Disease Endpoint Advancement (RDEA), CMC Development and Readiness, and the Platform Technology Designations Program. At DKP we are hopeful that these initiatives will improve support for development and eventual approval of groundbreaking treatments, providing much-needed hope to patients. #FDA #raredisease #rarediseases #genetherapy #neurodegenerative #pediatricraredisease #rettsyndrome #sanfilipposyndrome #vanishingwhitematterdisease #canavandisease #ngly1deficiency #friedreichsataxia

Today is Rare Disease Day - the official international awareness raising campaign for the 300 million people currently living with one of the 6,000+ rare diseases worldwide. The primary goal of the campaign is to increase awareness amongst the general public and decision-makers about rare diseases and their influence on the lives of families living with these conditions. In the article below, BAP Pharma's Group Director of Medicines Access and UK General Manager - Rebecca Bibby - highlights our Medicines Access team's commitment to bridge the gap between patients and companies developing advanced medical treatments, providing access to combat rare diseases across the globe. https://lnkd.in/ej5q4BwU For more information on how BAP Pharma can support your medicines access needs, please contact us at bd@bappharma.com or visit our website at www.bappharma.com #rarediseaseday #raredisease #medicinesaccess #marketaccess #clinicaltrials #cellandgenetherapy #celltherapy #generaltherapy #biotech #medicine #pharmaceuticals #oncology #patientexperience #pharmacy #pharmacists #orphandrugs #biotechnology #nhs

Napo Pharmaceuticals’ parent Jaguar Health is on a mission to develop novel proprietary prescription drugs sustainably derived from rainforest plants for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, and bowel incontinence. Many rainforests plants have untapped medicinal properties that may have the potential to address significant unmet medical needs. Our FDA-approved drug harnesses the antidiarrheal properties of the Croton lechleri tree. Learn more about our #pipeline of #plantbased drugs derived from biodiverse rainforests at www.jaguar.health #biopharma #pharma #ethnobotany #sustainability #naturalmedicine #HIV #cancer #rarediseases #pipeline #plantbased #biopharma #pharma #ethnobotany #sustainability #naturalmedicine #cancer #rarediseases

🚀 Exciting Developments in the Pharma Industry! 🌟 🔬 The FDA has given the green light to several innovative treatments, including: - Ohtuvayre (ensifentrine) for COPD maintenance. - Tepylute (thiotepa) targeting breast and ovarian cancers. - Capvaxive (pneumococcal 21-valent conjugate vaccine) for pneumococcal disease prevention. - Rytelo (imetelstat) for myelodysplastic syndrome. - Iqirvo (elafibranor for primary biliary cholangitis. 💊 "Sun Pharma" has achieved FDA approval for its new alopecia areata drug, Leqselvi, promising new hope for those affected by this condition. 🌍 Pfizer's gene therapy for hemophilia B has received approval from the European Commission, marking a significant milestone in treatment options available in the EU. 🏥 Despite strong sales reports from major companies like AstraZeneca, Roche, and Sanofi, the EU has controversially rejected Eisai and Biogen's Alzheimer's drug, Leqembi, after approvals in the US and other regions. 🤖 Strategic industry moves: - Caribou Biosciences and Roche have announced strategic shifts, including workforce adjustments. - Vertex Pharmaceuticals has initiated legal action over limits on fertility services for its Casgevy treatment patients. Stay tuned for more updates as the pharma industry continues to innovate and evolve! #Pharma #FDA #Biotech #HealthcareInnovation #MedicalResearch #DrugDevelopment #niper #pharmacy #mpharma #bpharma #Gpat #Niperhyderabad #nipermohali

Jaguar Health is on a mission to develop novel proprietary prescription drugs sustainably derived from rainforest plants for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, and bowel incontinence. Many rainforests plants have untapped medicinal properties that may have the potential to address significant unmet medical needs. Our FDA-approved drug harnesses the antidiarrheal properties of the Croton lechleri tree. Learn more about our #pipeline of #plantbased drugs derived from biodiverse rainforests at www.jaguar.health #biopharma #pharma #ethnobotany #sustainability #naturalmedicine #HIV #cancer #rarediseases #pipeline #plantbased #biopharma #pharma #ethnobotany #sustainability #naturalmedicine #cancer #rarediseases

𝐒𝐢𝐥𝐞𝐧𝐜𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐏𝐡𝐚𝐬𝐞 𝟐 𝐃𝐚𝐭𝐚 𝐟𝐨𝐫 𝐙𝐞𝐫𝐥𝐚𝐬𝐢𝐫𝐚𝐧 𝐢𝐧 𝐂𝐚𝐫𝐝𝐢𝐨𝐯𝐚𝐬𝐜𝐮𝐥𝐚𝐫 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐒𝐭𝐮𝐝𝐲 Silence Therapeutics plc, Nasdaq: SLN (“Silence”) Silence Therapeutics plc, a pioneering biotechnology company dedicated to combatting diseases with precision engineered medicines, announces positive 36-week data from the ongoing ALPACAR-360 phase 2 study of zerlasiran (SLN360) in 178 subjects with baseline lipoprotein(a), or Lp(a), levels at or over 125 nmol/L, at high risk of atherosclerotic cardiovascular disease (ASCVD) events. Zerlasiran, a siRNA, aims to lower Lp(a), a significant genetic risk factor for cardiovascular disease affecting up to 20% of the global population. In the double-blind placebo-controlled period, zerlasiran was administered at 300 mg subcutaneously every 16 or 24 weeks and 450 mg every 24 weeks to patients with a median baseline Lp(a) of approximately 215 nmol/L. Significant reduction from baseline in Lp(a) compared to placebo was observed at 36 weeks (primary endpoint). Median percentage reduction in Lp(a) of 90% or greater were noted for both doses at week 36. No new safety concerns emerged during this period. The 60-week study continues, with secondary endpoints including change in Lp(a) from baseline to 48 weeks, 60 weeks, and potential effects on other lipids/lipoproteins to be evaluated. “We are encouraged by the phase 2 data, consistent with phase 1 results, supporting zerlasiran as a competitive treatment for patients with high Lp(a),” said Steven Romano, MD Steve Romano, Head of Research and Development at Silence. “We anticipate reporting topline 48-week data in the second quarter of this year.” #Zerlasiran #CardiovascularDisease #Phase2Study #Biotechnology #PrecisionMedicine #HealthcareInnovation #Lipoprotein #HeartHealth #Research #MedicalAdvancements

We're thrilled to announce the clearance of our investigational new drug application (IND) by the U.S. FDA for VERVE-101 in patients living with heterozygous familial hypercholesterolemia (HeFH). VERVE-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood low-density lipoprotein cholesterol (LDL-C). This clearance, for the first time, enables clinical development of an in vivo base editing product candidate in the U.S. and opens a regulatory path for development of #geneediting medicines for atherosclerotic #cardiovascular disease. Read more here: https://lnkd.in/gk4UyRxp