Autoimmunity

Kymera Therapeutics Outlines Key 2024 Objectives and Strategy to Progress Leading Portfolio of Immunology and Oncology Programs

Retrieved on: 
Tuesday, January 9, 2024

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Key Points: 
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    WATERTOWN, Mass., Jan. 09, 2024 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of small molecule medicines using targeted protein degradation (TPD), today announced its corporate goals for 2024, including anticipated progress on its best-in-class pipeline of immunology and oncology programs.
  • Sanofi, which is collaborating with Kymera on the development of KT-474 outside of the oncology and immune-oncology fields, is conducting the Phase 2 studies.
  • KT-621 has potentially broad utility across a number of allergic diseases, including atopic dermatitis, asthma and chronic obstructive pulmonary disorder, among others.
  • Kymera is working to develop a biomarker-based patient selection strategy for subsequent development beyond Phase 1a.

European Medicines Agency (EMA) Greenlights Mitochon Pharmaceuticals to Initiate Phase I/IIa Biomarker Study in Neurodegenerative Diseases

Retrieved on: 
Tuesday, January 9, 2024

Mitochon Pharmaceuticals today announced that it was awarded approval from EMA to begin enrollment for a Phase I/IIa biomarker study in Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Huntington’s Disease (HD) and Alzheimer’s Disease (AD) participants.

Key Points: 
  • Mitochon Pharmaceuticals today announced that it was awarded approval from EMA to begin enrollment for a Phase I/IIa biomarker study in Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Huntington’s Disease (HD) and Alzheimer’s Disease (AD) participants.
  • Mitochon’s MP101, a once-daily, oral brain penetrant mitochondrial stimulator, that improves central nervous system survival and function will be used in this 14-day pilot study.
  • The aim of the study is to show safety in the target patient populations and demonstrate meaningful changes in disease specific biomarkers.
  • “We are delighted for the opportunity to explore this provocative idea that most, if not all neurodegenerative diseases are rooted in mitochondrial dysfunction.

Viridian Therapeutics to Present Key 2024 Corporate Priorities at J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024

As part of Viridian’s presentation at the J.P. Morgan Healthcare Conference, Mr. Mahoney will provide key updates on the company’s anti-insulin-like growth factor 1 receptor (IGF-1R) programs in thyroid eye disease (TED) and its programs targeting FcRn.

Key Points: 
  • As part of Viridian’s presentation at the J.P. Morgan Healthcare Conference, Mr. Mahoney will provide key updates on the company’s anti-insulin-like growth factor 1 receptor (IGF-1R) programs in thyroid eye disease (TED) and its programs targeting FcRn.
  • Viridian is currently evaluating VRDN-001 in two global Phase 3 clinical trials, THRIVE and THRIVE-2, for the treatment of active and chronic TED, respectively.
  • The company expects to report THRIVE and THRIVE-2 data in the middle of 2024 and by year end 2024, respectively.
  • The Inducement Grant was granted as an inducement material to this individual entering into employment with Viridian in accordance with Nasdaq Listing Rule 5635(c)(4).

ElevateBio to Highlight Growth of its Genetic Medicine Business Driven By Gene Editing and Manufacturing Partnerships at the 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024

Chairman and Chief Executive Officer, David Hallal, will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on January 8, 2024 at 9:30 am PT.

Key Points: 
  • Chairman and Chief Executive Officer, David Hallal, will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on January 8, 2024 at 9:30 am PT.
  • Announced two transformative R&D collaborations with Moderna and Novo Nordisk to discover and develop gene editing therapeutics.
  • BaseCamp is ElevateBio’s genetic medicine process development and cGMP business enabling biopharmaceutical partners with its end-to-end capabilities.
  • Developed additional C-base editors for gene knock-outs in novel cell therapies and A-base editors for in vivo gene editing therapeutics.

Incyte Highlights Growth Opportunities and Provides Business Updates at the 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024

Incyte (Nasdaq:INCY) will highlight growth opportunities and provide key updates across its investigational pipeline and commercial portfolio during a presentation today at 7:30 a.m. PT at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco.

Key Points: 
  • Incyte (Nasdaq:INCY) will highlight growth opportunities and provide key updates across its investigational pipeline and commercial portfolio during a presentation today at 7:30 a.m. PT at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco.
  • The overall safety profile of ruxolitinib cream is consistent with previous data, and no new safety signals were observed.
  • The Company intends to provide additional financial guidance and updates on key clinical programs during its 2023 fourth quarter and year-end earnings conference call.
  • The J.P. Morgan Healthcare Conference presentation and Q&A session can be accessed at investor.incyte.com .

CRISPR Therapeutics Highlights Strategic Priorities and 2024 Outlook

Retrieved on: 
Monday, January 8, 2024

ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth.

Key Points: 
  • ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth.
  • Exa-cel is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals.
  • Vertex leads global development, manufacturing, regulatory and commercialization of CASGEVY with support from CRISPR Therapeutics.
  • CRISPR Therapeutics continues to focus on resource efficiency and return on invested capital as it advances multiple clinical programs across its pipeline.

Cartesian Therapeutics Highlights Progress and 2024 Strategic Priorities Across Innovative Pipeline of mRNA Cell Therapies for Autoimmunity

Retrieved on: 
Monday, January 8, 2024

The Company’s RNA-engineering approach is designed to expand the reach of cell therapy to autoimmunity with potent therapies that can be dosed more reliably and safely in an outpatient setting without lymphodepletion.

Key Points: 
  • The Company’s RNA-engineering approach is designed to expand the reach of cell therapy to autoimmunity with potent therapies that can be dosed more reliably and safely in an outpatient setting without lymphodepletion.
  • Cartesian’s proprietary technology platform, RNA Armory®, is designed to enable precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities, including autologous, allogeneic, and in situ transfection.
  • Descartes-08 is an autologous anti-B cell maturation antigen (BCMA) mRNA CAR-T.
  • Cartesian continues to anticipate the initiation of Phase 2 basket studies in additional autoimmune indications in the second half of 2024.

Cartesian Therapeutics Announces Positive Long-Term Follow-Up Data from Phase 2a Study of Lead mRNA Cell Therapy Candidate Descartes-08 in Patients with Myasthenia Gravis

Retrieved on: 
Monday, January 8, 2024

GAITHERSBURG, Md., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC), (“the Company”) a clinical-stage biotechnology company pioneering mRNA cell therapies for autoimmune diseases, today announced positive twelve-month follow-up data from its Phase 2a trial of Descartes-08 in patients with generalized myasthenia gravis (MG), a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. The manuscript titled, “Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy,” has been submitted for peer-review and can be accessed on the online preprint server, medRxiv.

Key Points: 
  • The manuscript titled, “Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy,” has been submitted for peer-review and can be accessed on the online preprint server, medRxiv.
  • Descartes-08, Cartesian’s lead mRNA cell therapy candidate and a potential first-in-class mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T), is an autologous anti-B-cell maturation antigen (BCMA) mRNA CAR-T.
  • “Notably, most patients maintained robust, clinically meaningful improvements across all four standard MG severity scores approximately 10 months after the last infusion.
  • Enrollment is ongoing in a Phase 2b randomized, double-blind, placebo-controlled trial (NCT04146051) in patients with MG. Topline results are expected in mid-2024.

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
Monday, January 8, 2024

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.

Barinthus Bio Provides a Financial Update and Announces Anticipated 2024 Corporate Milestones

Retrieved on: 
Friday, January 5, 2024

OXFORD, United Kingdom, Jan. 05, 2024 (GLOBE NEWSWIRE) -- Barinthus Biotherapeutics plc (NASDAQ: BRNS), formerly Vaccitech plc, today provided a preliminary financial update and announced its 2024 corporate objectives.

Key Points: 
  • OXFORD, United Kingdom, Jan. 05, 2024 (GLOBE NEWSWIRE) -- Barinthus Biotherapeutics plc (NASDAQ: BRNS), formerly Vaccitech plc, today provided a preliminary financial update and announced its 2024 corporate objectives.
  • Barinthus Bio is a clinical-stage biopharmaceutical company developing novel T cell immunotherapeutic candidates designed to guide the immune system to overcome chronic infectious diseases, autoimmunity, and cancer.
  • “2024 promises to be another exciting year for Barinthus Bio, with multiple data readouts expected across our hepatitis B virus (HBV) infection, human papillomavirus (HPV) infection and prostate cancer programs, as well as the planned initiation of the first in human study of our SNAP platform-based candidate VTP-1000 in Celiac Disease,” said Gemma Brown, Chief Financial Officer of Barinthus Bio.
  • * Barinthus Bio has worldwide rights for all product candidates.