Passage Bio and Invitae Announce Collaboration to Facilitate Genetic Testing to Support Early Diagnosis and Greater Awareness of Clinical Trials for Patients with GM1
Passage Bio (NASDAQ: PASG) partners with Invitae to enhance genetic testing for GM1 gangliosidosis. This collaboration offers free genetic testing and counseling through Invitae’s Detect LSDs program, promoting early diagnosis and access to clinical trials. The initiative aims to support patients, especially infants, as GM1's severe form has a life expectancy of only 2-4 years. Passage Bio plans to initiate its Phase 1/2 trial for PBGM01 shortly, positioning the partnership as crucial for early intervention and treatment.
- Collaboration with Invitae to provide no-cost genetic testing and counseling, facilitating earlier diagnosis of GM1 gangliosidosis.
- Support for patients and families with educational clinical trial information.
- Planned initiation of Phase 1/2 trial for PBGM01, potentially advancing treatment options.
- GM1 gangliosidosis has no currently approved disease-modifying therapies, indicating a high unmet medical need.
- Challenges related to the disease's severe progression and limited life expectancy for affected infants.
Genetic testing, educational materials and counseling offered at no charge to patients
PHILADELPHIA, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, and Invitae (NYSE: NVTA), a leading medical genetics company, announce a collaboration to facilitate genetic testing and support early identification of GM1 gangliosidosis (GM1) through Invitae’s Detect Lysosomal Storage Disorders (Detect LSDs). The two companies also are partnering to provide educational clinical trial information to clinicians and patients.
“Early identification and intervention are crucial steps in providing effective treatment to patients with GM1, particularly in the infantile form where onset occurs within the first six months of a patient’s life,” said Bruce Goldsmith, Ph.D, president and chief executive officer of Passage Bio. “By combining Invitae’s no-charge testing and counseling with information on clinical trials, clinicians will be able to intervene sooner and more efficiently, shortening the timeline to reliable diagnosis. As we plan to initiate our Phase 1/2 trial for PBGM01 soon, this partnership will be an important part of our support for patients and will also serve as a key resource to patients with GM1 and their families.”
The Detect LSDs program offers genetic testing and genetic counseling at no charge to patients to encourage earlier diagnosis of lysosomal storage disorders like GM1 and, as a result, earlier access to clinical trials. In addition, Invitae provides clinical trial information and education to clinicians and patients who may benefit. Currently, the Detect LSDs program is available to patients within the United States and Canada.
“Increasing access to genetic testing supports earlier diagnosis, enables clinical trials to develop new treatments and helps clinicians provide precision therapies sooner for better overall outcomes,” said Robert Nussbaum, M.D., chief medical officer of Invitae. “We’re pleased Passage Bio has joined us in this effort.”
Additional details, as well as terms and conditions of the Detect LSDs program, can be found at https://www.invitae.com/en/detectLSDs/.
About GM1
GM1 gangliosidosis (GM1) is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (β-gal). Reduced β-gal activity results in the accumulation of toxic levels of GM1 ganglioside in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the Infantile form, which is characterized by onset in the first 6 months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately
About Passage Bio
At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania’s Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with unparalleled access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.
About Invitae
Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
For further information, please contact:
Investors:
Sarah McCabe and Zofia Mita
Stern Investor Relations, Inc.
sarah.mccabe@sternir.com and zofia.mita@sternir.com
Media:
Media:
Gwen Fisher
Passage Bio
215-407-1548
gfisher@passagebio.com
FAQ
What is the purpose of Passage Bio's collaboration with Invitae regarding GM1 gangliosidosis?
When is Passage Bio planning to initiate its Phase 1/2 trial for PBGM01?
What is GM1 gangliosidosis and why is early diagnosis important?
How does the Detect LSDs program benefit patients with GM1?
Where is the Detect LSDs program available?
