Research Portfolio Archives - CureDuchenne https://staging.cureduchenne.org/./research-portfolio Together, we WILL cure Duchenne Wed, 26 Jan 2022 20:31:13 +0000 en-US hourly 1 https://wordpress.org/?v=6.7.2 https://staging.cureduchenne.org/wp-content/uploads/2019/06/favicon-1-100x100.png Research Portfolio Archives - CureDuchenne https://staging.cureduchenne.org/./research-portfolio 32 32 CureDuchenne Ventures Commits $500,000 to 4D Molecular Therapeutics to Advance Next-Generation AAV Delivery of Gene Therapy https://staging.cureduchenne.org/press-release/4d-molecular-therapeutics-gene-therapy Fri, 07 Sep 2018 17:05:07 +0000 https://www.cureduchenne.org/?p=10511 NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D […]

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NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, in an effort to advance gene therapy.

“Our investment into 4DMT will be the fourth investment by CureDuchenne Ventures, the investment arm of CureDuchenne, into gene therapy and gene editing. So far, we have had preclinical success in this area for Duchenne with our investments in duplication 2 exon skipping at Nationwide Children’s Hospital, Bamboo Therapeutics/Pfizer and Exonics Therapeutics, and we are very positive about the prospect of 4DMT’s work,” said Debra Miller, founder and CEO of CureDuchenne. “CureDuchenne’s investments are moving quickly through the drug development process and we look forward to safety and efficacy data for these potential therapies.”

The Duchenne community is aware of possible limitations of the current delivery system for gene therapy and gene editing due to pre-existing neutralizing antibodies, and manufacturing challenges due to the large amount of vector needed to treat the entire muscular system in the body.

4DMT focuses on the discovery and development of targeted, customized and proprietary next-generation AAV gene therapy products for use in patients with severe genetic diseases with high unmet medical need. Therapeutic Vector Evolution empowers 4DMT to create customized gene therapy products to deliver genes specifically to any tissue or organ in the body, by the optimal clinical route of administration for any given disease, at lower doses and with resistance to pre-existing antibodies.

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Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans. https://staging.cureduchenne.org/blog/wired_article_crispr_cureduchenne_8-30-2018 Thu, 30 Aug 2018 18:48:37 +0000 https://www.cureduchenne.org/?p=10129 Dear Friends, Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in […]

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Dear Friends,

Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and to provide the seed funding necessary to accelerate his work in this company, we could not be more thrilled. This is a true testament to the power of venture philanthropy and I want to thank each and every donor and supporter who has believed in our work, and we are grateful to Dr. Olson for his dedication to Duchenne patients. Although we still have a long way to go before we confirm this therapy will be safe and effective in humans, this dramatic dystrophin restoration offers hope to all families affected by this disease.

The story began in 2015 when news of CRISPR technology first began surfacing. The CureDuchenne science and investment teams started to research and talk to different scientists. We met with a couple different groups before landing on Dr. Eric Olson, who had a long history of working in muscular disorders and had a true entrepreneurial spirit, which is crucial when working in this space.

After several meetings with UT Southwestern and Dr. Olson, we came to an agreement that the fastest way to accelerate CRISPR technology to human clinical trials was to form a company to fast track development. That’s when the three groups worked together in a truly collaborative partnership to form Exonics.

Within a matter of weeks, CureDuchenne committed $5 million in seed money to Exonics. We also put together an LLC to secure funding from other Duchenne organizations to help us to raise the $5 million, which we did not have, but every organization we talked to turned us down because they did not believe enough in the research project. Thankfully, after our first payment of $2 million, which was every penny we had, The Column Group came in with Series A funding and we no longer needed to fund the remaining $3 million.

With a mission to find, foster and fund investments in early stage projects, which many VCs and other investment groups avoid because of the high risk involved, this potential success story proves the incredible value in being a knowledgeable risk taker when it comes to finding cures for rare diseases.

On average, a drug takes 10 years to get to FDA approval after three years of clinic trials. With an average lifespan into the mid-20s, those with Duchenne need solutions faster. CureDuchenne Ventures’ goal is to identify the best science and to accelerate drug development timelines. We will continue to operate with the highest standards of ethics and integrity by re-investing the earnings back into research and programs that help support the whole journey of a Duchenne patient until we have a cure.

Read the full article HERE

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TRiNDS https://staging.cureduchenne.org/research-portfolio/trinds Thu, 16 Feb 2017 01:29:39 +0000 https://www.cureduchenne.org/?p=4024 Approach: Neuromuscular focused Contract Research Organization
Funding Impact: Clinical trial support for Biotech/Pharma from a patient-centric perspective

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TRiNDS is a new contract research organization born from the Cooperative International Neuromuscular Research Group (CINRG) academic consortium. Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS) provides specialized clinical trial support and post marketing solutions for neuromuscular, particularly Duchenne, clinical research.

Research focus: Clinical trial support

Status: TRiNDS is involved in all phases of the clinical trial process and can help guide the development of neuromuscular clinical research studies.

Website: www.trinds.com

TRiNDS announcement release

“We are proud to fill an emerging need in drug development for rare and orphan diseases like Duchenne. We are honored to collaborate with CureDuchenne Ventures who continues to be an innovator in funding Duchenne research and provides a strong link to the Duchenne community.”  — Lauren Morgenroth, Chief Operating Officer of TRiNDS.

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Summit https://staging.cureduchenne.org/research-portfolio/summit Thu, 16 Feb 2017 01:26:56 +0000 https://www.cureduchenne.org/?p=4021 Approach: Utrophin upregulation
Funding Impact: Enabled initiation of Phase 2 clinical trial

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Summit Therapeutics is a clinical-stage drug discovery and development company advancing innovative therapies to significantly advance the current standard of treatment for serious unmet medical needs such as Duchenne.

Research focus: Utrophin upregulation.

Status: Phase 2

Website: http://www.summitplc.com/

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Sarepta Therapeutics https://staging.cureduchenne.org/research-portfolio/sarepta-therapeutics Thu, 16 Feb 2017 01:24:55 +0000 https://www.cureduchenne.org/?p=4018 Approach: Exon skipping
Funding Impact: First drug approved by FDA to treat Duchenne

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Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases.

Research focus: Exon Skipping.

Status: Accelerated approval of EXONDYS 51 (eteplirsen) by FDA.  Other candidates for exon skipping in various stages of clinical development.

Website:  https://www.sarepta.com

Approval press release

“CureDuchenne helped us very early on in in the development of the program. They gave us a jump start on clinical trial development.  This is a very long journey. I would look at eteplirsen as the first step in a long process of trying to get better and better drugs for this community.” —  Ed Kaye, M.D., CEO and Chief Medical Officer of Sarepta.

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PTC Therapeutics https://staging.cureduchenne.org/research-portfolio/ptc-therapeutics Thu, 16 Feb 2017 01:16:41 +0000 https://www.cureduchenne.org/?p=4008 Approach: Stop codon read-through
Funding Impact: Conditional approval of Translanrna in EU

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PTC Therapeutics is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology they refer to as post-transcription control.

Research focus: Stop Codon Readthrough.

Status:  Clinical.  Conditional approval in the EU.

Website: http://www.ptcbio.com/en/

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Prosensa/BioMarin https://staging.cureduchenne.org/research-portfolio/prosensabiomarin Thu, 16 Feb 2017 01:08:03 +0000 https://www.cureduchenne.org/?p=4000 Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015

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BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders.

Research focus: Exon Skipping

Status: The clinical program has been halted.

Website: http://www.biomarin.com

Venture philanthropy timeline and case study

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cTAP https://staging.cureduchenne.org/research-portfolio/ctap Thu, 16 Feb 2017 00:58:11 +0000 https://www.cureduchenne.org/?p=3991 Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design

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The Collaborative Trajectory Analysis Project (cTAP) brings together all the stakeholders in Duchenne Muscular Dystrophy to solve the critical problems in Duchenne drug development with a sense of urgency for the thousands of patients who are waiting for treatments.

Research focus: Statistical analysis/Duchenne Natural History/Clinical trial design

Website: http://ctap-duchenne.org

cTAP announcement release

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Nationwide Children’s Hospital https://staging.cureduchenne.org/research-portfolio/nationwide-childrens-hospital Thu, 16 Feb 2017 00:43:26 +0000 https://www.cureduchenne.org/?p=3985 Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys

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Led by Kevin Flanigan, MD at Nationwide Children’s Hospital, this investigator initiated therapy will focus on genetic and molecular characterization of inherited neuromuscular diseases and toward the development of therapies directed toward these diseases.

Research focus: Exon skipping (Duplication).

Status: Pre-clinical. Submitting pre-IND package to FDA.

Website: https://staging.cureduchenne.org/flanigan/

Recent Funding

Funding Need: $500,000 – In collaboration with Duchenne community partners in initial funding. Total of $1.4M including scale-up of a gene-therapy delivery vector needed for a clinical trial.

DONATE NOW

“CureDuchenne recognized early on the importance of our research focused on rare and duplication mutations and we wouldn’t be here today without their support. CureDuchenne supported the development of the mouse model and the personnel to work on this research. CureDuchenne plays a vital role in funding early phase research projects to help accelerate their development.” — Dr. Kevin Flanigan

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RASRx https://staging.cureduchenne.org/research-portfolio/rasrx Tue, 01 Nov 2016 23:43:53 +0000 https://www.cureduchenne.org/?p=2534 Approach: Anti-fibrotic
Funding Impact: Accelerated identification of lead compound and de-risked pre-clinical program

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RASRx is a start-up company dedicated to developing therapies for rare diseases. RASRx will develop and commercialize technology licensed from the University of Southern California.

Research focus: Anti-inflammatory.

Status: Preclinical

Status: https://www.rasrx.com

RASRx announcement release

“We are passionate about making an impact in healthcare in areas of unmet needs like Duchenne muscular dystrophy.  Our lead program at RASRx, which has previously received DoD funding, takes a mutation independent approach to slowing and potentially reversing disease progression to bring relief to patients with Duchenne, and possibly other muscular dystrophies. We are thrilled by the potential of this relationship as CureDuchenne’s knowledge of the disease and familiarity with the needs of the patients are invaluable as we move forward in the development of this promising treatment.”   — Kathleen Rodgers, a founder of RASRx

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