Choroideremia

4DMT Reports Full Year 2023 Financial Results and Operational Highlights

Retrieved on: 
Thursday, February 29, 2024

We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027.

Key Points: 
  • We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027.
  • R&D Expenses: Research and development expenses were $97.1 million for 2023, as compared to $80.3 million for 2022.
  • G&A Expenses: General and administrative expenses were $36.5 million for 2023, as compared to $32.9 million for 2022.
  • Net Loss: Net loss was $100.8 million for 2023, as compared to net loss of $107.5 million for 2022.

Solid Biosciences Provides Third Quarter Business Update and Financial Results

Retrieved on: 
Wednesday, November 8, 2023

CHARLESTOWN, Mass., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the third quarter ended September 30, 2023, and provided a business update.

Key Points: 
  • “We are pleased with the continued advancement of our diversified pipeline of neuromuscular and cardiac gene therapies; including an IND submission in this quarter for SGT-003 for patients with Duchenne,” said Bo Cumbo, President and CEO of Solid Biosciences.
  • “We continue to expand our pipeline and last quarter licensed in a gene transfer program to treat catecholaminergic polymorphic ventricular tachycardia (CPVT).
  • Net loss for the third quarter of 2023 was $21.0 million, compared to $20.4 million for the third quarter of 2022.
  • Solid had $142.9 million in cash, cash equivalents, and available-for-sale securities as of September 30, 2023, compared to $213.7 million as of December 31, 2022.

Solid Biosciences Appoints Gabriel Brooks, M.D., as Chief Medical Officer

Retrieved on: 
Monday, October 2, 2023

CHARLESTOWN, Mass., Oct. 02, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for both neuromuscular and cardiac diseases, today announced the appointment of Gabriel Brooks, M.D., as Chief Medical Officer.

Key Points: 
  • “We are excited to welcome Dr. Brooks to Solid Biosciences during this pivotal time in our company’s history, advancing towards the clinic with our next generation gene therapy for Duchenne and furthering our diversified pipeline of both neuromuscular and cardiac gene therapies,” said Bo Cumbo, President and Chief Executive Officer of Solid Biosciences.
  • “Dr.
  • Brooks’ impressive accomplishments, specifically in cardiovascular genetic medicine, will bring valuable expertise and insights to our leadership team.
  • “Solid has a tremendous legacy of leadership in the gene therapy field and is deeply committed to developing and manufacturing novel gene therapies.

Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases

Retrieved on: 
Friday, May 12, 2023

While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

Key Points: 
  • While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.
  • Jean Bennett is a gene therapy expert and a professor emeritus of ophthalmology at the University of Pennsylvania.
  • She and her laboratory developed the first gene therapy drug for a genetic disease to be approved in the U.S.

What is gene therapy and how does it work?


    Gene therapy is a set of techniques that harness DNA or RNA to treat or prevent disease. Gene therapy treats disease in three primary ways: by substituting a disease-causing gene with a healthy new or modified copy of that gene; turning genes on or off; and injecting a new or modified gene into the body.

How has gene therapy changed how doctors treat genetic eye diseases and blindness?

    • In the past, many doctors did not think it necessary to identify the genetic basis of eye disease because treatment was not yet available.
    • Over time, we were able to create a treatment designed for individuals with particular gene defects that lead to congenital blindness.
    • Gene therapy treatments are now available in pharmacies and operating rooms all over the world.

You created one of the first gene therapies approved in the US. What is the current state of the clinical use of gene therapy?

    • There are now many approved gene therapies in the U.S., but the majority are combined with cell therapies in which a cell is modified in a dish and then injected back into the patient.
    • The majority of those therapies target different forms of cancer, although there are several for devastating inherited diseases.
    • The gene therapy that my team and I developed was the first FDA-approved project involving injection of a gene therapy directly into a person – in this case, into the retina.

What are you currently working on that you’re most excited about?

    • I am very excited about some upcoming clinical trials that my team will soon initiate to target some other devastating blinding diseases.
    • This test utilizes a virtual reality game that is not only fun for the user but promises to provide an objective measure of the person’s functional vision.

What are the biggest challenges gene therapy faces?

    • The biggest challenges involve systemic diseases, or diseases affecting the entire body rather than a single organ or body part.
    • For those diseases, super-high doses of gene therapy reagents must be delivered.
    • Cost remains a key issue in this effort – gene therapy drugs are enormously expensive.

4D Molecular Therapeutics Announces Updates on Clinical Pipeline and Additional Preclinical Programs

Retrieved on: 
Monday, January 9, 2023

The doses to be evaluated in DME are expected to be similar to those used in the 4D-150 wet AMD clinical trial.

Key Points: 
  • The doses to be evaluated in DME are expected to be similar to those used in the 4D-150 wet AMD clinical trial.
  • It is estimated that there are over one million individuals with GA in the United States according to published data.
  • In addition, I’m excited by the potential of 4D-175, the new preclinical product candidate utilizing the same R100 vector.
  • Cardiac clinical endpoint data (MRI, echocardiography, cardiopulmonary exercise testing [CPET] and QOL assessment) from evaluations at baseline and 12 months after treatment were assessed.

4D Molecular Therapeutics to Participate in the Upcoming Evercore ISI HealthCONx 2022 Conference

Retrieved on: 
Monday, November 21, 2022

EMERYVILLE, Calif., Nov. 21, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced that David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT, will participate in a fireside chat at the Evercore ISI HealthCONx 2022 Conference.

Key Points: 
  • EMERYVILLE, Calif., Nov. 21, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced that David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT, will participate in a fireside chat at the Evercore ISI HealthCONx 2022 Conference.
  • The fireside chat will take place on Wednesday, November 30, 2022 at 3:55 p.m.
  • A live audio webcast of the fireside chat will be available by visiting the Investors & Media section of the 4DMT website at www.4dmoleculartherapeutics.com .
  • 4D Molecular Therapeutics, 4DMT, Therapeutic Vector Evolution, and the 4DMT logo are trademarks of 4DMT.

4D Molecular Therapeutics Announces Interim Clinical Data from On-going Phase 1/2 Clinical Trial of Intravitreal 4D-150 for Wet Age-Related Macular Degeneration (wet AMD)

Retrieved on: 
Monday, November 14, 2022

4D Molecular Therapeutics will host a conference call today, Monday November 14, 2022 at 8:00 AM E.T.

Key Points: 
  • 4D Molecular Therapeutics will host a conference call today, Monday November 14, 2022 at 8:00 AM E.T.
  • This clinical data on 4D-150 marks an important milestone for 4DMT, said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • We are developing 4D-150 for the treatment of large and sustainable markets in ophthalmology, including wet AMD and diabetic macular edema.
  • I am looking forward to enrolling patients in the randomized Phase 2 expansion stage of this clinical trial.

Choroideremia Drug Pipeline Insights Report 2022 Featuring Spark Therapeutics, Molecular Therapeutics, Curative Biotechnology, & Ray Therapeutics - ResearchAndMarkets.com

Retrieved on: 
Wednesday, November 16, 2022

This report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Choroideremia pipeline landscape.

Key Points: 
  • This report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Choroideremia pipeline landscape.
  • A detailed picture of the Choroideremia pipeline landscape is provided which includes the disease overview and Choroideremia treatment guidelines.
  • The assessment part of the report embraces, in depth Choroideremia commercial assessment and clinical assessment of the pipeline products under development.
  • The companies which have their Choroideremia drug candidates in the most advanced stage, i.e phase I/II include Spark Therapeutics
    Choroideremia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.

4D Molecular Therapeutics Interim Clinical Data from the On-going Phase 1/2 Clinical Trial of 4D-150 for Wet AMD to be Released Monday, November 14, 2022

Retrieved on: 
Thursday, November 10, 2022

4D Molecular Therapeutics also announced that interim clinical data from cohort 1 of this Phase 1/2 clinical trial will be released at 7:30 am E.T., on Monday, November 14, 2022.

Key Points: 
  • 4D Molecular Therapeutics also announced that interim clinical data from cohort 1 of this Phase 1/2 clinical trial will be released at 7:30 am E.T., on Monday, November 14, 2022.
  • 4D will host a conference call and live webcast on November 14th, 2022, at 8:00 am E.T.
  • 4D Molecular Therapeutics will host a conference call and live webcast on November 14, 2022, at 8:00 am E.T.
  • 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements.

4D Molecular Therapeutics Reports Third Quarter 2022 Financial Results

Retrieved on: 
Wednesday, November 9, 2022

EMERYVILLE, Calif., Nov. 09, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics, Inc. (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today reported third quarter 2022 financial results.

Key Points: 
  • Revenue: Total revenue for the quarter ended September 30, 2022, was $0.5 million, as compared to $1.4 million for the quarter ended September 30, 2021.
  • Net Loss: Net loss was $25.7 million for the quarter ended September 30, 2022, as compared to $22.2 million for the quarter ended September 30, 2021.
  • 4D Molecular Therapeutics, 4DMT, Therapeutic Vector Evolution, and the 4DMT logo are trademarks of 4DMT.
  • Our results for the quarter ended September 30, 2022, are also not necessarily indicative of our operating results for any future periods.