• Live Feeds
    • Press Releases
    • Insider Trading
    • FDA Approvals
    • Analyst Ratings
    • Insider Trading
    • SEC filings
    • Market insights
  • Analyst Ratings
  • Alerts
  • Subscriptions
  • Settings
  • RSS Feeds
Quantisnow Logo
  • Live Feeds
    • Press Releases
    • Insider Trading
    • FDA Approvals
    • Analyst Ratings
    • Insider Trading
    • SEC filings
    • Market insights
  • Analyst Ratings
  • Alerts
  • Subscriptions
  • Settings
  • RSS Feeds
Dashboard
    Quantisnow Logo

    © 2025 quantisnow.com
    Democratizing insights since 2022

    Services
    Live news feedsRSS FeedsAlerts
    Company
    AboutQuantisnow PlusContactJobs
    Legal
    Terms of usePrivacy policyCookie policy

    December 8, 2023 - FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

    12/8/23 11:12:21 AM ET
    $BLUE
    $VRTX
    Biotechnology: Biological Products (No Diagnostic Substances)
    Health Care
    Biotechnology: Pharmaceutical Preparations
    Health Care
    Get the next $BLUE alert in real time by email
    For Immediate Release:
    December 08, 2023

    Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy. 

    Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African Americans and, while less prevalent, also affects Hispanic Americans. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death. 

    “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.” 

    Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology. Patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology. 

    CRISPR/Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut. The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.

    Lyfgenia is a cell-based gene therapy. Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events. With Lyfgenia, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease. Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient. 

    Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, a patients’ own stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in Casgevy and Lyfgenia. Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness. 

    “These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health.”

    Data Supporting Casgevy

    The safety and effectiveness of Casgevy were evaluated in an ongoing single-arm, multi-center trial in adult and adolescent patients with SCD. Patients had a history of at least two protocol-defined severe VOCs during each of the two years prior to screening. The primary efficacy outcome was freedom from severe VOC episodes for at least 12 consecutive months during the 24-month follow-up period. A total of 44 patients were treated with Casgevy. Of the 31 patients with sufficient follow-up time to be evaluable, 29 (93.5%) achieved this outcome. All treated patients achieved successful engraftment with no patients experiencing graft failure or graft rejection. 

    The most common side effects were low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache and itching. 

    Data Supporting Lyfgenia

    The safety and effectiveness of Lyfgenia is based on the analysis of data from a single-arm, 24-month multicenter study in patients with sickle cell disease and history of VOEs between the ages of 12- and 50- years old. Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between 6 and 18 months after infusion with Lyfgenia. Twenty-eight (88%) of 32 patients achieved VOE-CR during this time period. 

    The most common side effects included stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with chemotherapy and underlying disease. 

    Hematologic malignancy (blood cancer) has occurred in patients treated with Lyfgenia. A black box warning is included in the label for Lyfgenia with information regarding this risk. Patients receiving this product should have lifelong monitoring for these malignancies. 

    Both the Casgevy and Lyfgenia applications received Priority Review, Orphan Drug, Fast Track and Regenerative Medicine Advanced Therapy designations. 

    The FDA granted approval of Casgevy to Vertex Pharmaceuticals Inc. and approval of Lyfgenia to Bluebird Bio Inc.

    ###

    Boilerplate

    The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.


    Inquiries

    Media:
    Carly (Kempler) Pflaum
    240-672-8872
    Consumer:
    888-INFO-FDA

     
    Get the next $BLUE alert in real time by email

    Chat with this insight

    Save time and jump to the most important pieces.

    Recent Analyst Ratings for
    $BLUE
    $VRTX

    CompanyDatePrice TargetRatingAnalyst
    Vertex Pharmaceuticals Incorporated
    $VRTX
    5/7/2025Outperform → Peer Perform
    Wolfe Research
    Vertex Pharmaceuticals Incorporated
    $VRTX
    5/6/2025$503.00Outperform → Market Perform
    Leerink Partners
    Vertex Pharmaceuticals Incorporated
    $VRTX
    4/22/2025$535.00Overweight
    Cantor Fitzgerald
    bluebird bio Inc.
    $BLUE
    2/24/2025Underweight → Neutral
    Analyst
    bluebird bio Inc.
    $BLUE
    2/24/2025$29.00Overweight
    Analyst
    Vertex Pharmaceuticals Incorporated
    $VRTX
    2/12/2025$408.00 → $424.00Sell → Hold
    Canaccord Genuity
    Vertex Pharmaceuticals Incorporated
    $VRTX
    2/11/2025Sell → Hold
    Canaccord Genuity
    Vertex Pharmaceuticals Incorporated
    $VRTX
    1/30/2025$460.00Overweight → Equal Weight
    Wells Fargo
    More analyst ratings

    $BLUE
    $VRTX
    SEC Filings

    See more
    • SEC Form 10-Q filed by Vertex Pharmaceuticals Incorporated

      10-Q - VERTEX PHARMACEUTICALS INC / MA (0000875320) (Filer)

      5/6/25 4:04:39 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Amendment: SEC Form SC 14D9/A filed by bluebird bio Inc.

      SC 14D9/A - bluebird bio, Inc. (0001293971) (Subject)

      5/6/25 7:47:43 AM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Amendment: SEC Form SC TO-T/A filed by bluebird bio Inc.

      SC TO-T/A - bluebird bio, Inc. (0001293971) (Subject)

      5/6/25 7:33:39 AM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $BLUE
    $VRTX
    Press Releases

    Fastest customizable press release news feed in the world

    See more
    • Vertex Reports First Quarter 2025 Financial Results

      — Total revenue of $2.77 billion, a 3% increase compared to Q1 2024; raised the low end of total revenue guidance by $100 million to a new range of $11.85 to $12 billion — — Strong progress with CASGEVY®, ALYFTREK™ and JOURNAVX™ launches — — Povetacicept (pove) IgAN Phase 3 interim analysis (IA) cohort fully enrolled and zimislecel Phase 3 program to complete dosing this quarter, setting up potential filings in 2026; inaxaplin Phase 3 IA cohort on track to complete enrollment in the second half of 2025 — — Four programs already in pivotal development, with pivotal study of pove in primary membranous nephropathy (pMN) to start this year — Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)

      5/5/25 4:01:00 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Carlyle and SK Capital Receive All Required Regulatory Approvals to Complete the Acquisition of bluebird bio

      bluebird Board of Directors unanimously supports the transaction as the only viable option for stockholders to receive consideration for their shares and recommends that all stockholders immediately tender their shares into the offer Parties expect the merger to be completed promptly following the successful completion of the ongoing tender offer bluebird bio, Inc. (NASDAQ:BLUE) ("bluebird" or "the Company"), Carlyle (NASDAQ:CG) ("Carlyle"), SK Capital Partners, LP ("SK Capital") and Beacon Parent Holdings, L.P. ("Parent") announced today that all required regulatory approvals to complete the previously announced acquisition of the Company by Carlyle and SK Capital have been received. No

      5/5/25 4:00:00 PM ET
      $BLUE
      $CG
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
      Investment Managers
      Finance
    • bluebird bio confirms that Ayrmid, Ltd. has not delivered a binding offer or obtained necessary financing despite extensive engagement

      bluebird Board reaffirms unanimous recommendation in support of transaction with Carlyle and SK Capital and recommends all stockholders tender into the current agreement by May 2, 2025 bluebird bio, Inc. (NASDAQ:BLUE) ("bluebird" or "the Company") today announced that after three weeks of engagement, including a timeline extension, Ayrmid Ltd. ("Ayrmid") has not submitted a binding proposal to acquire bluebird and has not obtained necessary financing. In consultation with its financial and legal advisors, the bluebird Board of Directors (the "Board") reaffirms its recommendation in support of the transaction with Carlyle and SK Capital and recommends all stockholders tender into the curren

      4/16/25 8:00:00 AM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $BLUE
    $VRTX
    Leadership Updates

    Live Leadership Updates

    See more
    • 2Flo Ventures Announces Addition of Renowned Life Sciences Executive Charlotte Jones-Burton, MD, MS as a Partner

      CHICAGO, June 2, 2024 /PRNewswire/ -- 2Flo Ventures, an emerging venture capital firm and startup studio focused on the discovery, development, and commercialization of healthcare solutions that address disparities in outcomes and health equity broadly, is pleased to announce the addition of Dr. Charlotte Jones-Burton as a Partner and Head of Life Science Product Development and Strategy. 2Flo Ventures Announces Addition of Renowned Life Sciences Executive Charlotte Jones-Burton, MD, MS as a PartnerDr. Jones-Burton brings to 2Flo Ventures nearly two decades of experience in th

      6/2/24 10:15:00 PM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • bluebird bio Appoints O. James Sterling as Chief Financial Officer

      bluebird bio, Inc. (NASDAQ:BLUE) today announced that O. James Sterling, has been appointed chief financial officer (CFO), effective June 10, 2024. Mr. Sterling most recently served as chief financial officer of Renalytix plc, a diagnostics company focused on clinical management of kidney disease. "We are thrilled to welcome James to bluebird bio. We are confident that his extensive experience in the healthcare sector and knowledge of the capital markets will position us well as we work to prove the commercial gene therapy model, and demonstrate progress on our path to profitability," said Andrew Obenshain, chief executive officer, bluebird bio. "It is an honor to be joining bluebird bi

      5/29/24 7:00:00 AM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Engine Capital Sends Letter to 2seventy's Board of Directors Outlining Steps to Maximize Shareholder Value

      Believes 2seventy Could Be Conservatively Worth ~$9 Per Share if the Company Exclusively Focuses on Its Most-Valuable Asset Abecma, Reduces Corporate Overhead and Makes Improvements to the Composition of the Management Team and Board Engine Capital LP, which owns approximately 3% of 2seventy bio, Inc.'s (NASDAQ:TSVT) ("2seventy" or the "Company") outstanding shares, today announced that it sent the below letter the Company's Board of Directors (the "Board"). *** December 6, 2023 2seventy bio, Inc. 60 Binney Street Cambridge, MA 02142 Attention: Board of Directors Dear Members of the Board: Engine Capital LP (together with its affiliates, "Engine" or "we"), is a meaningful shareh

      12/6/23 8:00:00 AM ET
      $BLUE
      $TSVT
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
      Biotechnology: Pharmaceutical Preparations

    $BLUE
    $VRTX
    Large Ownership Changes

    This live feed shows all institutional transactions in real time.

    See more
    • Amendment: SEC Form SC 13G/A filed by bluebird bio Inc.

      SC 13G/A - bluebird bio, Inc. (0001293971) (Subject)

      11/14/24 4:39:36 PM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Amendment: SEC Form SC 13G/A filed by bluebird bio Inc.

      SC 13G/A - bluebird bio, Inc. (0001293971) (Subject)

      11/14/24 4:27:32 PM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Amendment: SEC Form SC 13G/A filed by bluebird bio Inc.

      SC 13G/A - bluebird bio, Inc. (0001293971) (Subject)

      11/14/24 10:32:08 AM ET
      $BLUE
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $BLUE
    $VRTX
    FDA approvals

    Live FDA approvals issued by the Food and Drug Administration and FDA breaking news

    See more
    • January 30, 2025 - FDA Approves Novel Non-Opioid Treatment for Moderate to Severe Acute Pain

      For Immediate Release: January 30, 2025 Today, the U.S. Food and Drug Administration approved Journavx (suzetrigine) 50 milligram oral tablets, a first-in-class non-opioid analgesic, to treat moderate to severe acute pain in adults. Journavx reduces pain by targeting a pain-signaling pathway involving sodium channels in the peripheral nervous system, before pain signals reach the brain.  Journavx is the first dr

      1/30/25 5:46:14 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • December 8, 2023 - FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

      For Immediate Release: December 08, 2023 Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signali

      12/8/23 11:12:21 AM ET
      $BLUE
      $VRTX
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
      Biotechnology: Pharmaceutical Preparations
    • FDA Approval for TRIKAFTA (COPACKAGED) issued to VERTEX PHARMS INC

      Submission status for VERTEX PHARMS INC's drug TRIKAFTA (COPACKAGED) (SUPPL-11) with active ingredient ELEXACAFTOR, IVACAFTOR, TEZACAFTOR; IVACAFTOR has changed to 'Approval' on 08/03/2023. Application Category: NDA, Application Number: 212273, Application Classification: Labeling

      8/4/23 4:36:00 AM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $BLUE
    $VRTX
    Insider Trading

    Insider transactions reveal critical sentiment about the company from key stakeholders. See them live in this feed.

    See more
    • Director Upadhyay Suketu returned 1,001 shares to the company and was granted 796 shares, decreasing direct ownership by 8% to 2,354 units (SEC Form 4)

      4 - VERTEX PHARMACEUTICALS INC / MA (0000875320) (Issuer)

      5/5/25 5:00:58 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Director Thornberry Nancy was granted 398 shares, increasing direct ownership by 24% to 2,035 units (SEC Form 4)

      4 - VERTEX PHARMACEUTICALS INC / MA (0000875320) (Issuer)

      5/5/25 4:59:17 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Director Schneider Jennifer was granted 796 shares, increasing direct ownership by 86% to 1,718 units (SEC Form 4)

      4 - VERTEX PHARMACEUTICALS INC / MA (0000875320) (Issuer)

      5/5/25 4:57:54 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $BLUE
    $VRTX
    Financials

    Live finance-specific insights

    See more
    • Vertex Reports First Quarter 2025 Financial Results

      — Total revenue of $2.77 billion, a 3% increase compared to Q1 2024; raised the low end of total revenue guidance by $100 million to a new range of $11.85 to $12 billion — — Strong progress with CASGEVY®, ALYFTREK™ and JOURNAVX™ launches — — Povetacicept (pove) IgAN Phase 3 interim analysis (IA) cohort fully enrolled and zimislecel Phase 3 program to complete dosing this quarter, setting up potential filings in 2026; inaxaplin Phase 3 IA cohort on track to complete enrollment in the second half of 2025 — — Four programs already in pivotal development, with pivotal study of pove in primary membranous nephropathy (pMN) to start this year — Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)

      5/5/25 4:01:00 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Vertex to Announce First Quarter 2025 Financial Results on May 5th

      Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) will report its first quarter 2025 financial results on Monday, May 5, 2025 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the "Vertex Pharmaceuticals First Quarter 2025 Earnings Call." The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast

      4/7/25 4:05:00 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Vertex Reports Fourth Quarter and Full Year 2024 Financial Results

      — Full year product revenue of $11.02 billion, a 12% increase compared to full year 2023 — — Company provides full year 2025 total revenue guidance of $11.75 to $12.0 billion — — ALYFTREK™ approved in the U.S. for patients ages 6+ with cystic fibrosis; JOURNAVX™ approved in the U.S. for moderate-to-severe acute pain — — Diverse late-stage clinical pipeline accelerates with four programs in pivotal development — — Stuart Arbuckle, Chief Operating Officer, announces intent to retire July 1, 2025; as part of planned transition, Charlie Wagner, Chief Financial Officer, to assume additional role of Chief Operating Officer; Duncan McKechnie, SVP and current head of North America Commercia

      2/10/25 4:01:00 PM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $BLUE
    $VRTX
    Analyst Ratings

    Analyst ratings in real time. Analyst ratings have a very high impact on the underlying stock. See them live in this feed.

    See more
    • Vertex Pharma downgraded by Wolfe Research

      Wolfe Research downgraded Vertex Pharma from Outperform to Peer Perform

      5/7/25 8:37:12 AM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Vertex Pharma downgraded by Leerink Partners with a new price target

      Leerink Partners downgraded Vertex Pharma from Outperform to Market Perform and set a new price target of $503.00

      5/6/25 8:04:29 AM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Cantor Fitzgerald resumed coverage on Vertex Pharma with a new price target

      Cantor Fitzgerald resumed coverage of Vertex Pharma with a rating of Overweight and set a new price target of $535.00

      4/22/25 7:35:34 AM ET
      $VRTX
      Biotechnology: Pharmaceutical Preparations
      Health Care