Beta thalassemia

Editas Medicine Receives FDA Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Beta Thalassemia

Retrieved on: 
Tuesday, April 26, 2022
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CAMBRIDGE,Mass., April 26, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leadinggenome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia.

Key Points: 
  • CAMBRIDGE,Mass., April 26, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leadinggenome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia.
  • The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease.
  • Receiving Rare Pediatric Disease designation for EDIT-301 for beta thalassemia highlights the dire need for new treatment options, said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine.
  • The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years.

DGAP-News: BB BIOTECH AG: Increased investment level balanced by established core holdings

Retrieved on: 
Friday, April 22, 2022
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As a consequence of the general sector correction the investment level increased to 113.0% (previous quarter: 110.9%).

Key Points: 
  • As a consequence of the general sector correction the investment level increased to 113.0% (previous quarter: 110.9%).
  • For the first quarter 2022, BB Biotech shares' total return was -11.3% in CHF and -8.6% in EUR.
  • The team will continue to work within the established and successful investment guidelines, such as investment levels of 95% to 115%.
  • BB Biotech expects the FDA to continue its constructive, purposeful but moderating interactions with the biopharmaceutical industry under his leadership.

Vertex to Announce First Quarter 2022 Financial Results on May 5

Retrieved on: 
Thursday, April 21, 2022
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Beta thalassemia, Pain, Vertex, CF, Vertex Pharmaceuticals, Duchenne muscular dystrophy, Twitter, Research, International, Cystic fibrosis, Duchenne, YouTube, VRTX, Sickle cell disease, LinkedIn, Webcast, Biology, Instagram, Pharmaceutical industry, Innovation district, Science, Facebook

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2022 financial results on Thursday, May 5, 2022 after the financial markets close.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2022 financial results on Thursday, May 5, 2022 after the financial markets close.
  • To access the call, please dial (877) 270-2148 (U.S.) or +1 (412) 902-6510 (International) and reference the Vertex Pharmaceuticals First Quarter 2022 Earnings Call.
  • To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

Vertex Announces Statistically Significant and Clinically Meaningful Results From Two Phase 2 Proof-of-Concept Studies of VX-548 for the Treatment of Acute Pain

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Thursday, March 31, 2022
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Two patients in the mid-dose VX-548 arm discontinued treatment due to AEs, none of which were related to treatment.

Key Points: 
  • Two patients in the mid-dose VX-548 arm discontinued treatment due to AEs, none of which were related to treatment.
  • The remarkable consistency in the safety, tolerability and efficacy results in these two studies demonstrate the potential of VX-548 to be a first-in-class non-opioid treatment for acute pain.
  • NaV1.8 is a genetically validated target for the treatment of pain, and Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8 in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Fulcrum Therapeutics® Reports Recent Business Highlights and Fourth Quarter and Full Year 2021 Financial Results

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Thursday, March 3, 2022
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CAMBRIDGE, Mass., March 03, 2022 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the fourth quarter and full year of 2021.

Key Points: 
  • Collaboration Revenue: Collaboration revenue was $5.1 million for the fourth quarter of 2021, as compared to $4.2 million for the fourth quarter of 2020.
  • R&D Expenses: Research and development expenses were $18.9 million for the fourth quarter of 2021, as compared to $16.1 million for the fourth quarter of 2020.
  • G&A Expenses: General and administrative expenses were $9.7 million for the fourth quarter of 2021, as compared to $5.9 million for the fourth quarter of 2020.
  • ET to discuss the Companys fourth quarter and full year 2021 recent business highlights and financial results.

Vertex to Present at Cowen’s 42nd Annual Health Care Conference on March 8

Retrieved on: 
Tuesday, March 1, 2022
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Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at Cowens 42nd Annual Health Care Conference on Tuesday, March 8, 2022 at 2:50 p.m.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at Cowens 42nd Annual Health Care Conference on Tuesday, March 8, 2022 at 2:50 p.m.
  • A live webcast of management's remarks will be available through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page.
  • A replay of the conference webcast will be archived on the company's website.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

DGAP-News: BB BIOTECH AG: 2021 was a difficult year for biotech equity markets - increased dividend of CHF 3.85 proposed

Retrieved on: 
Friday, January 21, 2022
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Media release as at January 21, 2022

Key Points: 
  • Media release as at January 21, 2022
    Portfolio of BB Biotech AG as at December 31, 2021
    The year 2021 was a volatile market environment for all involved in biotechnology investing, including BB Biotech.
  • BB Biotech carefully adjusted the portfolio in the last three months of 2021.
  • The net loss for BB Biotech in 2021 was CHF 405 mn.
  • The highly specialized investment team at BB Biotech believes that the growth case for biotechnology and valuations for portfolio companies are both compelling.

Vertex to Announce Fourth Quarter 2021 Financial Results on January 26

Retrieved on: 
Tuesday, January 18, 2022
Biotechnology, Pharmaceutical, Genetics, Health, Webcast, Research, Vertex, LinkedIn, Vertex Pharmaceuticals, International, Biology, Human Rights Campaign, VRTX, CF, Duchenne muscular dystrophy, Beta thalassemia, Instagram, Pain, Sickle cell disease, YouTube, LGBTQ, Twitter, Cystic fibrosis, Pharmaceutical industry, Innovation district, Science, Facebook

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2021 financial results on Wednesday, January 26, 2022 after the financial markets close.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2021 financial results on Wednesday, January 26, 2022 after the financial markets close.
  • To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).
  • To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

Vertex to Present at the 40th Annual J.P. Morgan Healthcare Conference on January 10

Retrieved on: 
Tuesday, January 4, 2022
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Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 9:00 a.m.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that management will present at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 9:00 a.m.
  • A live webcast of management's remarks will be available through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page.
  • A replay of the conference webcast will be archived on the company's website.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

Editas Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for EDIT-301 for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Monday, December 20, 2021
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CAMBRIDGE, Mass., Dec. 20, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND for EDIT-301 for the treatment of transfusion-dependent beta thalassemia (TDT), enabling the Company to initiate a Phase 1/2 clinical study of EDIT-301 in TDT patients.

Key Points: 
  • The FDAs clearance of our IND for EDIT-301 for TDT is an important milestone for Editas and for the patients we hope to serve.
  • With this IND clearance, Editas Medicine is preparing to initiate a Phase 1/2 trial designed to assess the safety, tolerability, and preliminary efficacy of EDIT-301 for the treatment of TDT.
  • Based on clinical severity and transfusion requirements, beta thalassemia can be classified into non-transfusion-dependent (NTDT) and transfusion-dependent beta thalassemia.
  • EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).